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FINAL PROGRAM - American Society of Gene & Cell Therapy

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<strong>FINAL</strong> <strong>PROGRAM</strong><strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>13 th Annual Meeting | Washington, DC USA May 19-22, 2010Marriott Wardman ParkASGCT-1209-111Jointly sponsored by Indiana University School <strong>of</strong> Medicine and the <strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>www.asgct.org


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 1Table <strong>of</strong> ContentsA Welcome from Kenneth Cornetta, MD .. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 2Committee Meeting Schedule .. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 3Committee Rosters. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 4Planning Committee and Group Coordinating Reviewers. . . . . . . . . . . . . . . . . . . . . . . . . . 8Annual Meeting Supporters.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 9Annual Meeting Exhibitors.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10Abstract Reviewers.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11Excellence in Research Award and Travel Grant Recipients. . . . . . . . . . . . . . . . . . . . . . . . 13Outstanding New Investigator & Outstanding Achievement Award Recipients. . . . . . . . . . . 14Faculty Listing.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 15<strong>Gene</strong>ral Meeting Information.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19Marriott Wardman Park Hotel Floor Plans .. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 23Schedule-at-a-Glance. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 25Detailed Program Schedule.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 27Exhibit Hall Floor Plan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 76Exhibitor Descriptions.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 77Exhibitor Information.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 84Washington, DC Fun Facts. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 85Housing Information and Map <strong>of</strong> Hotel Area... . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 85Abstract Directory. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 86Abstract Author Index.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 143ASGCT Officers and Board <strong>of</strong> DirectorsPresident (09/10)Kenneth Cornetta, MDPresident-Elect (09/10)Barrie J. Carter, PhDVice President (09/10)R. Jude Samulski, PhDSecretary (10)John F. Engelhardt, PhDTreasurer (11)Beverly L. Davidson, PhDBoard MembersChristopher Baum, MDMichele P. Calos, PhDCynthia Dunbar, MDHelen E. Heslop, MDHarry L. Malech, MDStephen J. Russell, MD, PhDBrian P. Sorrentino, MDSamuel C. Wadsworth, PhDMatthew D. Weitzman, PhDJohn J. Rossi, PhDChair, Advisory Council(ex-<strong>of</strong>ficio)Malcolm K. Brenner, MD, PhDEditor-in-Chief, Molecular <strong>Therapy</strong>(ex-<strong>of</strong>ficio)ASGCT Executive Office555 East Wells Street, Suite 1100Milwaukee, WI 53202Phone: 414.278.1341Fax: 414.276.3349info@asgct.orgwww.asgct.orgMary Dean – Executive DirectorErin Hankey – Administrative AssistantKen Janowski – Program ManagerRoseann Marotz – Meetings ManagerDavid Wood – Senior Meetings & Membership ManagerTara Withington, CAE – Consulting Partner


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 3Committee Meeting ScheduleCommittee terms technically commence and conclude at the Annual Meeting. Incoming chair and committee member terms <strong>of</strong>ficially go into effect on May 22, 2010at the Business Meeting. All meetings will be held at the Marriott Wardman Park Hotel unless otherwise noted.WEDNESDAY, MAY 19th11:45 am – 12:45 pm Membership Committee Meeting Harding RoomTHURSDAY, MAY 20thInternational Committee MeetingHoover Room12:45 pm – 1:45 pm Education Committee Meeting Park Tower 8216FRIDAY, MAY 21stPublications Committee Meeting Park Tower 821212:30 pm – 1:30 pm Scientific Committee Meetings:Cancer <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>; Cardiovascular <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>;Embryonic/Somatic Stem <strong>Cell</strong> and Tissue Engineering; <strong>Gene</strong> &<strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> <strong>Gene</strong>tic Diseases; <strong>Gene</strong>tic Vaccines; Hemopoietic<strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong>; Immunology <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>; InfectiousDiseases; Musculo-Skeletal Disorders <strong>Gene</strong> <strong>Therapy</strong>; NeuralDisorders <strong>Gene</strong> <strong>Therapy</strong>; Oligonucleotide Based Therapies; Physical<strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> and Vectorology; Respiratory Tract <strong>Gene</strong> & <strong>Cell</strong><strong>Therapy</strong>; Tissue Engineering; Viral <strong>Gene</strong> Transfer VectorsThurgood Marshall North and East12:45 pm – 1:45 pm Advisory Council Meeting Park Tower 8222Clinical & Regulatory Affairs Committee Meeting Park Tower 8228Ethics Committee MeetingCongressional RoomTranslational Science & Product Development Meeting Park Tower 82198:30 pm – 10:00 pm Molecular <strong>Therapy</strong> Editorial Board Meeting Maryland SuiteSATURDAY, MAY 22nd8:00 am – 8:30 am <strong>Society</strong> Annual Business Meeting Delaware Suite1:15 pm – 2:15 pm Bio-Industry Liaison Committee Meeting Harding Room<strong>Society</strong> Annual Business MeetingThe ASGCT Business Meeting will be conducted on Saturday, May 22, 8:00 am – 8:30 am. A continental breakfast will be served at 7:45 am in the Delaware Suite.All ASGCT members are welcome to attend. Election results will be announced, new <strong>of</strong>ficers will be installed and the <strong>Society</strong>’s finances and membership statistics willbe reviewed. We welcome your input and participation!


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 5Committee RostersEducation CommitteeCo-ChairsLinda B. Couto, PhD (2010 - 2011)Roland W. Herzog, PhD (2008 - 2010)Fulvio Mavilio, PhD (2009 - 2011)MembersLinda B. Couto, PhD (2008 - 2011)William F. Goins, PhD (2007 - 2013)Scott Q. Harper, PhD (2010 - 2013)Karin Jooss, PhD (2007 - 2010)Tal Kafri, PhD (2007 - 2013)Harry L. Malech, MD (2010 - 2013)Robin J. Parks, PhD (2007 - 2010)Suzie H. Pun, PhD (2009 - 2012)Luk Vandenberghe, PhD (2010 - 2013)Embryonic/Somatic Stem <strong>Cell</strong> andTissue Engineering CommitteeChairGay Crooks, MD (2009 - 2011)MembersKaren S. Aboody, MD (2009 - 2012)Fernando D. Camargo, PhD (2010 - 2013)Gay Crooks, MD (2008 - 2011)James Ellis, PhD (2009 - 2012)Jane Lebkowski, PhD (2010 - 2013)Jan A. Nolta, PhD (2009 - 2010)Kazuo Ohashi, MD, PhD (2006 - 2012)Evan Y. Snyder, MD, PhD, FAAP (2006 - 2012)Alice F. Tarantal, PhD (2009 - 2012)Mark E. Westhusin, PhD (2009 - 2010)Ethics CommitteeChairJonathan Kimmelman, PhD (2008 - 2010)Nelson Wivel, MD (2010 - 2011)MembersRoger H. Bertolotti, PhD (2009 - 2012)Theodore Friedmann, MD (2008 - 2011)Edward I. Ginns, MD, PhD (2009 - 2012)Dieter C. Gruenert, PhD (2010 - 2013)Diane Wara, MD (2007 - 2010)Nelson Wivel, MD (2009 - 2012)<strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> <strong>Gene</strong>ticDiseases CommitteeChairValder Arruda, MD, PhD (2010 - 2011)Fabio Candotti, MD (2009 - 2010)MembersValder Arruda, MD, PhD (2007 - 2010)Fatima Bosch, PhD (2010 - 2013)Fabio Candotti, MD (2010 - 2011)Lawrence C. Chan, MD (2010 - 2013)Andrew David<strong>of</strong>f, MD (2010 - 2013)Jim Hu, PhD (2007 - 2010)Stefan Karlsson, MD, PhD (2007 - 2010)Nuria Morral, PhD (2009 - 2012)Amit Nathwani, MD, PhD (2007 - 2010)Mark S. Sands, PhD (2009 - 2010)Thierry C. VandenDriessche, PhD (2009 - 2012)Charles P. Venditti, MD, PhD (2008 - 2011)Robin Ziegler, MS (2010 - 2013)<strong>Gene</strong>tic Vaccines CommitteeChairHelen E. Heslop, MD (2009 - 2011)MembersRonald Alvarez, MD (2006 - 2012)John A. Barrett, MD (2010 - 2013)Si-Yi Chen, MD, PhD (2010 - 2013)K. Reed Clark, PhD (2004 - 2010)Ruxandra Draghia-Akli, MD, PhD (2007 - 2010)Mary Lynne Hedley, PhD (2006 - 2012)Jeffrey J. Molldrem, MD (2010 - 2013)Sattva S. Neelapu, MD (2010 - 2013)David B. Weiner, PhD (2003 - 2010)Hemopoietic <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong>CommitteeChairPunam Malik, MD (2009 - 2010)Michel Sadelain, MD, PhD (2010 - 2011)MembersAlessandro Aiuti, MD, PhD (2008 - 2011)Gianpietro Dotti, MD (2010 - 2013)David W. Emery, PhD (2006 - 2012)Karin Gaensler, MD (2006 - 2012)Manuel Grez, PhD (2009 - 2012)R. Keith Humphries, MD, PhD (2007 - 2010)Harry L. Malech, MD (2009 - 2010)Derek A. Persons, MD, PhD (2007 - 2013)Karen E. Pollok, PhD (2006 - 2012)Barbara Savoldo, MD, PhD (2010 - 2013)Immunology <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>CommitteeChairAdrian J. Thrasher, MD, PhD (2009 - 2011)MembersAndrea Amalfitano, DO, PhD (2010 - 2013)Andrew Byrnes, PhD (2010 - 2013)Youhai H. Chen, MD, PhD (2007 - 2010)Gianpietro Dotti, MD (2009 - 2010)Soosan Ghazizadeh, PhD (2007 - 2010)Roland W. Herzog, PhD (2010 - 2013)Gerald S. Lipshutz, MD (2010 - 2013)John D. Mountz, MD, PhD (2007 - 2013)Daniel A. Muruve, MD (2007 - 2010)Maria-Grazia Roncarolo, MD, PhD (2004 - 2010)Denise Sabatino, PhD (2008 - 2011)Yiping Yang, MD, PhD (2010 - 2013)Infectious Diseases CommitteeChairRoberto Cattaneo, PhD (2009 - 2011)MembersRonald G. Crystal, MD (2009 - 2010)Wayne A. Marasco, MD, PhD (2006 - 2012)Steven Polyak, MD (2010 - 2013)Bruce E. Torbett, PhD, MSPH (2009 - 2012)Jeffrey B. Ulmer, PhD (2006 - 2012)James M. Wilson, MD, PhD (2007 - 2013)Stefan Worgall, MD, PhD (2006 - 2012)


6<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Committee RostersInternational CommitteeChairJohn Rasko, MBBS, PhD (2009 - 2011)MembersAlberto Auricchio, MD (2010 - 2013)Christopher Baum, MD (2010 - 2013)Olivier Danos, PhD (2009 - 2010)Guangping Gao, PhD (2009 - 2012)Bobby Gaspar, MD, PhD (2008 - 2011)Salima Hacein-Bey Abina, PhD (2008 - 2011)Takashi Shimada, MD, PhD (2009 - 2012)Sander Van Deventer, MD, PhD (2007 - 2013)Thierry C. VandenDriessche, PhD (2010 - 2013)Seppo Ylä-Herttuala, MD, PhD, FESC (2005 -2011)Membership CommitteeChairDexi Liu, PhD (2010 - 2011)Savio L.C. Woo, PhD (2008 - 2010)MembersMargaret Black, PhD (2010 - 2013)Michele P. Calos, PhD (2010 - 2013)Larry A. Couture, PhD (2009 - 2010)Dexi Liu, PhD (2006 - 2012)Toya Ohashi, MD (2008 - 2011)Isabelle Rivière, PhD (2004 - 2010)Maria-Grazia Roncarolo, MD, PhD (2005 - 2011)John J. Rossi, PhD (2005 - 2011)Savio L.C. Woo, PhD (2010 - 2011)Ex-OfficioRobert M. Frederickson, PhD (2005 - 2011)Musculo-Skeletal Disorders <strong>Gene</strong> &<strong>Cell</strong> <strong>Therapy</strong> CommitteeChairPaula Clemens, MD (2008 - 2010)Jerry R. Mendell, MD (2010 - 2011)MembersJeffrey Chamberlain, PhD (2010 - 2013)Alan R. Davis, PhD (2007 - 2013)Dongsheng Duan, PhD (2004 - 2010)Dan Gazit, DMD, PhD (2004 - 2010)Richard J. Gregory, PhD (2010 - 2013)Dwight Koeberl, MD, PhD (2010 - 2013)Alan J. Nixon, DVM, MS (2007 - 2013)Shin’ichi Takeda, MD, PhD (2010 - 2013)Bing Wang, MD, PhD (2010 - 2013)Neural Disorders <strong>Gene</strong> & <strong>Cell</strong><strong>Therapy</strong> CommitteeChairMaria G. Castro, PhD (2009 - 2011)MembersRobin R. Ali, PhD (2010 - 2013)William J. Bowers, PhD (2009 - 2012)Xandra O. Breakefield, PhD (2009 - 2012)Mary Bartlett Bunge, PhD (2007 - 2010)Maria G. Castro, PhD (2007 - 2010)Beverly L. Davidson, PhD (2008 - 2011)Nicole Deglon, PhD (2004 - 2010)David J. Fink, MD (2010 - 2013)John Forsayeth, PhD (2004 - 2010)Bill Hauswirth, PhD (2010 - 2013)Jeffrey H. Kordower, PhD (2007 - 2010)Anne Messer, PhD (2010 - 2013)Deborah J. Watson, PhD (2009 - 2012)John H. Wolfe, VMD, PhD (2010 - 2013)Nominating CommitteeChairMalcolm K. Brenner, MD, PhD (2008 - 2010)MembersTerence R. Flotte, MD (2009 - 2010)Luigi M. Naldini, MD, PhD (2009 - 2010)Jan A. Nolta, PhD (2009 - 2010)Samuel C. Wadsworth, PhD (2009 - 2011)Oligonucleotide Based TherapiesCommitteeChairBruce Sullenger, PhD (2009 - 2011)MembersC. Frank Bennett, PhD (2010 - 2013)Brian D. Brown, PhD (2010 - 2013)Dirk Grimm, PhD (2010 - 2013)Dieter C. Gruenert, PhD (2004 - 2010)Mark A. Kay, MD, PhD (2009 - 2010)Anton P. McCaffrey, PhD (2006 - 2012)Paul B. McCray, Jr., MD (2008 - 2011)Kevin V. Morris, PhD (2010 - 2013)Matthew H. Porteus, MD, PhD (2006 - 2010)Bruce Sullenger, PhD (2004 - 2010)Marco Weinberg, PhD (2010 - 2013)Jon A. Wolff, MD (2004 - 2010)Physical <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> andVectorology CommitteeChairShulin Li, PhD (2009 - 2011)MembersMichele P. Calos, PhD (2007 - 2013)Michael Fons, PhD (2009 - 2012)Richard Heller, PhD (2009 - 2012)R. Scott McIvor, PhD (2009 - 2012)Carol H. Miao, PhD (2009 - 2012)Lluis M. Mir, PhD, DSc (2009 - 2012)Matthew H. Porteus, MD, PhD (2010 - 2012)Sean M. Sullivan, PhD (2010 - 2013)EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 7Committee RostersProgram CommitteeCo-ChairsKenneth Cornetta, MD (2010 - 2011)Ronald G. Crystal, MD (2010 - 2011)Stanton L. Gerson, MD (2008 - 2010)John J. Rossi, PhD (2009 - 2011)MembersValder Arruda, MD, PhD (2010 - 2011)Barry J. Byrne, MD, PhD (2010 - 2011)Bruce A. Bunnell, PhD (2009 - 2011)Fabio Candotti, MD (2009 - 2010)Maria G. Castro, PhD (2009 - 2011)Roberto Cattaneo, PhD (2007 - 2011)Seng H. Cheng, PhD (2010 - 2011)Paula Clemens, MD (2008 - 2010)Laurence JN Cooper, MD, PhD (2009 - 2011)Larry A. Couture, PhD (2010 - 2011)Gay Crooks, MD (2009 - 2011)Todd Giorgio, PhD (2009 - 2010)H. Kirk Hammond, MD (2008 - 2010)Helen E. Heslop, MD (2009 - 2011)Carl H. June, MD (2009 - 2010)Jonathan Kimmelman, PhD (2008 - 2010)Donald B. Kohn, MD (2009 - 2010)Shulin Li, PhD (2009 - 2011)Punam Malik, MD (2009 - 2010)Fulvio Mavilio, PhD (2009 - 2010)Jerry R. Mendell (2010 - 2011)Luigi M. Naldini (2010 - 2011)John Rasko, MBBS, PhD (2009 - 2011)Theresa M. Reinke, PhD (2010 - 2011)Michel Sadelain, MD, PhD (2010 - 2011)Daniel R. Salomon, MD (2010 - 2011)Bruce Sullenger, PhD (2009 - 2011)Adrian J. Thrasher, MD, PhD (2008 - 2011)Thomas Tillett (2008 - 2010)Savio L.C. Woo, PhD (2008 - 2010)Publications CommitteeChairDonald B. Kohn, MD (2009 - 2011)MembersHans-Peter Kiem, MD (2007 - 2013)Arthur W. Nienhuis, MD (2010 - 2013)Michel Sadelain, MD, PhD (2007 - 2013)Rachel Salzman, DVM (2008 - 2011)Brian P. Sorrentino, MD (2007 - 2010)Francis C. Szoka, Jr., PhD (2008 - 2011)Respiratory Tract <strong>Gene</strong> & <strong>Cell</strong><strong>Therapy</strong> CommitteeChairBruce A. Bunnell, PhD (2009 - 2011)MembersDavid A. Dean, PhD (2010 - 2013)John F. Engelhardt, PhD (2010 - 2013)Terence Flotte, MD (2010 - 2013)Deborah Gill, PhD (2008 - 2011)Uta Griesenbach, PhD (2007 - 2010)Larry G. Johnson, MD (2009 - 2010)Jay K. Kolls, MD (2007 - 2013)Carolyn Lutzko, PhD (2007 - 2010)David W. Parsons, PhD (2007 - 2013)Tissue Engineering CommitteeChairDaniel R. Salomon, MD (2010 - 2011)MembersDan Gazit, DMD, PhD (2010 - 2013)William Giannobile, DDS, DMedSc (2010 - 2013)Bruce Levine, PhD (2010 - 2013)Paul Y. Liu, MD, FACS (2010 - 2013)Tatiana Segura, PhD (2010 - 2013)Stephanie Simek, PhD (2010 - 2013)Translational Science & ProductDevelopment CommitteeChairLarry A. Couture, PhD (2010 - 2011)MembersJoy Cavagnaro, PhD, DABT, RAC (2010 - 2013)Kenneth Cornetta, MD (2010 - 2013)Derek Hei, PhD (2010 - 2013)Doug J. Jolly, PhD (2010 - 2013)Ian MacLachlan, PhD (2010 - 2013)Alain Rolland, PhD (2010 - 2013)Richard Snyder, PhD (2010 - 2013)Viral <strong>Gene</strong> Transfer VectorsCommitteeChairLuigi M. Naldini, MD, PhD (2010 - 2011)R. Jude Samulski, PhD (2009 - 2010)MembersMavis Agbandje-McKenna (2010 - 2013)Frederic D. Bushman, PhD (2007 - 2010)John A. Chiorini, PhD (2010 - 2013)John T. Gray, PhD (2007 - 2013)Brendan Lee, MD, PhD (2008 - 2011)Luigi M. Naldini, MD, PhD (2008 - 2011)Philip Ng, PhD (2009 - 2012)Samuel D. Rabkin, PhD (2009 - 2012)Stephen J. Russell, MD, PhD (2010 - 2013)Dmitry M. Shayakhmetov, PhD (2009 - 2012)Richard O. Snyder, PhD (2007 - 2010)David Strayer, MD, PhD (2009 - 2012)


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 11Abstract ReviewersCommittee ChairJohn F. Engelhardt, PhDRNA Virus VectorsCoordinating ReviewerArthur W. Nienhuis, MDReviewersChristopher Baum, MDFabio Candotti, MDRobert G. Hawley, PhDDavid W. Russell, MD, PhDAAV VectorsCoordinating ReviewerJohn A. Chiorini, PhDReviewersDouglas M. McCarty, PhDDavid V. Schaffer, PhDMatthew D. Weitzman, PhDXiao Xiao, PhDAdenovirus and Other DNA VirusVectorsCoordinating ReviewerPhilip Ng, PhDReviewersAnja Ehrhardt, PhDJoseph C. Glorioso, III, PhDFlorian Kreppel, PhDRobin J. Parks, PhDDNA Vectorology & <strong>Gene</strong> TargetingCoordinating ReviewerMichele P. Calos, PhDReviewersDeborah Gill, PhDPerry B. Hackett, PhDJakob Giehm Mikkelsen, PhDMatthew H. Porteus, MD, PhDPhysical Methods <strong>of</strong> DeliveryCoordinating ReviewerNancy Smyth Templeton, PhDReviewersMichael A. Barry, PhDJeffrey Hughes, PharmD, PhDChristian Plank, PhDChun Wang, PhDChemical and Molecular ConjugatesCoordinating ReviewerSuzie H. Pun, PhDReviewersHideyoshi Harashima, PhDTheresa M. Reineke, PhDTatiana Segura, PhDMillicent Sullivan, PhD<strong>Gene</strong>tic and MeTABolic Diseases <strong>Gene</strong>& <strong>Cell</strong> <strong>Therapy</strong>Coordinating ReviewerCary Harding, MDReviewersDwight Koeberl, MD, PhDBrendan Lee, MD, PhDJon A. Wolff, MDCardiovascular <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Coordinating ReviewerBarry J. Byrne, MD, PhDReviewersDavid A. Dichek, MDRoger J. Hajjar, MDCathryn S. Mah, PhDChristina A. Pacak, PhDNeurologic & Ophthalmic <strong>Gene</strong> & <strong>Cell</strong><strong>Therapy</strong>Coordinating ReviewerMichael Kaplitt, MD, PhDReviewersKryst<strong>of</strong> Bankiewicz, MD, PhDNicholas Boulis, MDMiguel Sena-Esteves, PhDDeborah J. Watson, PhDMusculo-skeletal <strong>Gene</strong> & <strong>Cell</strong><strong>Therapy</strong>Coordinating ReviewerAlan R. Davis, PhDReviewersJeffrey Chamberlain, PhDPaula Clemens, MDScott Q. Harper, PhDTerri A. Zachos, DVM, PhDInfectious Diseases and VaccinesCoordinating ReviewerJohn A. Zaia, MDReviewersAnn M. Leen, PhDBruce E. Torbett, PhD, MSPHGrant D. Trobridge, PhDDorothee von Laer, MDCancer - ImmunotherapyCoordinating ReviewerHelen E. Heslop, MDReviewersRonald Alvarez, MDJeffrey J. Molldrem, MDJames J. Mulé, PhDDavid B. Weiner, PhDCancer – Oncolytic VirusesCoordinating ReviewerRonald Rodriguez, MD, PhDReviewersWinald Gerritsen, MD, PhDChinghai Kao, PhDDavid H. Kirn, MDMasato Yamamoto, MD, PhDCancer - Targeted <strong>Gene</strong> & <strong>Cell</strong><strong>Therapy</strong>Coordinating ReviewerWilliam S. M. Wold, PhDReviewersKaren S. Aboody, MDFrank Marini, PhDStephen J. Russell, MD, PhDRichard G. Vile, PhDHematologic and Immunologic <strong>Gene</strong>& <strong>Cell</strong> <strong>Therapy</strong>Coordinating ReviewerMichel Sadelain, MD, PhDReviewersGay Crooks, MDDavid W. Emery, PhDDavid Klatzmann, MD, PhDAdrian J. Thrasher, MD, PhD


12<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract ReviewersLung and Respiratory Disease <strong>Gene</strong> &<strong>Cell</strong> <strong>Therapy</strong>Coordinating ReviewerTerence R. Flotte, MDReviewersDongsheng Duan, PhDLarry G. Johnson, MDMaria Limberis, PhDPatrick L. Sinn, PhDImmunologic & Host Responses in<strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Coordinating ReviewerPedro Lowenstein, MD, PhDReviewersRenier J. Brentjens, MD, PhDEvanthia Galanis, MD, DScMaciej Lesniak, MD, FACSLily Wu, MD, PhD<strong>Gene</strong> RegulationCoordinating ReviewerOlivier Danos, PhDReviewersMichael N. Antoniou, PhDGiuliana Ferrari, PhDDirk M. Nettelbeck, PhDPaul D. Robbins, PhDOligonucleotide & RNAi TherapeuticsCoordinating ReviewerPeter Glazer, MD, PhDReviewersAlan Gewirtz, MDPaloma H. Giangrande, PhDDieter C. Gruenert, PhDMark A. Kay, MD, PhDStem <strong>Cell</strong> TherapiesCoordinating ReviewerDonald B. Kohn, MDReviewersEmanuela M. Bruscia, PhDR. Keith Humphries, MD, PhDCarolyn Lutzko, PhDAlice F. Tarantal, PhD<strong>Cell</strong> Processing and VectorProductionCoordinating ReviewerIsabelle Rivière, PhDReviewersAdrian P. Gee, PhDStephen M. Kaminsky, PhDBruce L. Levine, PhDJ. Fraser Wright, PhDSpecial thanks to the abstract reviewers for their time and expertise.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 19<strong>Gene</strong>ral Meeting InformationAMERICAN SOCIETY OF GENE & CELL THERAPYMission and GoalsThe <strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> (ASGCT) is a pr<strong>of</strong>essionalnon-pr<strong>of</strong>it medical and scientific organization dedicated to the understanding,development and application <strong>of</strong> genetic and cellular therapies and the promotion<strong>of</strong> pr<strong>of</strong>essional and public education in the field. ASGCT is the largest medicalpr<strong>of</strong>essional organization representing researchers and scientists dedicated todiscovering new gene and cell therapies. ASGCT was established in 1996, andhas grown to 2,000 members from around the world.The <strong>Society</strong>’s goals are to:• Promote Research, Development and Application <strong>of</strong> <strong>Gene</strong>tic and <strong>Cell</strong>ularTherapies to a Wide Variety <strong>of</strong> Human Diseases• Exchange Information and Promote Education Among Pr<strong>of</strong>essionals andthe Public Through the <strong>Society</strong>’s Annual Meetings, the ASGCT Journal,Molecular <strong>Therapy</strong>, and Interactions with Scientific, Medical, and RegulatoryCommunitiesThe ASGCT 13th Annual Meeting will serve as a comprehensive forum to achievethese goals.Abstract Volume – Molecular <strong>Therapy</strong>All abstracts, except Late Abstract Submissions, accepted for presentation atthe ASGCT 13th Annual Meeting have been published in the May supplement<strong>of</strong> Molecular <strong>Therapy</strong>. Each attendee will receive one copy <strong>of</strong> the supplementalong with their registration materials. Late Abstract Submissions accepted forpresentation have been printed in a booklet that will be distributed to eachattendee.AdmissionOfficial name badges will be required for admission to all ASGCT sessions. AllAnnual Meeting attendees receive a name badge with their registration bag.Name badges should be worn at all times inside the hotel, as badges will beused to control access to sessions and activities. Attendees are cautioned againstwearing their name badges while away from the hotel as badges may drawunwanted attention to your status as visitors to Washington, DC.Business Center (FedEx Office)There is a FedEx Office located in the Marriott Wardman Park Hotel. It is openMonday – Friday from 7:00 am to 7:00 pm and closed on Saturday andSunday.Business MeetingJoin the <strong>Society</strong>’s leadership for a business meeting and continental breakfast.Saturday, May 22, 20108:00 am – 8:30 am (A light continental breakfast will be <strong>of</strong>fered at 7:45 am)Room Delaware SuiteAll members are welcome. See page 3 for more details.Camera/Recording PolicyDue to privacy issues, with the exception <strong>of</strong> a hired pr<strong>of</strong>essional photographer,it is the policy <strong>of</strong> ASGCT that no cameras, camera phones, or video recorders arepermitted in the meeting sessions, exhibit hall, or poster sessions. Please refrainfrom taking any photos in those locations. Audio taping is also prohibited.<strong>Cell</strong> Phones & PDAsFor the courtesy <strong>of</strong> all presenters and attendees, please ensure that cell phoneand PDA ringers are turned <strong>of</strong>f or silenced during all sessions as they mayinterfere with session audio.Committee MeetingsMost standing and scientific committees will meet during the Annual Meeting.See page 3 for Committee Meeting information.Concierge DeskWashington D.C. and the Woodley Park neighborhood <strong>of</strong>fers a wide variety <strong>of</strong>activities and restaurants to meet all tastes and budgets. A Concierge Desk islocated in the Lobby <strong>of</strong> the hotel that can help you with your city needs andreservations.Concierge Desk HoursSunday through Saturday7:00 am – 7:00 pmAdmission by Ticket OnlySome sessions at the Annual Meeting will require an additional ticket to controlaccess and capacity. Tickets for these sessions are not included in the meeting’sregular registration fees. Tickets will be collected at the door by ushers. Thereare still a limited number <strong>of</strong> tickets available for the Meet-the-Investigatorsessions, which may be obtained at the Registration Desk. Meet-the-Investigatortickets are $30.


20<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><strong>Gene</strong>ral Meeting InformationContinuing Medical EducationAccreditation/Credit DesignationThis activity has been planned and implemented in accordance with the EssentialAreas and policies <strong>of</strong> the Accreditation Council for Continuing Medical Education(ACCME) through the joint sponsorship <strong>of</strong> Indiana University School <strong>of</strong> Medicineand the <strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>. Indiana University School <strong>of</strong>Medicine is accredited by the ACCME to provide continuing medical education forphysicians.NOTE: Sessions marked with an asterisk (*) are not part <strong>of</strong> the CME program.Designation StatementIndiana University School <strong>of</strong> Medicine designates this educational activity for amaximum <strong>of</strong> 37.5 AMA PRA Category 1 Credits. Physicians should only claimcredit commensurate with the extent <strong>of</strong> their participation in the activity.CME CertificatesParticipants requiring CME certificates must go to the Registration Desk andcomplete a CME form. CME certificates will be mailed to participants after themeeting. There is a CME fee <strong>of</strong> $50 USD for the 13th Annual Meeting.DisclosureIn accordance with the Accreditation Council for Continuing Medical Education(ACCME) Standards for Commercial Support, educational programs sponsoredby Indiana University School <strong>of</strong> Medicine (IUSM) must demonstrate balance,independence, objectivity, and scientific rigor. All faculty, authors, editors, andplanning committee members participating in an IUSM-sponsored activity arerequired to disclose any relevant financial interest or other relationship with themanufacturer(s) <strong>of</strong> any commercial product(s) and/or provider(s) <strong>of</strong> commercialservices that are discussed in an educational activity.NoteWhile it <strong>of</strong>fers CME credits, this activity is not intended to provide extensivetraining or certification in the field.Educational Methods and MaterialsLectures, Case Presentations, Panel Discussions, Question and Answer Sessions,Audio/Video Presentations, Abstracts, PostersEducational ObjectivesAt the conclusion <strong>of</strong> the activity, the participant should be able to:Provide advice to patients who inquire about the potential <strong>of</strong> gene and celltherapy or the availability <strong>of</strong> open clinical trials, based on their exposure to thecurrent clinical trials in gene and cell therapies.Better instruct their students in medical school and other health venues using thestate-<strong>of</strong>-the-art basic science and clinical trials data presented at the meeting.Use the latest advances in gene and cell therapy to enhance their researchmission, as physician scientists conducting basic and clinical research.Demonstrate improved regulatory compliance in conducting gene and celltherapy clinical trials, through exposure to NIH and FDA faculty during theeducational program.Evaluation MethodEvaluation by questionnaire will address program content, presentation, andpossible bias.NeedsClinical gene transfer has become more and more complex due to ongoingdevelopments in the fields <strong>of</strong> gene and cell therapy itself, together withbioethics, research integrity, and financial conflicts, as well as federalmandates, regulations and guidelines. Oligonucleotide Therapies, Novel VectorDevelopment, Host-Vector Interactions and Vaccine Therapies will be discussedas well as many other scientific topics. This meeting will provide an educationalforum for scientists and clinicians to expand their knowledge about the broaddevelopments in these fields.Target AudienceThe target audience includes basic science and translational researchers, clinicalinvestigators, physicians, postdoctoral fellows, graduate students, employees <strong>of</strong>federal government and regulatory agencies, and other healthcare pr<strong>of</strong>essionalswith an interest in the latest advancements in the field <strong>of</strong> gene and cell therapy.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 21<strong>Gene</strong>ral Meeting InformationExhibitsExhibits will be located in Exhibit Hall C, <strong>of</strong> the Marriott Wardman Park Hotel.The companies, organizations and institutions exhibiting at the ASGCT AnnualMeeting will be providing you with the latest information on products andservices directly related to your pr<strong>of</strong>essional needs, as well as recruiting for openpositions. An Exhibit Hall Social will be held on Wednesday evening and a c<strong>of</strong>feesocial will be <strong>of</strong>fered on Thursday in the exhibit hall. All participants are urged toallow adequate time in your daily schedule to visit the exhibits, as they are anintegral part <strong>of</strong> the success <strong>of</strong> the meeting.Exhibit Hall HoursExhibits will be open Wednesday, May 19 through Friday, May 21, 2010.Wednesday, May 19 7:00 pm – 9:00 pmThursday, May 20 9:15 am – 10:30 amThursday, May 20 6:15 pm – 8:15 pmFriday, May 21 6:30 pm – 8:30 pmGuest AttendanceASGCT asks registered attendees to refrain from taking children, spouses, orguests to any educational session or functions <strong>of</strong>fered at the 13th AnnualMeeting.Internet Access & PolicyInternet access will be available at the Marriott Wardman Park Hotel to allmeeting attendees. Limited computer stations will be provided. When others arewaiting at the internet kiosks, please limit your Internet use to 15 minutes ata time so other attendees can access the internet as well. If you have a laptop,the Marriott Wardman Park <strong>of</strong>fers wireless internet access at certain locationswithin the facility. For guests staying at the Marriott Wardman Park in the ASGCTroom block, free internet access is available in their hotel rooms as well as thehotel’s lobby.Job BankAll openings posted as <strong>of</strong> April 23, 2010 on the ASGCT Job Bank have beenincluded in the Jobs Available Booklet found in the registration bags. Contactinformation is included in the booklet. Job postings for open positions andrésumés may be posted on the Job Posting boards in Exhibit Hall C. You areresponsible for providing multiple copies <strong>of</strong> your posting. A number <strong>of</strong> tables andchairs will also be available in the job exchange center for interview use.Lead RetrievalA lead retrieval system has been made available to all exhibitors <strong>of</strong> the ASGCT13th Annual Meeting. Exhibitors may ask to scan attendee name badges with ahand held scanner in order to obtain attendee contact information.Materials DistributionPlease refrain from distributing promotional materials throughout the meeting;these may only be distributed from an exhibit booth in Exhibit Hall C.MediaMembers <strong>of</strong> the working media may register for the 13th Annual Meeting inthe Press Room – Buchanan Room. Interview space, telephones, and internetaccess are available for the convenience <strong>of</strong> media representatives covering themeeting. Press must register, provide credentials, and wear their press badge foradmittance to ASGCT sessions. Assistance will be provided to members <strong>of</strong> themedia that would like to schedule interviews.No SmokingSmoking is prohibited at all 13th Annual Meeting sessions and events.The Marriott Wardman Park is a non-smoking hotel.


22<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><strong>Gene</strong>ral Meeting InformationPostersAbstract Posters will be on display in Halls B-South and C.Thursday, May 20Abstract Poster Session I10:00 am – 12:00 pm Poster Setup by Authors12:30 pm – 8:15 pm Poster Viewing(authors present from 6:15 pm – 8:15 pm)6:15 pm – 8:15 pm Exhibit Hall Open6:15 pm – 8:15 pm Welcome Reception, Exhibits, and PosterSession8:15 pm – 8:45 pm Authors Remove PostersFriday, May 21Abstract Poster Session II7:00 am – 12:00 pm Poster Setup by Authors12:00 pm – 8:30 pm Poster Viewing(authors present from 6:30 pm – 8:30 pm)6:30 pm – 8:30 pm Exhibit Hall Open6:30 pm – 8:30 pm Networking Reception, Exhibits, and PosterSession8:30 pm – 9:00 pm Authors Remove PostersSaturday, May 22Abstract Poster Session III7:00 am – 12:00 pm Poster Setup by Authors12:00 pm – 8:00 pm Poster Viewing(authors present from 6:00 pm – 8:00 pm)5:00 pm – 6:00 pm Poster Hall Social in Atrium6:00 pm – 8:00 pm Poster Session8:00 pm – 8:30 pm Authors Remove PostersPress RoomThe Press Room is located in the Marriott Wardman Park Hotel, Buchanan Room.Press Room HoursWednesday, May 19Thursday, May 20Friday, May 21Saturday, May 223:00 pm – 6:00 pm8:00 am – 5:00 pm8:00 am – 5:00 pm8:00 am – 5:00 pmRegistration DeskName badges, final programs, and abstract supplements will be distributed atthe registration desk located outside <strong>of</strong> Exhibit Hall C in the atrium, exhibitionlevel <strong>of</strong> the Marriott Wardman Park Hotel.Registration Desk HoursTuesday, May 185:00 pm – 8:00 pmWednesday, May 19 7:00 am – 8:00 pmThursday, May 207:00 am – 5:00 pmFriday, May 217:30 am – 5:00 pmSaturday, May 227:30 am – 5:00 pmShuttle BusASGCT will not be providing shuttle transportation for the 13th Annual Meeting.Speaker Ready RoomThe Speaker Ready Room is located behind the registration desk located outside<strong>of</strong> Exhibit Hall C in the atrium <strong>of</strong> the Marriott Wardman Park Hotel.All faculty must deliver their presentations to the Speaker Ready Room the daybefore their session or at least four hours prior to their presentations. Equipmentis available for faculty to review their materials. Audiovisual personnel will beavailable for assistance. Please mark your materials (your name, session andspeaker order) so the materials can be returned to you. The <strong>Society</strong> stronglyencourages faculty to pre-load presentations in the Speaker Ready Room; thosefaculty who load presentations in the meeting rooms during the sessions willhave that time deducted from their presentation time by the Chair.ASGCT strongly encourages faculty presenting on Wednesday <strong>of</strong> the AnnualMeeting to check-in at the Speaker Ready Room on Tuesday, May 18 to avoidcongestion.Avoid delays and check-in early!Speaker Ready Desk/Room HoursTuesday, May 185:00 pm – 8:00 pmWednesday, May 19 6:30 am – 9:00 pmThursday, May 206:30 am – 7:00 pmFriday, May 217:00 am – 7:00 pmSaturday, May 227:00 am – 3:00 pmSpecialty NeedsIf you require special accommodations in order to fully participate in themeeting, please visit the Registration Desk; ASGCT staff will be happy to assistyou with your specific needs.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 23Hotel Floor PlanseXHIBIT hALL• Exhibit Hall• Internet Kosks• Job Exchange• Poster Hall• Registration• Speaker Ready Room


24<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Hotel Floor PlansLobby level• Plenary Lecture Hall• Session Rooms(Education, Oral Abstract,Scientific)82098210821182128216821782188219822282238224822682288229mezzanine level• Committee Meetings• Press Room – Buchanan• Session RoomsEXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 25Schedule-at-a-Glance (This program is subject to updates/changes)tuesday, may 185:00 pm – 8:00 pm Registration OpenWednesday, may 197:00 am – 8:00 pm Registration Open8:30 am – 12:15 pm ASGCT Board <strong>of</strong> Directors Meeting8:30 am – 11:30 pm Scientific Symposia Session I:<strong>Cell</strong> <strong>Therapy</strong> BioProcessing Symposium and International Symposium: A Global <strong>Gene</strong> <strong>Therapy</strong> Renaissance1:00 pm – 3:00 pm Scientific Symposia Session II3:30 pm – 5:00 pm Education Session I5:30 pm – 7:00 pm Education Session II7:00 pm – 9:00 pm Exhibit Hall Welcome ReceptionThursday, may 207:00 am– 5:00 pm Registration Open8:00 am– 9:15 am George Stamatoyannopoulos Lecture – Keynote: Hal E. Broxmeyer, PhD:Understanding and Enhancing Stem/Progenitor <strong>Cell</strong> Function and Engraftment9:15 am– 10:30 am Exhibit Hall C<strong>of</strong>fee Social10:30 am– 12:30 pm Scientific Symposia Session III12:30 pm– 1:45 pm Meet-the-Investigator Sessions12:30 pm– 8:15 pm Poster Viewing (Authors present from 6:15 pm – 8:15 pm)2:00 pm– 4:00 pm Oral Abstract Session I4:30 pm– 6:15 pm Outstanding New Investigator Symposium6:15 pm– 8:15 pm Exhibit Hall Open & Networking Reception Poster Session I7:00 pm– 10:00 pm Foundation Supported Symposium: Expanding Frontiers in Spinal Muscular Atrophy <strong>Gene</strong> <strong>Therapy</strong>


26<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Schedule-at-a-Glance (This program is subject to updates/changes)friday, may 217:30 am– 5:00 pm Registration Open8:00 am– 10:00 am Plenary Session: Top Oral Abstract Presentations10:30 am– 12:30 pm Scientific Symposia Session IV12:30 pm– 8:30 pm Poster Viewing (Authors present from 6:30 pm – 8:30 pm)2:00 pm– 4:00 pm Oral Abstract Session II4:30 pm– 6:30 pm Presidential Symposium – Keynote Speakers:Katherine A. High, MD: Outstanding Achievement Award LectureJean Bennett, MD, PhD: An Aye for <strong>Gene</strong> <strong>Therapy</strong>: Lessons from a Clinical Trial for Congenital BlindnessDavid A. Williams, MD: New Approaches to <strong>Gene</strong> <strong>Therapy</strong> for Fanconi Anemia6:30 pm– 8:30 pm Exhibit Hall Open & Networking Reception Poster Session II7:00 pm– 10:00 pm Foundation Supported Symposium:Progress and Prospects for <strong>Gene</strong> <strong>Therapy</strong> for CGD and Other Primary ImmunodeficienciesSATURDAY, MAY 227:30 am– 5:00 pm Registration Open8:00 am– 8:30 am ASGCT Business Meeting (with continental breakfast)8:30 am– 10:30 am Scientific Symposia Session V11:00 am– 1:00 pm Oral Abstract Sessions III1:00 pm– 8:00 pm Poster Viewing (Authors present from 6:00 pm – 8:00 pm)1:00 pm– 2:15 pm Meet-the-Investigator Sessions1:00 pm– 2:45 pm ASGCT Board <strong>of</strong> Directors Meeting3:00 pm– 5:00 pm Oral Abstract Sessions IV5:00 pm– 6:00 pm Poster Hall Social in Atrium6:00 pm– 8:00 pm Poster Session III8:00 pm 13th Annual Meeting ConcludesEXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 27Program ScheduleWednesday, May 19Scientific Symposium 1008:30 am - 11:30 amRoom: Maryland Suite<strong>Cell</strong> <strong>Therapy</strong> BioProcessing SymposiumCo-ChairsBoro Dropulic, PhD & Thomas TillettSpeakersDavid L. Urdal, PhDManufacturing <strong>of</strong> Sipuleucel-T for the Treatment <strong>of</strong> Men with Metastatic, Castrate Resistant Prostate CancerSipuleucel-T is an autologous active cellular immunotherapy for men with late stage prostate cancer. It is comprised <strong>of</strong> a patient’s peripheral blood mononuclear cells, includingantigen presenting cells that have been cultured with a recombinant antigen delivery cassette composed <strong>of</strong> prostatic acid phosphatase and granulocyte-macrophagecolony stimulating factor. The development <strong>of</strong> sipuleucel-T will be discussed and the manufacture <strong>of</strong> this novel product candidate will be described.Gary C. du Moulin, PhD, MPHIncorporating the Concepts <strong>of</strong> Modern Quality Systems into <strong>Cell</strong>-based Product DevelopmentWell understood manufacturing processes that identify critical sources <strong>of</strong> variability can lower risk and lead to safe and efficacious treatment modalities that consistentlydeliver the intended therapeutic performance. The evolution <strong>of</strong> pharmaceutical quality systems and its focus on patient safety brings the application <strong>of</strong> a scientific approachand quality risk assessment into practice. This presentation introduces developers to the principles <strong>of</strong> risk management and quality by design, concepts that can assist ininsuring that quality attributes are identified early in the product design phase and incorporated into the control <strong>of</strong> critical processes for cell and gene therapy products.Wednesday, May 19 thJane S. Lebkowski, PhDDevelopment <strong>of</strong> Human Embryonic Stem <strong>Cell</strong>s for Therapeutic ApplicationsHESC-based regenerative cell therapies require 1) evidence for reliable production and quality control <strong>of</strong> product manufacturing, 2) rigorous safety testing in preclinicalmodels, and 3) the design <strong>of</strong> clinical trial protocols that assess the safety and benefit <strong>of</strong> the therapy in appropriate patient populations. GRNOPC1 is a population <strong>of</strong>allogeneic cells containing oligodendrocyte progenitors derived from characterized, dedicated, human embryonic stem cell banks. GRNOPC1 induces myelination <strong>of</strong> axonsin rats with spinal cord injuries and in Shiverer mice, which lack compact myelin, and also produces numerous neurotrophic factors such as midkine, BDNF, and activin.Extensive preclinical studies were performed to determine the distribution <strong>of</strong> GRNOPC1 as well as any potential toxicities after injection near the thoracic injury epicenter.Based on this IND application, the FDA has granted clearance for Geron to initiate a human clinical trial to assess the safety <strong>of</strong> GRNOPC1 in patients with subacute,complete ASIA A, thoracic injuries whose last fully preserved neurological level is T3 to T10.Madhusudan V. Peshwa, PhDUse <strong>of</strong> Flow Electroporation to Load Human <strong>Cell</strong>s to Deliver Clinically Relevant <strong>Gene</strong>s for <strong>Cell</strong> TherapeuticsThe ability to specifically enhance/augment biological function <strong>of</strong> cells using safe, non-gene modifying technologies with ability to deliver the product at clinical/commercialscale using automated, regulatory-compliant, cost-effective processes overcomes the key hurdle in developing “effective” cellular therapies. This presentation providesan overview <strong>of</strong> our experiences in development and characterization <strong>of</strong> cellular products using immune cells and stem cells that have been engineered to deliver clinicallyrelevant potency/biological activity. Specific case studies discussed will include clinical/commercial delivery <strong>of</strong> a (1) a patient-specific cellular vaccine marketed as anOncology therapy in Japan and (2) an engineered endothelial stem cell product for treatment <strong>of</strong> pulmonary arterial hypertension (PAH) in phase I/II trials in Canada.Mitchell Finer, PhDChallenges in the Manufacturing Scale-Up <strong>of</strong> hES <strong>Cell</strong> Derived TherapeuticsHuman Embryonic Stem (hES) <strong>Cell</strong>s have been traditionally derived and passaged as adherent clumps <strong>of</strong> cells on mouse or human fibroblast feeder layers. In order toproduce the quantities <strong>of</strong> cells sufficient for preclinical development and human clinical trials at an acceptable quality standard, methodology must be developed to enablexeno product free/serum free culture <strong>of</strong> hES cells as single cells in the absence <strong>of</strong> feeder lines. Further, while growth on plastic can support limited preclinical and clinicaldevelopment in some indications, achieving the scale <strong>of</strong> hES cell amplification necessary to treat diseases such as Type 1 Diabetes that require significant cell numbers(~100 billion differentiated cells), the ability to expand ES cells in suspension in the absence <strong>of</strong> differentiation, and the ability to synchronously differentiate large numbers<strong>of</strong> cells is required for product development in Type I Diabetes. Data describing the generation <strong>of</strong> feeder-free cGMP derived hES cell banks, expansion <strong>of</strong> large scalehES cell cultures in the undifferentiated state and scaled differentiation in suspension <strong>of</strong> hES-cells into pancreatic b-cell progenitors will be presented.


28<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleWednesday, May 19 thMichelle Williams, PhDDevelopment <strong>of</strong> Prochymal, an Adult MSC <strong>Therapy</strong>Osiris has been developing stem cell therapies to treat unaddressed medical needs since 1992. An important part <strong>of</strong> this process has been navigating the regulatorypathway, particularly through work in the lead indication, graft-versus-host-disease. In 2005, Osiris secured the first ever Fast Track and Orphan Drug designations fromFDA for a stem cell product, followed by Orphan drug designation in Europe as well. In 2008, FDA and Health Canada granted approval for a pediatric expanded accessprogram, providing all children with severe GvHD access to our stem cell drug, Prochymal. The late stage clinical development process is an instructive case study <strong>of</strong>development <strong>of</strong> a novel therapy for a orphan indication.Scientific Symposium 1018:30 am - 11:30 amRoom: Marriott Ballroom Salon 1A Global <strong>Gene</strong> <strong>Therapy</strong> RenaissanceChairJohn Rasko, MBBS, PhDSpeakersDe-Chao Michael Yu, PhDClinical Development <strong>of</strong> KH901, a Conditionally Replicating Oncolytic Adenovirus Encoding Human GM-CSF, in Patients with Recurrent Head andNeck CancersKH901 is a conditionally replicating oncolytic adenovirus, in which the viral E1 genes are under the control <strong>of</strong> a modified telomerase promoter and a cDNA gene <strong>of</strong> granulocytemacrophage colony-stimulating factor (GM-CSF) is inserted in the E3 region. Preclinical studies have demonstrated that KH901 selectively replicates in telomeraseupregulatedand E2F-positive tumor cells and has strong anti-tumor activity. A total <strong>of</strong> 110 patients with recurrent head and neck cancer have been enrolled into clinicaltrials in which KH901 was given intratumorally while chemotherapy was the standard <strong>of</strong> care. KH901 was found to be well tolerated and safe while a significant immuneresponses and pro-longed progression-free survival were observed when compared to the chemotherapy group.Christoph Klein, MD<strong>Gene</strong> <strong>Therapy</strong> for Wiskott-Aldrich SyndromeWiskott-Aldrich syndrome (WAS) is a primary immunodeficiency disorder associated with thrombocytopenia, eczema and autoimmunity. We treated ten WAS patients bytransfusion <strong>of</strong> autologous, genetically modified hematopoietic stem cells (HSC) between 2006 and 2009. We demonstrate sustained WAS protein (WASP) expressionin HSC, lymphoid and myeloid cells, and platelets after gene therapy. T and B lymphocytes, natural killer (NK) cells, and monocytes were functionally corrected. Thepatients´ clinical condition markedly improved with respect to hemorrhagic diathesis, eczema, autoimmunity, and predisposition to severe infections. Genome-wide insertionsite analysis demonstrated vector integration that targeted multiple genes controlling growth development and immunological responses in a persistently polyclonalhematopoiesis.Yasutomo Nasu, MD, PhDProstate Cancer <strong>Gene</strong> <strong>Therapy</strong> in Japan - from HSV-tk to REIC/Dkk-3Intraprostatic therapeutic gene transduction (in situ gene therapy) is one <strong>of</strong> the potent therapeutic option for prostate cancer gene therapy aiming at antimetastatic benefitsthrough the generation <strong>of</strong> immune cell-mediated cytotoxic activities that affect not only the primary tumor but also metastatic lesion. Many clinical trials with HerpesSimplex Virus Thymidine Kinase (HSV-tk) gene therapy have been conducted in many institutes showing clinical safety and efficacy including Japanese trial (Molecular<strong>Therapy</strong> 15:834, 2007). Also immunomodulatory in situ gene therapy using adenoviral Interleukin-12 gene is under way in Japan. As a preclinical study, new therapeuticgene REIC/Dkk3 has been proved to be potential therapeutic gene for prostate cancer as it has induces local apoptosis and systemic immune activation. (Cancer Research65:9617, 2005, Cancer <strong>Gene</strong> <strong>Therapy</strong> 14:765,2007, Int J Oncol 34:657,2009, J Bio Chem 2009 online). In this symposium ongoing development <strong>of</strong> in situ genetherapy for prostate cancer will be reported with regard to systemic immune activation.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 29Program ScheduleChae-Ok Yun, PhDA Phase I Study <strong>of</strong> Relaxin-Expressing Oncolytic AdenovirusOncolytic virotherapy is the use <strong>of</strong> genetically engineered viruses that specifically target and destroy tumor cells via their cytolytic replication cycle. An important issue inreplicating virus-based cancer gene therapy that has received little attention is the limited distribution such viruses have been able to achieve in solid tumors. With the aim<strong>of</strong> improving viral distribution and tumor penetration for cancer gene therapy, we have developed a novel relaxin-expressing oncolytic adenovirus, DWP418. We carried outa phase I study <strong>of</strong> DWP418 in patients with recurrent solid tumors. The preliminary results show that intratumoral administration <strong>of</strong> DWP418 is feasible, well tolerated,and associated with biological activity.Wednesday, May 19 thSeppo Ylä-Herttuala, MD, PhD, FESCAdVEGF-D Local Vascular <strong>Gene</strong> Transfer Program - To Reduce Graft Stenosis in Dialysis PatientsOncolytic virotherapy is the use <strong>of</strong> genetically engineered viruses that specifically target and destroy tumor cells via their cytolytic replication cycle. An important issue inreplicating virus-based cancer gene therapy that has received little attention is the limited distribution such viruses have been able to achieve in solid tumors. With the aim<strong>of</strong> improving viral distribution and tumor penetration for cancer gene therapy, we have developed a novel relaxin-expressing oncolytic adenovirus, DWP418. We carried outa phase I study <strong>of</strong> DWP418 in patients with recurrent solid tumors. The preliminary results show that intratumoral administration <strong>of</strong> DWP418 is feasible, well tolerated,and associated with biological activity.Alessandro Aiuti, MD, PhD<strong>Gene</strong> <strong>Therapy</strong> for Primary Immunodeficiencies<strong>Gene</strong> therapy with hematopoietic stem cells is an attractive therapeutic strategy for several forms <strong>of</strong> primary immunodeficiencies. Results <strong>of</strong> the ADA-SCID and SCID-X1trials have shown long-term restoration <strong>of</strong> immune competence and clinical benefit. The use <strong>of</strong> a reduced-dose conditioning was crucial to allow substantial engraftment <strong>of</strong>multipotent gene-corrected hematopoietic stem cells. Self-inactivating lentiviral vectors have recently entered into the clinic and should provide significant advantages overgammaretroviral vectors.Lunch Break11:30 am – 1:00 pmScientific Symposium 1101:00 pm - 3:00 pmRoom: Thurgood Marshall W<strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong> Translational StudiesChairJ. Kevin Donahue, MDSpeakersJ. Kevin Donahue, MD<strong>Gene</strong> <strong>Therapy</strong> for Atrial FibrillationAtrial fibrillation affects 2-5 million people in the US and millions more around the world. Mechanisms known to sustain the arrhythmia include structural and electrical remodeling.We developed a novel method for atrial gene transfer that paints the vector on the atrial epicardium. Using this method with adenovirus vectors and a transgeneencoding a dominant negative potassium channel mutation, we show elimination <strong>of</strong> atrial fibrillation.Douglas W. Losordo, MDAutologous CD34 + <strong>Cell</strong> <strong>Therapy</strong> for Ischemic Tissue RepairPreclinical studies documented the potential therapeutic potency <strong>of</strong> endothelial progenitor cells, both as cultured and freshly isolated CD34+ cells. Phase I and II clinical trialsusing autologous, GCSF mobilized autologous CD34+ cells have been completed in patients with refractory angina and critical limb ischemia. These studies provide data supportingfeasibility, safety and bioactivity. Importantly, the early evidence suggests the efficacy <strong>of</strong> CD34+ cells and provides important data regarding endpoints and powercalculations for later phase studies. Accordingly, the goal <strong>of</strong> ischemic tissue repair appears feasible and is being approached in human clinical trials.


30<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleWednesday, May 19 thBarry J. Byrne, MD, PhDTherapeutic Strategies for Pompe Disease: Fact or FictionEarly presentation <strong>of</strong> cardiac dysfunction and the diagnosis <strong>of</strong> primary cardiomyopathy in infancy and childhood is most <strong>of</strong>ten associated with a genetic etiology. Oneexample <strong>of</strong> early cardiomyopathy is Pompe disease due to striated muscle glycogen storage. In order to develop a specific treatment based gene transfer, the presentationwill focus on systemic and cardiac gene transfer as an approach to disease management. Additional insight into therapeutic strategies is gained from the ongoing study <strong>of</strong>a cohort <strong>of</strong> patients who have received enzyme replacement therapy. The rational for the first clinical gene transfer trial in Pompe disease will be presented.Patrick Most, MDS100A1: A Novel Molecular Therapeutic Advance for Heart FailureAdvanced understanding <strong>of</strong> the functional networks controlling cardiomyocyte performance together with the evolution <strong>of</strong> safe and efficient gene transfer technologieshave provided the foundation for the development <strong>of</strong> an innovative and novel genetically-targeted HF therapy resulting from the unique molecular pr<strong>of</strong>ile <strong>of</strong> the cardiomyocyteCa2+ sensor protein S100A1. Here we show pr<strong>of</strong>ound long-term therapeutic effectiveness and a favorable safety pr<strong>of</strong>ile for AAV9-S100A1 gene therapy in apreclinical HF model employing cardiac-targeted gene delivery techniques and a gene dosage in pigs that are directly applicable to humans.Scientific Symposium 1111:00 pm - 3:00 pmRoom: Wilson ABCEthical Issues with Embryonic Stem <strong>Cell</strong>s and Induced Pluripotent Stem <strong>Cell</strong>sChairJonathan Kimmelman, PhDSpeakersJonathan D. Moreno, PhDUpdate on Ethical Issues in Stem <strong>Cell</strong> ResearchIn this talk I review the history <strong>of</strong> the embryonic stem cell debate and describe the current policy situation in the US and abroad. I then characterize the significance <strong>of</strong>work on induced pluripotent stem cell (iPSC) technology for the ongoing debate. In brief, with iPSC’s the controversy is likely to shift from the origins <strong>of</strong> the cell lines inhuman embryos to their fate as human regenerative and even reproductive materials.Insoo Hyun, PhDMedical Innovation Outside <strong>of</strong> Clinical Trials: Proceed with CautionRecently the U.S. Food and Drug Administration inaugurated a new policy allowing expanded access to investigational interventions by clinicians attempting to treatpatients outside the research context. For those interested in creating reliable platforms for a whole new generation <strong>of</strong> medical tools through genomics, gene transfer, andstem cell research, the FDA’s new policy holds many hazards. New models for local oversight <strong>of</strong> evidence-based medical innovation will be needed in order to avoid theuncontrolled and premature clinical exploitation <strong>of</strong> these platform technologies and still translate them successfully to the clinic.Jeremy Sugarman, MD, MPH, MAEthical Issues in Translational Research Involving Human Pluripotent Stem <strong>Cell</strong>sThe clinical translation <strong>of</strong> human pluripotent stem cells may take several different paths including clinical translational research, clinical innovation, and the use <strong>of</strong> untestedor unproven interventions. Each <strong>of</strong> these translational pathways raises a unique set <strong>of</strong> ethical issues. Furthermore, there are distinct standards <strong>of</strong> practice and proceduralmechanisms for addressing the ethical issues inherent to each pathway. As such, it is essential that these differences be recognized to best protect not only the well-being<strong>of</strong> patients, but also <strong>of</strong> the scientific enterprise as it seeks to understand the true value <strong>of</strong> stem-cell based interventions.PanelRoger H. Bertolotti, PhDJames Ellis, PhDDeborah A. Hursh, PhDEXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 31Program ScheduleScientific Symposium 1121:00 pm - 3:00 pmRoom: Marriott Ballroom Salon 1Barriers to HSC <strong>Gene</strong> <strong>Therapy</strong>Co-ChairsManuel Grez, PhD & Harry L. Malech, MDSpeakersMary C. Dinauer, MD, PhDNew Approaches for Marrow Conditioning in HSC <strong>Gene</strong> <strong>Therapy</strong>In many genetic blood disorders, there is no selective advantage for hematopoietic stem cells following gene correction. Hence, in addition to expression <strong>of</strong> adequatelevels <strong>of</strong> the therapeutic protein, successful use <strong>of</strong> HSC gene therapy in these disorders will require efficient transduction, homing and engraftment <strong>of</strong> transduced HSC, aswell as cytoreduction <strong>of</strong> uncorrected HSC. It is highly desirable to develop effective conditioning regimens that reduce marrow content <strong>of</strong> uncorrected HSC yet minimizeexposure <strong>of</strong> patients to genotoxic agents and acute and long-term multiorgan toxicities. This talk will present new approaches for marrow conditioning for transplantationin HSC gene therapy, including more selective targeting <strong>of</strong> HSC in their niche.Luigi M. Naldini, MD, PhDExploiting and Antagonizing miRNA in <strong>Gene</strong> <strong>Therapy</strong>C. Anthony Blau, MDPharmacologically Regulated <strong>Cell</strong> <strong>Therapy</strong>Hematopoietic stem cells (HSC) compete for the opportunity to contribute to various mature cell lineages in the blood, however little is known about the rules that governthis competition. Eight years ago, two dogs were transplanted with autologous CD34+ cells that had been transduced with an gammaretrovirus vector encoding greenfluorescent protein (GFP) and a conditionally activatable derivative <strong>of</strong> the thrombopoietin receptor (F36VMpl). Responses to F36VMpl signaling, elicited through administration<strong>of</strong> a chemical inducer <strong>of</strong> dimerization (CID), were reported previously. Now long-term monitoring shows no spontaneous rises in GFP+ cells over time, suggestingthat F36VMpl is inert in the absence <strong>of</strong> CID. Stably higher frequencies <strong>of</strong> GFP+ B and T cells relative to other lineages suggests that CID administration within the first 18months post-transplantation generates stable increases in transduced lymphocytes, whereas only myeloid lineages responded when later courses <strong>of</strong> CID were administeredin one animal. Clonal tracking identified functionally distinct categories <strong>of</strong> hematopoietic stem cells: those that persisted and remained CID responsive, those that persistedbut were CID unresponsive, and those that did not persist.Christopher Baum, MDApproaches to Avoid Adverse Events in the <strong>Gene</strong>tic Modification <strong>of</strong> Hematopoietic <strong>Cell</strong>sAdverse events related to insertional mutagenesis or ectopic transgene expression may compromise the therapeutic prospects <strong>of</strong> gene therapy in the hematopoietic system.Over the past years, major progress has been made in the understanding <strong>of</strong> the underlying mechanisms, opening rational approaches to increase the therapeutic index. Wehave established sensitive nonclinical models, in transplanted C57BL/6J mice or using cultured cells, to reveal the transforming capacity <strong>of</strong> insertional activation <strong>of</strong> cellularproto-oncogenes such as Evi1 or Prdm16, and developed integrating gene vectors designed to reduce the risk <strong>of</strong> insertional transformation by altering the insertion patternand the expression cassette <strong>of</strong> the transgene. In a murine model <strong>of</strong> Mpl deficiency, major adverse events related to ectopic protein expression could be controlled bytranscriptionally targeted vectors. Two important principles emerge to ensure undisturbed long-term hematopoiesis from gene-modified cells. The first is the establishment<strong>of</strong> polyclonal hematopoiesis using vectors with an untargeted integration pr<strong>of</strong>ile and “physiological” transgene cassettes. The second is the generation <strong>of</strong> clonally definedhematopoiesis derived from induced pluripotent cells with genetic modification in bona fide safe harbours.Wednesday, May 19 th


32<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleWednesday, May 19 thScientific Symposium 1131:00 pm - 3:00 pmRoom: Maryland SuiteDelivery <strong>of</strong> Oligonucleotide TherapeuticsChairBruce Sullenger, PhDSpeakersLeaf Huang, PhDDeliver siRNA for Cancer <strong>Therapy</strong>siRNA can not fulfill its promise to become the next generation pharmaceutics unless it is efficiently delivered to the cytoplasm <strong>of</strong> the target cells. Membrane/core typenanoparticles with appropriate surface modification evade the reticuloendothelial system and enable high level accumulation <strong>of</strong> siRNA in the tumor cells. Recent effortfocuses on de-assembly <strong>of</strong> the nanoparticles and cargo release from the endosome. Specific examples <strong>of</strong> siRNA mediated therapy in animal models will be presented.Joseph DeSimone, PhDMicro- and Nano-Fabricated Particles for Therapeutics and VaccinesWe are employing top-down particle fabrication technologies adapted from the microelectronics industry to make highly uniform shape specific particles <strong>of</strong> controlledcomposition for use as therapeutics and vaccines. Strategies will be discussed for the intracellular release <strong>of</strong> various cargos and for selective targeting <strong>of</strong> particles.Steven F. Dowdy, PhDsiRNA Delivery: Tackling the 800 Pound GorillasiRNA induced RNAi responses have great potential to treat human disease, especially cancer and viral infections. However, siRNAs are negatively charged, 14,000 Daltonmacromolecules with no bioavailability to enter cells and siRNA cellular delivery remains a significant rate-limiting step for development <strong>of</strong> RNAi therapeutics. To addressthe siRNA delivery problem, we developed a Peptide Transduction Domain-dsRNA Binding Domain (PTD-DRBD) fusion protein. DRBDs bind siRNAs in a sequence-independentmanner that masks the negative charge, allowing for PTD-mediated siRNA delivery in vivo. PTD-DRBD fusion proteins begin to address the siRNA delivery problem witha high efficiency and low toxicity, biodegradable platform.Joshua Mendell, MD, PhDMicroRNA Replacement TherapiesMicroRNAs (miRNAs) are ~21-23 nucleotide RNAs that regulate the stability and translation <strong>of</strong> target messenger RNAs. We and others have demonstrated that dysregulatedexpression <strong>of</strong> miRNAs is a ubiquitous feature <strong>of</strong> cancer cells and specific miRNAs can act as oncogenes and tumor suppressors. More recently, we showed that dueto their ability to strongly inhibit tumorigenesis, select miRNAs represent potent and non-toxic anti-cancer therapeutic agents when delivered systemically. I will present ourlatest findings regarding the roles <strong>of</strong> miRNAs in cancer and other diseases and the therapeutic potential <strong>of</strong> miRNA delivery.Break3:00 pm – 3:30 pmEDUCATION SESSIONSThe goal <strong>of</strong> the ASGCT Education Program is to provide participants with a broad background and introduction to topics relevant to the field <strong>of</strong> gene and cell therapy.Education Sessions will be subdivided into two headings. “Topical Review” sessions will provide a broad perspective <strong>of</strong> an area <strong>of</strong> importance to the gene and cell therapycommunity such as major vector systems, specific target organs or diseases, and funding/regulatory issues. “Emerging Field Review” sessions will provide a broadperspective <strong>of</strong> a technological or scientific discipline that has recently become <strong>of</strong> great significance to ASGCT members.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 33Program ScheduleEducation Session 1203:30 pm - 5:00 pmRoom: Virginia SuiteEmerging Field Review: Genomic Basis <strong>of</strong> Human DiseaseThis program will be presented by three established investigators at the NIH National Human Genome Research Institute and will highlight active research programs <strong>of</strong>direct relevance to the field <strong>of</strong> gene and cell based therapies. The topics to be covered include: 1) The Genomics Landscape Circa 2010, 2) The ClinSeq Project: PilotingGenome Sequencing for Research in Genomic, and 3) The NIH Undiagnosed Diseases Program.Wednesday, May 19 thChairPaul B. McCray, Jr., MDSpeakersEric D. Green, MD, PhDThe Genomics Landscape Circa 2010The Human Genome Project’s completion <strong>of</strong> the human genome sequence in 2003 was a scientific achievement <strong>of</strong> historic proportions. It also signified a critical transition,as this new foundation <strong>of</strong> genetic information started to be used in powerful ways by researchers and clinicians to tackle increasingly complex problems in biomedicine.Current efforts in genomics research are focused on using genomic data and technologies to acquire a deeper understanding <strong>of</strong> biology and to uncover the genetic basis <strong>of</strong>human disease. Together, these pursuits are moving us down an exciting path towards genomic medicine and fulfilling the promise <strong>of</strong> a sequenced human genome.Leslie G. Biesecker, MDLarge Scale Sequencing and Hypothesis-<strong>Gene</strong>rating Clinical ResearchTo date, high-throughput DNA sequencing has been used to elucidate the complete sequence <strong>of</strong> various species’ genomes. In contrast, its application to clinically directedresearch has been minimal. It is postulated that this technology will be applied to clinical care in the future – such as for pre-symptomatic diagnoses <strong>of</strong> medical conditions,customization <strong>of</strong> preventive strategies, and determination <strong>of</strong> individuals’ pharmacogenetic pr<strong>of</strong>iles. The National Human Genome Research Institute (NHGRI) has a longtermgoal <strong>of</strong> developing the technology and infrastructure for performing large-scale medical sequencing (LSMS) for health research and, eventually, routine clinical care.Thus, at this time pilot studies are needed to start addressing some <strong>of</strong> the issues surrounding the development and implementation <strong>of</strong> LSMS in the clinical research setting.We have launched a pilot project, called ClinSeq, within the NHGRI intramural program. The objective <strong>of</strong> ClinSeq is to pilot approaches to the technical, medical,and genetic counseling issues related to LSMS by generating a large amount <strong>of</strong> sequence data from a set <strong>of</strong> participants. Unlike other projects that maintain anonymizedsamples, ClinSeq is being designed to maintain identified samples to allow us to implement an iterative clinical research strategy that over time will lead to return <strong>of</strong> individualresults to study participants. With this design we hope not only to contribute to the knowledge about susceptibility genes, but also to understand how to integratethis technology into clinical practice.William A. Gahl, MD, PhDThe NIH Undiagnosed Diseases ProgramThe NIH Undiagnosed Diseases Program (UDP) has as its goals to provide answers to patients with mysterious conditions that have long eluded diagnosis and to advancemedical knowledge about rare and common diseases. In the past two years, the UDP has received 2700 inquiries and 1100 medical records. Approximately 240 patientshave been accepted; a diagnosis has been obtained for 10-15% <strong>of</strong> patients seen. The application <strong>of</strong> state-<strong>of</strong>-the-art genetic analysis makes this program a model for theuse <strong>of</strong> genetics in the investigation <strong>of</strong> diagnostic dilemmas. One new disease has been discovered, along with unusual variants <strong>of</strong> known diseases. The Program alsoprovides insights into the current practice <strong>of</strong> medicine in the United States.


34<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleWednesday, May 19 thEducation Session 1213:30 pm - 5:00 pmRoom: Marriott Ballroom Salon 1Emerging Field Review: Regulatory RNAsAbout a decade ago, it became clear that RNA has a widespread, general role in gene regulation. At least part <strong>of</strong> this regulation is mediated through a mechanism termedRNA interference (RNAi). The first talk will discuss the basic principles <strong>of</strong> RNAi and the biology <strong>of</strong> the small RNA species which mediate RNAi. The discovery <strong>of</strong> RNAi hasopened up new avenues in genetic engineering which will be covered by the subsequent talks: Delivery <strong>of</strong> a small interfering RNA (siRNA) or an artificial microRNA(miRNA) with a specific sequence directed against an endogenous mRNA can result in potent knockdown <strong>of</strong> any gene <strong>of</strong> interest. Conversely, endogenous regulatoryRNAs can be exploited as an additional layer <strong>of</strong> regulation to restrict the expression <strong>of</strong> an exogenously introduced transgene to cells <strong>of</strong> a specific tissue origin, lineage ordifferentiation state. Finally, as miRNAs are increasingly being implicated in human disease, technologies that allow their manipulation (knockdown and overexpression)become relevant.Co-ChairsBernhard Rudolf Gentner, MD & Alfred Lewin, PhDSpeakersBrian D. Brown, PhDThe Biology and Application <strong>of</strong> Regulatory RNAsOver the past few years major advances have been made in understanding the action and mechanism <strong>of</strong> regulatory RNAs. These advances have helped to identify some<strong>of</strong> the important factors that influence microRNA and target interaction, which, in turn, is facilitating the improvement <strong>of</strong> technologies that utilize or target the microRNApathway, including RNA interference (RNAi). Here we will discuss some <strong>of</strong> the recent findings in regulatory RNA biology and how these findings are being applied.Ryan L. Boudreau, PhDRNAi-based Strategies for Silencing DiseaseWith a better understanding <strong>of</strong> endogenous miRNA biogenesis and gene silencing processes, scientists have devised strategies to co-opt the cellular RNAi machinery inorder to specifically silence various genes <strong>of</strong> interest. In this way, RNAi serves as a powerful molecular tool used to study gene function in biological processes and providesa novel strategy to treat a variety <strong>of</strong> diseases (e.g. dominant genetic disorders, cancer and viral invasion, among others). The application <strong>of</strong> RNAi as a biological ortherapeutic tool is primarily limited by our ability to introduce inhibitory RNAs into target cells or tissues. Inhibitory RNAs can be designed to mimic primary miRNA stemloops(artificial miRNAs), processed pre-miRNAs (short-hairpin RNAs or shRNAs) or mature miRNAs with perfect complementarity to their targets (small interfering RNAs orsiRNAs). This presentation will overview these RNAi vectors, delivery strategies, and highlight important results regarding RNAi vector safety.Bernhard Rudolf Gentner, MDHow to Study and Exploit microRNAs for <strong>Gene</strong> <strong>Therapy</strong>Chromatin state and the presence <strong>of</strong> a particular set <strong>of</strong> transcription factors binding to the promoter sequence determines the transcription <strong>of</strong> a gene into mRNA. Morerecently, it has become clear that the mRNAoma is extensively subjected to negative regulation by a system involving hundreds <strong>of</strong> small RNA molecules, the so called“microRNAs”. Similar to the transcription factor complement, the “microRNAoma” is specific for tissues, lineages as well as cellular differentiation and activation states,<strong>of</strong>fering an opportunity to exploit this endogenous layer <strong>of</strong> regulation to tune the expression <strong>of</strong> engineered DNA. microRNA regulation has proved a powerful tool toshut down expression in entire tissues like hematopoietic cells, hepatocytes, muscle, and nervous tissue, and thus control transgene immune responses, limit <strong>of</strong>f-targettransgene toxicity and even change the tropism <strong>of</strong> oncolytic viruses. Another emerging application for microRNA regulated expression cassettes is stem cell research andtherapy. microRNAs allow to dissect different stages <strong>of</strong> differentiation, from the bona fide stem cell down to the progeny <strong>of</strong> different lineages. Applications for iPS cells andhematopoietic stem cells will be discussed. A potential side effect <strong>of</strong> exploiting miRNA regulation is the saturation <strong>of</strong> miRNA activity and thus deregulation <strong>of</strong> natural miRNAtargets. Guidelines how to avoid miRNA saturation will be presented.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 35Program ScheduleEducation Session 1223:30 pm - 5:00 pmRoom: Wilson ABCTopical Review: <strong>Cell</strong> <strong>Therapy</strong>ChairEdwin M. Horwitz, MD, PhDSpeakersEdwin M. Horwitz, MD, PhDFundamentals <strong>of</strong> <strong>Cell</strong> <strong>Therapy</strong>Peter Johnston, MD, FACCStem <strong>Cell</strong> <strong>Therapy</strong> for the HeartNearly 6 million people in the United States suffer from heart failure. This burden <strong>of</strong> disease has driven interest in stem cell therapy for the heart in the hope that dysfunctionalmyocytes and vasculature might be repaired or replaced. Clinical trials to date have explored the use <strong>of</strong> various skeletal, bone marrow-derived, endothelial, andendogenous cardiac precursor cells for cardiac repair. Along with much promise, there remain many questions about not only the optimal type <strong>of</strong> cell, but also the timingand delivery method necessary to achieve the maximal therapeutic benefit.Dao Pan, PhD<strong>Cell</strong> and <strong>Gene</strong> <strong>Therapy</strong> for CNS Abnormalities in Lysosomal Storage DisordersLysosomal storage diseases (LSD) are a group <strong>of</strong> ~40 inherited metabolic disorders with a combined incidence <strong>of</strong> 1 in 1500 to 7000 live births. More than 70% <strong>of</strong> allLSD affects the central nervous system (CNS). Bone marrow stem cell transplantation has been shown to ameliorate many visceral symptoms in some LSD patients;however CNS disease associated with LSD remains as a major challenge. Here we will discuss experimental and preclinical studies toward new therapeutic strategies,including stem cell-based brain transplantation therapies, hematopoietic stem cell gene therapies, and CNS-targeted gene therapies. Issues that are pertinent to theirpotential translational applications will also be addressed.Wednesday, May 19 thEducation Session 1233:30 pm - 5:00 pmRoom: Thurgood Marshall WTopical Review: <strong>Gene</strong> <strong>Therapy</strong> for Eye DiseasesRecent successes in viral vector-based gene therapy clinical trials underscore the potential <strong>of</strong> gene therapy as a means to provide definitive solutions for currently incurableocular diseases. This session will focus on the basic biology and vector development for ocular diseases. In addition, it will provide recent updates on novel strategies invector design, as well as the status and prospects <strong>of</strong> gene therapy clinical trials for ocular diseases.Co-ChairsPeter A. Campochiaro, MD & Tal Kafri, PhDSpeakersBill Hauswirth, PhDAAV Vector Considerations for the RetinaConsiderations for AAV vectors intended for transducing cells <strong>of</strong> the retina include 1) the anatomical site <strong>of</strong> vector injection, 2) the vector serotype, 3) the size <strong>of</strong> thevector genome, 4) the strandedness <strong>of</strong> the vector genome and 5) the ability <strong>of</strong> novel AAV vectors to penetrate retinal tissue. Each issue will be discussed in the context <strong>of</strong>gene-based therapies for degenerative retinal diseases.John G. Flannery, PhDEvolution <strong>of</strong> AAV Vectors for Ocular <strong>Gene</strong> <strong>Therapy</strong>


36<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleWednesday, May 19 thPeter A. Campochiaro, MDOverview <strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong> for Ocular DiseasesThe potential <strong>of</strong> ocular gene therapy has been demonstrated in animal models and early clinical trials. Pro<strong>of</strong>-<strong>of</strong>-concept has been provided for gene replacement <strong>of</strong> mutatedgenes in inherited retinal degenerations by 3 clinical trials demonstrating improved visual function by replacement <strong>of</strong> the RPE65 gene in patients with Leber’s congenitalamaurosis. Studies are being considered for X-linked retinoschisis and Stargardt’s disease. A trial in which an antiangiogenic protein was delivered with an adenoviralvector for neovascular age-related macular degeneration (AMD) has shown good safety and some suggestion <strong>of</strong> biological activity. A trial in which a vascular endothelialgrowth factor binding protein is delivered with an AAV vector in patients with neovascular AMD has recently started. Preclinical studies suggest that gene delivery <strong>of</strong> neurotrophicfactors or antioxidant proteins may be considered for retinal degenerations. Thus early clinical trials suggest several promising avenues for ocular gene therapy.Education Session 1243:30 pm - 5:00 pmRoom: Maryland SuiteTopical Review: Stem <strong>Cell</strong> BasicsIn order for stem cells to fulfill their potential as viable cell transplant/gene transfer therapeutics, it will be necessary to understand the process <strong>of</strong> stem cell self-renewalto obtain sufficient cell numbers to be applicable for transplant. Additionally, the genes/factors involved in differentiating these pluripotent cells down specific lineagepathways will also be crucial in the development <strong>of</strong> a transplantable therapeutic. The speakers in this session will address these important issues that not only providefurther insight into organ and tissue development but also open up therapeutic avenues <strong>of</strong> practical application <strong>of</strong> these cells in regenerative medicine.ChairWilliam F. Goins, PhDSpeakersG. Ian Gallicano, PhDStem <strong>Cell</strong>s: Past, Present, FutureThe idea <strong>of</strong> stem cell based therapies is particularly prevalent in the fields <strong>of</strong> diabetes, cardiac and neurological disorders. However, while the field <strong>of</strong> stem cell therapiesmay be relatively new, the road for stem cell research has been long and winding. Understanding hoe the evolution <strong>of</strong> the stem cell field has led to its current state isnot only interesting, it can serve as a powerful tool for predicting its future. Consequently, this presentation will focus on the basic evolution <strong>of</strong> stem cells and their use intherapies.David F. Stroncek, MD<strong>Gene</strong> and MicroRNA Expression in Human Embryonic Stem <strong>Cell</strong>sGlobal gene expression analysis has been used to identify factors responsible for the pluripotency <strong>of</strong> human embryonic stem (hES) cells. Most genes expressed by hES areinvolved with RNA post-transcriptional modification, DNA replication, recombination and repair, RNA Damage repair, cell cycle, and cell growth and proliferation. Transcriptfactors are among the most highly expressed hES genes. Studies focusing on specific genes found that the transcription factors OCT4, NANOG and SOX2 are important inhES development and pluripotency. Transcription factors likely interact with chromatin remodeling factors and histone modifying enzymes to modulate chromatin conformation.The knockout <strong>of</strong> dicer in hES, a nuclease critical in microRNA generation, compromises hES cell proliferation and differentiation and demonstrates an important rolefor microRNA in hES cells. MicroRNA in the miR-302 and miR-520 clusters are expressed in hES cells and may contribute to chromatin structure modifications.Ming Zhan, PhDComputational Biology Studies to Decipher Molecular Mechanisms Controlling Embryonic Stem <strong>Cell</strong> PluripotencyComputational biology has played a significant role in investigation <strong>of</strong> embryonic stem cells (ESCs). In this presentation, I will describe comparative genomics andcomparative transcriptomics studies conducted on ESCs and genetic regulatory network analysis <strong>of</strong> ESCs through mathematical modeling. All the studies contribute to asystems understanding <strong>of</strong> molecular mechanisms controlling ESC self-renewal and differentiation.Break5:00 pm – 5:30 pmEXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 37Program ScheduleEducation Session 1305:30 pm - 7:00 pmRoom: Marriott Ballroom Salon 1Emerging Field Review: Progress Towards Using ES/iPS <strong>Cell</strong>s for Regenerative MedicineThe excitement over embryonic stem cells and induced pluripotent stem cells is that they are a potential source <strong>of</strong> cells for tissues in which tissue specific stem cells haveeither not been identified, not easily isolated, or not easily expanded. That pluripotent stem cells can now be generated from somatic cells, without the destruction <strong>of</strong> anembryo, removes a major ethical barrier to their use. Nonetheless, a continued barrier to the translation <strong>of</strong> pluripotent stem cells to clinical use is to expand and differentiatethem in both a safe and quantitatively sufficient manner that they can be used for tissue specific regenerative medicine applications. This educational session willfocus on the progress that has been made in generating sufficient numbers <strong>of</strong> safe tissue specific cells towards this end.Wednesday, May 19 thChairMatthew H. Porteus, MD, PhDSpeakersRonald D.G. McKay, PhDElias T. Zambidis, MD, PhD<strong>Gene</strong>ration <strong>of</strong> HSC and Red Blood <strong>Cell</strong>s from Normal and Hemoglobinopathic iPSCEvan Y. Snyder, MD, PhD, FAAPCross-Talk and Developmental Programs in Stem <strong>Cell</strong> Biology – A Key to Regenerative MedicineEducation Session 1315:30 pm - 7:00 pmRoom: Maryland SuiteEmerging Field Review: Targeted IntegrationUncontrolled insertion <strong>of</strong> foreign DNA into the human genome may have deleterious consequences, and is a serious safety concern in the clinical application <strong>of</strong> genetherapy. New molecular tools are being developed to target transgene integration to specific sites in the genome and increase the frequency <strong>of</strong> homologous recombination,with the purpose <strong>of</strong> achieving gene correction or replacement in the original genomic environment. These advances include the use <strong>of</strong> Zn-finger nucleases and homingendonucleases, and the delivery <strong>of</strong> homologous recombination cassettes by viral and non-viral vectors. They will be discussed in the context <strong>of</strong> developing better and safergene transfer technology.ChairFulvio Mavilio, PhDSpeakersJ. Keith Joung, MD PhDEngineered Zinc Finger Nucleases for Targeted Genome ModificationEngineered zinc finger nucleases (ZFNs) form the basis <strong>of</strong> a broadly applicable approach for inducing targeted alterations with high efficiency in human and other mammaliancells. This session will provide an introduction to this important technology by: (1) discussing methods for engineering customized ZFNs including OligomerizedPool ENgineering (OPEN) and modular assembly; (2) describing how ZFNs have been used to introduce knockout mutations, insertions and other specific alterations vianon-homologous end-joining and homologous recombination; and (3) considering future experiments and goals for advancing the use <strong>of</strong> ZFNs for therapeutic and researchapplications.Andrew M. Scharenberg, MDHoming Endonucleases as Tools for Targeted IntegrationThe idiosyncratic nature <strong>of</strong> side effects resulting from random integration <strong>of</strong> promoter/gene cassettes in the context <strong>of</strong> viral vector-mediated gene therapy has led toincreasing interest in targeted integration as an alternative approach. A promising methodology for targeted integration involves application <strong>of</strong> rare cleaving nucleases toinduce site specific homologous recombination. Homing endonucleases are an important class <strong>of</strong> rare cutting nucleases whose potential applications in targeted integrationare only now emerging. Homing endonuclease biology, properties, engineering, and applications in targeted integration will be reviewed.


38<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleWednesday, May 19 thDavid W. Russell, MD, PhDAAV-Mediated <strong>Gene</strong> TargetingAAV vectors efficiently recombine with homologous chromosomal sequences in mammalian cells. Up to 1% <strong>of</strong> the entire cell population exposed to vector particlesundergoes gene targeting under optimal conditions. Insertions, deletions and substitutions can all be introduced at precise chromosomal locations with high fidelity. Thetechnique works well in many cell types, including transformed cell lines, stem cells, and even in vivo in murine hepatocytes. Importantly, the gene targeting occurswithout genotoxicity and endonucleases are not required to obtain high targeting frequencies.Education Session 1325:30 pm - 7:00 pmRoom: Thurgood Marshall WTopical Review: <strong>Gene</strong> Delivery to the HeartHeart disease is one <strong>of</strong> the leading causes <strong>of</strong> morbidity and mortality worldwide. While molecular causes are known for many heart diseases, it requires effective heartgene delivery methods to translate these findings into therapy. Because <strong>of</strong> the dynamic nature <strong>of</strong> the heart physiology and the unique anatomic location, cardiac genetransfer has been a great challenge. Significant progress has been made over the last few years. In this session, a historical overview <strong>of</strong> heart gene transfer will be provided.Considering the importance <strong>of</strong> the large animal model in translational medicine, special emphasis will be given to cardiac gene delivery in the canine and porcinemodels.ChairDongsheng Duan, PhDSpeakersDongsheng Duan, PhDOverview <strong>of</strong> Heart <strong>Gene</strong> Transfer in MiceEfficient cardiac gene transfer holds great promise in treating heart diseases, a major health threat worldwide. Over the last decades, tremendous progresses have beenmade in delivering the marker and/or therapeutic genes to the mouse heart. This presentation will provide a historic review <strong>of</strong> the literature and an update <strong>of</strong> the currentdevelopments.Roger J. Hajjar, MD<strong>Gene</strong> <strong>Therapy</strong> for Heart Failure: New Targets and New VectorsDespite the proliferation <strong>of</strong> therapies, congestive heart failure remains a progressive disease where a desperate need for innovative rather than incremental therapies toreverse the course <strong>of</strong> ventricular dysfunction. Recent advances in understanding the molecular basis <strong>of</strong> myocardial dysfunction, together with the evolution <strong>of</strong> increasinglyefficient gene transfer technology; have placed heart failure within reach <strong>of</strong> gene-based therapies. One <strong>of</strong> the key abnormalities in both human and experimental HF isa defect in sarcoplasmic reticulum (SR) function, which is responsible for abnormal intracellular Ca2+ handling. Restoring SERCA2a levels has been shown to improvefunction, metabolism and/or survival in a number <strong>of</strong> experimental models <strong>of</strong> heart failure. Over the last ten years we have undertaken a program <strong>of</strong> targeting importantcalcium cycling proteins in experimental models <strong>of</strong> heart by somatic gene transfer. This has led to the completion <strong>of</strong> a first-in-man phase 1 clinical trial <strong>of</strong> gene therapy forheart failure using adeno-associated vector (AAV) type 1 carrying SERCA2a. In this Phase I trial, there was evidence <strong>of</strong> clinically meaningful improvements in functionalstatus and/or cardiac function which were observed in the majority <strong>of</strong> Neutralizing antibody negative patients at various time points. The safety pr<strong>of</strong>ile <strong>of</strong> AAV genetherapy along with the positive biological signals obtained from this phase 1 trial has led to the recent completion <strong>of</strong> a phase 2 trial <strong>of</strong> AAV1.SERCA2a in NYHA class III/IV patients.Charles R. Bridges, MD, ScDMethods for Cardiac <strong>Gene</strong> DeliveryThis presentation will review the available methods <strong>of</strong> cardiac vector mediated gene delivery including those in use in current clinical trials. Methods considered will includeintracoronary injection, intramuscular injection, pressurized coronary venous retroinfusion, and cardiopulmonary bypass-mediated techniques. The relative strengths andweaknesses <strong>of</strong> these delivery methods will be discussed with reference to efficiency <strong>of</strong> transfection, cardiac versus collateral organ specificity and relative propensity toinduce a T-cell mediated immune response.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 39Program ScheduleEducation Session 1335:30 pm - 7:00 pmRoom: Virginia SuiteTopical Review: Immunotherapy <strong>of</strong> CancerThe immune system can be properly modified to eradicate cancer. Currently, effector molecules, such as monoclonal antibodies and cells such as tumor-reactive lymphocytes,are able to selectively target and eliminate tumors, may be isolated and/or engineered. The potency <strong>of</strong> cellular adoptive immunotherapy has been revealed bypersistent and complete clinical responses obtained with by the transfer <strong>of</strong> allogeneic T lymphocytes in the context <strong>of</strong> allogeneic hemopoietic cell transplantation (allo-HSCT) and by the transfer <strong>of</strong> autologous tumor specific cytotoxic T lymphocytes (CTLs) in cancer patients. Major hurdles limiting adoptive T cell therapy relate to toxicity(ie: graft-versus-host disease-GvHD in allo-HSCT), feasibility and efficacy (ie: difficulty in expanding rare, high-avidity tumor-specific CTLs). <strong>Gene</strong> transfer technologies mayallow to overcome all these hurdles. The transfer <strong>of</strong> tumor-specific TCR or chimeric antigen receptors (CARs), confers tumor-specificity and activity to autologous T cells.The transfer <strong>of</strong> a suicide gene into allogeneic lymphocytes allows to tame toxicity <strong>of</strong> allogeneic lymphocytes. In this session, we will discuss clinical results, challenges andadvances recently made to improve the potency and specificity <strong>of</strong> adoptive immunotherapy with genetically modified lymphocytes to treat cancer.ChairChiara Bonini, MDSpeakersRichard A. Morgan, PhDUsing Engineered T <strong>Cell</strong>s for the Treatment <strong>of</strong> CancerAdoptive cell transfer (ACT) using anti-tumor antigen reactive T cells has proven to be a useful strategy for the treatment <strong>of</strong> metastatic melanoma, with objective responserates <strong>of</strong> up to 72%, with 16% <strong>of</strong> patients rendered disease free. These studies involved the extraction and ex-vivo expansion <strong>of</strong> naturally occurring tumor infiltratinglymphocytes (TIL). Only one-half <strong>of</strong> patients are able to receive this therapy due to lack <strong>of</strong> harvestable tumors, inability to grow TIL, or a lack <strong>of</strong> cellular reactivity. Recentstrategies aimed at broadening ACT involve the use <strong>of</strong> chimeric antigen receptors (CAR) or T-cell receptor (TCR) genes specific for tumor associated antigens and the use <strong>of</strong>viral vectors to transduce these genes into peripheral blood lymphocytes. TCR gene therapy has been applied with some success in recent trials targeting MART-1, gp100and NY-ESO-1. CAR gene therapy trials are also showing promising results in protocols targeting CD19+ lymphomas. Efforts aimed at improving the response rate andminimizing on-target toxicity are in progress.Gianpietro Dotti, MDCar-Based <strong>Gene</strong> Transfer in T LymphocytesExpression <strong>of</strong> a transgenic “T-body” or “chimeric antigen receptor” (CAR) in human T lymphocytes combines the specificity <strong>of</strong> an antibody with the homing and target-celldestruction capabilities <strong>of</strong> T cells. This technology was first described 15 years ago, and after significant improvements in gene transfer technologies and better understanding<strong>of</strong> immunological pathways is now reaching a critical phase <strong>of</strong> clinical evaluation. In this presentation we will summarize the concept <strong>of</strong> CAR-based immunotherapy,describe the steps accomplished, and outline the future progress we need to make if this approach is truly to improve cancer immunotherapy.Chiara Bonini, MDT-<strong>Cell</strong> Based <strong>Gene</strong> <strong>Therapy</strong> in Hematopoietic Stem <strong>Cell</strong> TransplantationThe potency <strong>of</strong> cellular adoptive immunotherapy for the treatment <strong>of</strong> cancer has been clearly revealed by the persistent and complete clinical responses observedin patients undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT) followed by the adoptive transfer <strong>of</strong> donor T lymphocytes. However, severalhurdles remain to be overcome before adoptive immunotherapy can become a broadly applicable therapeutic approach to treat cancer, and gene transfer approachesrepresent ideal solutions to overcome these hurdles. In allo-HSCT, most <strong>of</strong> the antileukemic potential resides in alloreactivity towards patient-specific antigens, such asminor and major histocompatibility antigens, by donor lymphocytes. Unfortunately, alloreactivity is also responsible for the most serious and frequent complication <strong>of</strong>allo-HSCT: the Graft-versus-Host-Disease (GvHD). The risk <strong>of</strong> GvHD increases with the level <strong>of</strong> HLA disparity between host and donor, and leads to impaired quality <strong>of</strong>life and reduced survival expectancy, particularly in patients transplanted from HLA-mismatched donors. <strong>Gene</strong> transfer technologies are promising tools to manipulatedonor T cell immunity to enforce the graft-versus-tumor (GvT) effect, to promote a functional post-transplant immune reconstitution (graft-versus-infection-GvI) and toprevent or control GvHD. To this purpose, several cell and gene transfer approaches, have been investigated at pre-clinical level, and are being implemented in clinicaltrials. The majority <strong>of</strong> these approaches, based on negative or positive selection <strong>of</strong> specific T cell subpopulations, aim at reducing the repertoire <strong>of</strong> donor lymphocytesinfused with or after the graft, so to prevent alloreactivity. In contrast, suicide gene therapy aims at a selective control <strong>of</strong> GvHD in treated patients, thus permittingthe full exploitation <strong>of</strong> alloreactivity against cancer cells. In a phase I-II clinical trial enrolling 54 pts undergoing haploidentical HSCT, we showed that the transfer <strong>of</strong> asuicide gene into donor lymphocytes, allows to provide rapid and effective immune reconstitution, and control <strong>of</strong> GvHD. By taming the toxicity <strong>of</strong> allo-HSCT the suicidegene approach renders haploidentical HSCT a feasible, safe and more effective option for cancer patients <strong>of</strong> all ages. <strong>Cell</strong> and gene therapy approaches currentlyimplemented for a safe exploitation <strong>of</strong> donor immunity against cancer, in the context <strong>of</strong> allo-HSCT will be discussed.Wednesday, May 19 th


40<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleWednesday, May 19 thEducation Session 1345:30 pm - 7:00 pmRoom: Wilson ABCTopical Review: Pre-existing Immunity to <strong>Gene</strong> Transfer VectorsMany viral gene therapy vectors are derived from parent viruses that are present in the human population. Pre-existing immunity to such vectors in form <strong>of</strong> antibodies andmemory B and T cell responses can severely hamper gene transfer in humans. This session will review basic aspects <strong>of</strong> B cell biology, prevalence <strong>of</strong> antibodies to DNAviruses (AAV and adenovirus) in the human population, and CD8+ T cell responses to AAV capsid in humans.ChairRoland W. Herzog, PhDSpeakersMichael Cancro, PhDThe Persistent Problem <strong>of</strong> B <strong>Cell</strong> MemoryPrimary and memory/effector B cell subsets occupy distinct anatomic and homeostatic niches. Despite growing understanding <strong>of</strong> the factors governing pre-immune B celllifespan and homeostasis, the factors regulating antigen-experienced pools – including memory B cells and plasma cells – remain less clear. These likely include members<strong>of</strong> the BLyS family <strong>of</strong> cytokines and receptors, as well as molecular interactions between B lineage cells and survival-promoting stromal elements.Luk H. Vandenberghe, PhDB-cell Immunity to Viral VectorsMany <strong>of</strong> the commonly used vectors for gene therapy application are based on viruses that are endemic in the human population. Therefore, pre-existing immunity mayalter the safety and efficacy <strong>of</strong> gene therapy strategies. In addition, repeated administration <strong>of</strong> the vector may be required for certain therapeutic applications which willalso need to overcome the hurdle <strong>of</strong> pre-existing immunity. Here, we will focus on the breadth and implication for gene transfer <strong>of</strong> the presence and prevalence <strong>of</strong> antibodiesto Adeno and Adeno-Associated viruses in humans and animal models.Katherine A. High, MDHuman T <strong>Cell</strong> Responses to AAV CapsidStudies in healthy donors have shown antigen-specific memory CD8+ T cells to AAV capsid in a substantial portion <strong>of</strong> the human population. We have hypothesized thatthese cells may expand on re-exposure to AAV capsid through administration <strong>of</strong> AAV vectors. Studies in several AAV gene transfer trials have shown that vector administrationis followed by development <strong>of</strong> positive responses on IFN-g ELISPot assays. We have shown that capsid-specific CD8+ T cells can lyse AAV-transduced hepatocytes in anin vitro assay, but the clinical and biological correlates <strong>of</strong> a positive IFN-g ELISPOt response are still under investigation. A clear understanding <strong>of</strong> capsid antigen processingand presentation, and <strong>of</strong> T cell responses to AAV, will be key to optimal application <strong>of</strong> the vector.Exhibit Hall Open & Welcome Reception7:00 pm – 9:00 pmRoom: Exhibit Hall CEXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 41Program ScheduleThursday, May 20George Stamatoyannopoulos Lecture 2008:00 am - 9:15 amRoom: Marriott Ballroom Salons 1, 2 & 3George Stamatoyannopoulos LectureChairBarrie J. Carter, PhDSpeakerThursday, May 20 thHal E. Broxmeyer, PhDUnderstanding and Enhancing Stem/Progenitor <strong>Cell</strong> Function and EngraftmentThere are a number <strong>of</strong> cytokines, cytokine receptors, cell surface proteins, and intracellular molecules that regulate hematopoietic stem (HSC) and progenitorcell (HPC) function. Amongst these are the chemokine, stromal cell derived factor-1 (SDF-1/CXCL12), its receptor, CXCR4, the cell surface protein CD26/Dipeptidylpeptidase IV, which truncates and inactivates specific cytokines, the Rapamycin sensitive m-TOR pathway, and the sirtuin family member, SIRT1. Effortsare underway to modulate these proteins/molecules for enhanced engraftment <strong>of</strong> HSC/HPC and accelerated hematopoiesis.Exhibit Hall C<strong>of</strong>fee Social9:15 am – 10:30 amRoom: Exhibit Hall C


42<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleThursday, May 20 thScientific Symposium 21010:30 am - 12:30 pmRoom: Marriott Ballroom Salons 2 & 3Clinical TrialsChairR. Jude Samulski, PhDSpeakersSteven A. Rosenberg, MD, PhDCancer Immunotherapy Using <strong>Gene</strong>tically Modified T <strong>Cell</strong>sPatients with advanced cancer have been treated by the adoptive transfer <strong>of</strong> autologous T cells transduced with retroviral vectors encoding antitumor T cell receptors.Objective cancer regressions have been seen in patients with metastatic melanoma receiving cells targeting melanoma/melanocyte differentiation antigens and in patientswith synovial cell sarcoma who received cells targeting the NY-ESO-1 cancer-testes antigen. Clinical trials have also begun targeting CD19 on lymphomas and targetingcarcinoembryonic antigen on colorectal cancers.Ian MacLachlan, PhDA Phase I Study to Evaluate the Safety, Tolerability, and Pharmacokinetics <strong>of</strong> Lipid Nanoparticle ApoB siRNA (ApoB-SNALP) in Hypercholesterolemic SubjectsStabilized nucleic acid lipid particles (SNALP) are a modular lipid nanoparticle delivery platform developed for the delivery <strong>of</strong> nucleic acid based drugs. Two SNALP productcandidates, containing siRNA payloads, are currently being evaluated in clinical trials in oncology and metabolic indications. By the end <strong>of</strong> 2010 we expect that as many asfive SNALP based product candidates will be in clinical trials. SNALP containing siRNA that potently silence the conventionally non-druggable target, apolipoprotein B, havebeen evaluated in a Phase 1 clinical trial in hypercholesterolemic subjects. Early results, including the elucidation <strong>of</strong> previously cryptic immune stimulatory activity associatedwith the chemically modified siRNA payload, will be presented.Bill Hauswirth, PhDLeber Congenital Amaurosis (LCA) <strong>Gene</strong> <strong>Therapy</strong> Clinical TrialResults and implications <strong>of</strong> a recently initiated AAV vector clinical trial for LCA2 (recessive RPE65 mutations) will be discussed. LCA2 patients received 150ul (5.9 exp10vector genomes) <strong>of</strong> GMP grade AAV2-CBA-hRPE65 vector subretinally and their visual function then followed periodically over the next 18 months. No adverse eventswere noted for any patient, all demonstrated substantial and significant improvement in light sensitivity, up to 63,000 times better that pretreatment baselines, and somedeveloped a new preferred locus <strong>of</strong> visual fixation centered on the area <strong>of</strong> treated retina.R. Jude Samulski, PhDDesigning AAV Vectors for Translational Studies: Update on DMD Phase 1 TrialInitial AAV vectors for clinical studies have been derived from natural isolates. We have engineered and tested in Phase 1 DMD study chimeric vector derived from type 1and type 2. Outcome <strong>of</strong> these studies and next generation <strong>of</strong> DMD viral vectors suited for efficient gene delivery will be described and pre-clinical data presented.Scientific Symposium 21110:30 am - 12:30 pmRoom: Marriott Ballroom Salon 1Tissue Specific Stem <strong>Cell</strong>sChairCarolyn Lutzko, PhDSpeakersPamela Itkin-Ansari, PhDDevelopment <strong>of</strong> <strong>Cell</strong> Based Therapies for DiabetesThe presentation will address a range <strong>of</strong> issues regarding development <strong>of</strong> pancreas specific stem cell based therapies. First, evidence for stem cells in the adult pancreas andsuggested methods for improved identification <strong>of</strong> such populations will be discussed. Strategies for the recruitment and differentiation <strong>of</strong> tissue specific stem cell populationsin vitro and in vivo will also be addressed. Finally, because insulin producing β-cells are cell autonomous (in contrast to many cell targets, e.g. neurons and cardiomyocytes),they are amenable to novel transplantation and regeneration approaches which will be presented.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 43Program ScheduleMarkus Grompe, MDHepatobiliary Stem/Progenitor <strong>Cell</strong>sCarolyn Lutzko, PhDDeveloping Stem <strong>Cell</strong> Based Therapies for Lung Disease: Bringing <strong>Gene</strong>s to Lung Stem <strong>Cell</strong>s and Stem <strong>Cell</strong>s to the LungThis presentation will focus on different strategies for developing stem cell based therapies for lung diseases. One strategy that will be discussed is the genetic modification<strong>of</strong> endogenous lung progenitor cells during development. This presentation will also discuss progress on the differentiation <strong>of</strong> lung progenitor cells from pluripotent stemcells in vitro.Mervin C. Yoder, MDAre Endothelial Progenitor <strong>Cell</strong>s Derived from the Endothelial Lining <strong>of</strong> Blood Vessels?Circulating endothelial progenitor cells (EPC) have been reported to play important roles in vascular repair and regeneration and are altered in concentration in patientswith arthritis, diabetes, cancer, and cardiovascular disease. No unique marker exists to identify this cell. Indeed, evidence suggests that numerous cell types participate inneoangiogenesis under the term EPC. We will review recent literature that suggests the local endothelial lining to be a major site <strong>of</strong> vascular repair.Scientific Symposium 21210:30 am - 12:30 pmRoom: Virginia Suite<strong>Cell</strong> and <strong>Gene</strong> <strong>Therapy</strong> Approaches for <strong>Gene</strong>tic Diseases: Progress and ChallengesChairCharles P. Venditti, MD, PhDSpeakersNathalie Cartier-Lacave, MD<strong>Gene</strong> <strong>Therapy</strong> for AdrenoleukodystrophyWe report results <strong>of</strong> the first clinical trial using an HIV vector to correct hematopoietic stem cells (HSC) in adrenoleukodystrophy, a severe degenerative disease <strong>of</strong> thecentral nervous system. Three children received autologous corrected HSC after full myeloablative conditionning. Up to 36 months after treatment, stable expression wasdemonstrated in peripheral blood leukocytes and bone marrow CD34+ cells, with polyclonal integration <strong>of</strong> the lentiviral vector. Neurological outcome was comparable tothat observed after allogeneic transplantation. These results demonstrate that lentiviral vectors are promising tools for gene transfer into hematopoietic stem cells in theabsence <strong>of</strong> selective advantage.Alessandra Biffi, MDHSC <strong>Gene</strong> <strong>Therapy</strong> for Metachromatic and Globoid Leukodystrophies: The Next Trials for Inherited Neurological DisordersWe are implementing a gene therapy approach based on the transplantation <strong>of</strong> gene corrected hematopoietic stem cells (HSC) for the treatment <strong>of</strong> severe forms <strong>of</strong>Lysosomal Storage Disorders (LSD) lacking efficacious and safe alternative therapeutic opportunities. To this goal, we exploit the unique features <strong>of</strong> lentiviral vectors (LV),which are prime candidates for HSC gene transfer. Indeed, by using LV for HSC gene correction, we proved the therapeutic potential and safety <strong>of</strong> HSC gene therapy in themurine model <strong>of</strong> metachromatic leukodystrophy (MLD), a severe dysmyelinating LSD. According to these promising results, HSC gene therapy for MLD has now reachedclinical testing. Similarly, an advanced LV design allowed rendering HSC gene therapy a feasible and efficacious approach to be further developed also for globoid cellleukodystrophy, on which we obtained very promising evidences in the disease model.Ronald D.G. McKay, PhDRobert D. Steiner, MDCNS Transplantation <strong>of</strong> Neural Stem <strong>Cell</strong>s in Neuronal Ceroid Lip<strong>of</strong>uscinoses: Phase I Trial ResultsInfantile and late-infantile neuronal ceroid lip<strong>of</strong>uscinosis (INCL and LINCL) are universally fatal neurodegenerative lysosomal storage disorders caused by palmitoyl proteinthioesterase and tripeptidyl peptidase deficiency, respectively. A Phase I trial <strong>of</strong> human central nervous system stem cells (HuCNS-SC®) was conducted in subjects withINCL and LINCL. This study represents the first US FDA authorized use <strong>of</strong> human neural stem cells for clinical testing. The primary goal <strong>of</strong> the trial was to evaluate thesafety <strong>of</strong> direct CNS delivery <strong>of</strong> HuCNS-SC, the immunosuppression regimen, and the surgical technique. Six children underwent HuCNS-SC transplantation;. the studyresults to be presented will include adverse events as well as the neurological, seizure, EEG, radiological, neuropsychological, functional, and quality-<strong>of</strong>-life measuresassessed at frequent intervals post-transplant, as well as evidence <strong>of</strong> engraftment.Thursday, May 20 th


44<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleThursday, May 20 thScientific Symposium 21310:30 am - 12:30 pmRoom: Delaware SuiteHow Viruses Cause Disease: Implications for Vector and Vaccine DevelopmentChairRoberto Cattaneo, PhDSpeakersLynn W. Enquist, PhDResponse <strong>of</strong> PNS Neurons to Herpesvirus InfectionMost alpha herpesviruses are pantropic, neuroinvasive pathogens that establish a reactivateable, latent infection in the peripheral nervous system <strong>of</strong> their natural hosts.Various manifestations <strong>of</strong> herpes disease rely on extent and direction <strong>of</strong> the spread <strong>of</strong> infection between the surface epithelia and the nervous system components thatinnervate that surface. One aspect <strong>of</strong> such controlled spread <strong>of</strong> infection is the capacity for synaptically defined, transneuronal spread, a property that makes alphaherpesviruses useful tools for determining the connectivity <strong>of</strong> neural circuits and gene delivery. Spread <strong>of</strong> infection among neurons involves long distance travel <strong>of</strong> virioncomponent in axons. I will discuss our recent work on the response <strong>of</strong> cultured PNS neurons to infection.Jeffrey Bergelson, MD<strong>Cell</strong> Biology <strong>of</strong> Virus EntryViruses need cells, and they have evolved to manipulate cellular processes at many stages <strong>of</strong> their life cycle. In a number <strong>of</strong> cases, virus-induced signals are needed toprepare the cell for virus entry and infection. I will discuss several examples <strong>of</strong> viruses— including adenoviruses and picornaviruses— that use receptor-mediated signalsto initiate the entry process.Bernard Moss, MD, PhDPoxvirus Replication and Pathogenesis: Implications for Vector and Vaccine DevelopmentThe ability <strong>of</strong> vaccinia virus to infect most cells provides an important advantage for their extensive use as an expression vector and as an oncolytic agent. We have shownthat an unprecedented number <strong>of</strong> viral proteins are required for the membrane fusion step, which can occur either at the plasma membrane or in endosomes. These 12proteins are associated in a complex and conserved in all members <strong>of</strong> the poxvirus family.Roberto Cattaneo, PhDThe Force Behind Targeting: How the Receptor-Triggered Membrane Fusion Mechanism <strong>of</strong> Measles Virus WorksMeasles is one <strong>of</strong> the most infectious viruses, and its portable entry system has become the paradigm for targeting oncolysis and therapeutic gene delivery. While thissystem is strictly regulated, the mechanism triggering fusion <strong>of</strong> the viral envelope with the plasma membrane was unknown. We show that re-alignment <strong>of</strong> the subunits <strong>of</strong>the viral attachment protein (H) is the trigger, and that force exerted at an appropriate location on the H-dimer unlocks the trigger at the plasma membrane, just as lowpH or proteases trigger endosomal membrane fusion in other viral entry systems.Scientific Symposium 21410:30 am - 12:30 pmRoom: Maryland Suite<strong>Gene</strong> and <strong>Cell</strong> Therapeutic Approaches for Musculo-Skeletal DiseasesCo-ChairsPaula Clemens, MD & Alan J. Nixon, DVM, MSSpeakersEdward M. Schwarz, PhDRevitalizing Structural AllograftsTo the end <strong>of</strong> a tissue-engineering solution for large segmental defects in bone caused by trauma or tumors, we proposed rAAV-coated allografts, which introduce angiogenic,osteogenic and osteoclastogenic factors into the healing environment to stimulate vascular ingrowth, bone formation and remodeling <strong>of</strong> the necrotic tissue. Here Iwill describe the molecular biology <strong>of</strong> this technology, its methodology and application in mice, and the translational outcome measures that have been developed to proveefficacy in large animals and humans in which torsional biomechanical strength is the primary outcome measure.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 45Program ScheduleSteven C. Ghivizzani, PhD<strong>Gene</strong> <strong>Therapy</strong> for AchondroplasiaAchondroplasia is the most common form <strong>of</strong> short-limbed dwarfism and is caused by a specific mutation in the transmembrane portion <strong>of</strong> fibroblast growth factor receptor3 (FGFR3), a negative regulator <strong>of</strong> growth and maturation in growth plate chondrocytes. This “gain <strong>of</strong> function” mutation causes enhanced signaling from FGFR3, leadingto reduced growth in bones that grow by endochondral ossification. Using gene delivery, we have been exploring the utility <strong>of</strong> systemic delivery <strong>of</strong> a secreted FGFR3variant as a means to titrate circulating FGFR3 ligands and, thereby, reduce aberrant FGFR3 signaling to rescue bone growth. Our results thus far in explant culture and ina mouse model <strong>of</strong> achondroplasia have been highly encouraging, and indicate that a related post-natal treatment approach may be viable in humans.Joel R. Chamberlain, PhDSystemic Delivery <strong>of</strong> RNAi Therapeutics for Treatment <strong>of</strong> Myotonic DystrophyDominant genetic diseases pose a particular challenge for therapy since the disease-causing cell product must be eliminated. We have chosen an RNA interference-basedapproach to target toxic mRNA that causes myotonic dystrophy. We show that disease pathology in the transgenic HSALR mouse model <strong>of</strong> DM1 is mitigated by systemicapplication <strong>of</strong> AAV6 carrying an HSA-targeted RNAi expression cassette. Aspects <strong>of</strong> systemic delivery and endogenous RNAi pathway effects will be presented in the context<strong>of</strong> therapeutic development. These studies highlight the potential <strong>of</strong> RNAi as a therapy for dominant muscle disease and for other diseases where body-wide therapeuticRNAi distribution is required.Carmen Bertoni, PhDEnhanced <strong>Cell</strong>-Mediated Regenerative Approaches to DMD Using RNA InterferenceMany <strong>of</strong> the diseases affecting muscle are distinguished by the impaired ability <strong>of</strong> muscle progenitor cells to fuse and form new fibers and the inability <strong>of</strong> the existingfibers to maintain normal homeostasis. Among those, Duchenne muscular dystrophy (DMD) is the most severe and is caused by lack <strong>of</strong> dystrophin expression. The loss <strong>of</strong>muscle mass and strength typically seen in DMD patients is thought to be attributed to the progressive atrophy and loss <strong>of</strong> individual muscle fibers, which is concomitantto the accumulation <strong>of</strong> fibrotic and fat tissues in skeletal muscles. The mdx mouse has been instrumental in determining the potential beneficial effects <strong>of</strong> some <strong>of</strong> thetherapeutic approaches currently under development for DMD. We have used High Throughput Screen (HTS) to identify modulators <strong>of</strong> muscle progenitor cells fusionin vitro and screened several siRNA libraries in order to identify genes that play active role during terminal differentiation. Secondary and tertiary screens were used toconfirm the effects <strong>of</strong> RNAi on wild type or mdx muscle cultures and to select for candidate hits that significantly enhance myotube size.Transplantation <strong>of</strong> wild type muscleprogenitor cells undergoing silencing <strong>of</strong> the genes identified by HTS enhanced the ability <strong>of</strong> those cells to repair skeletal muscles <strong>of</strong> mdx/nude mice and resulted in asignificant increase in dystrophin expression. Direct intramuscular injection <strong>of</strong> a lentiviral vector expressing shRNAs <strong>of</strong> those genes was also shown to dramatically improveregeneration <strong>of</strong> injected muscles. These studies advance cell-mediated regenerative approaches to DMD and may enable us to identify treatments capable <strong>of</strong> slowing downprogressive muscle wasting characteristic <strong>of</strong> many neuromuscular disorders.Thursday, May 20 thScientific Symposium 21510:30 am - 12:30 pmRoom: Wilson ABCNovel <strong>Cell</strong> and <strong>Gene</strong> <strong>Therapy</strong> Interventions for the LungChairBruce A. Bunnell, PhDSpeakersDaniel Weiss, MD, PhDRecent Advances in <strong>Gene</strong> and <strong>Cell</strong> Therapies for Lung Diseases<strong>Gene</strong> and cell therapy approaches for lung diseases continue to rapidly develop. Progress has been made in vector development and several notable advancements havebeen made over the past year with use <strong>of</strong> gene transfer approaches for lung cancer. In parallel, cell therapy approaches for lung diseases have broadened and includestudies <strong>of</strong> ex vivo lung regeneration using state <strong>of</strong> the art bioengineering approaches as well as increasing appreciation <strong>of</strong> immunomodulatory effects <strong>of</strong> adult mesenchymalstem cells in the lung. Most notably, progress has been made in clinical trials <strong>of</strong> cell therapies for both pulmonary hypertension and for chronic obstructive pulmonarydisease. The introductory comments and subsequent presentations in this session will highlight these advances.


46<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleThursday, May 20 thPaul B. McCray, Jr., MDLentiviral Vector Development for Prevention and Treatment <strong>of</strong> Cystic Fibrosis Lung DiseaseThis talk will provide an overview <strong>of</strong> recent developments in lentiviral vector technology and their applications for lung disease treatment. Additional data will be presentedregarding animal models <strong>of</strong> CF lung disease and their potential utility in pre-clinical studies <strong>of</strong> efficiency and efficacy <strong>of</strong> gene transfer.Blair Roszell, MSTwo Step Differentiation <strong>of</strong> Mouse Embryonic Stem <strong>Cell</strong>s to Definitive Endoderm and Lung Epithelial <strong>Cell</strong>s<strong>Cell</strong>-based therapy is a novel and promising treatment option for lung disease, but deriving large quantities <strong>of</strong> lung epithelial cells from embryonic stem (ES) cells isstill inefficient. In my talk, I will compare growth factor-based and small molecule-based methods for deriving definitive endoderm. After an additional lung inductionstep, activin/wnt and IDE2 treated cells express a similar pr<strong>of</strong>ile <strong>of</strong> lung-specific markers. Furthermore, in fetal pulmonary cell co-cultures, activin/wnt treated cells canintegrate into alveolar forming units; whereas IDE2 treated cells express SP-C, but do not integrate into lung-like structures. Finally, ES-derived lung cells were endotracheallyinjected into preterm mice with evidence <strong>of</strong> AEII distribution within the lung parenchyma. These studies concludes that a recapitulation <strong>of</strong> development may enhancederivation <strong>of</strong> an enriched population <strong>of</strong> lung-like cells for use in cell based-therapy.Peter I. Lelkes, PhDBiomedical Engineering in the LungThe goal <strong>of</strong> this invited lecture is to present an overview <strong>of</strong> current tissue engineering and regenerative medicine -based approaches to repair dysfunctional lungs, withfocus on neonatal and adult pulmonary diseases. Specifically, we will present our developmental biology-based studies to create vascularized alveolar structures in vitroand in vivo. In addition, we will discuss attempts by us and others to optimize the directed differentiation <strong>of</strong> embryonic stem cells towards pulmonary epithelial and otherlung-specific cell types for distal lung tissue engineering and regeneration.John F. Engelhardt, PhDNew Developments in Animal Models <strong>of</strong> Cystic FibrosisCystic fibrosis (CF) is a recessive disease that affects multiple organs and is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.Animal modeling <strong>of</strong> this disease has been challenging, with species- and strain-specific differences in organ biology and CFTR function influencing the emergence <strong>of</strong> diseasepathology. CF mice have been an invaluable system for dissecting the biology <strong>of</strong> CFTR function in certain organs, however, they generally lack pancreatic and spontaneouslung disease seen in humans with CF. Recently, CF pig and ferret models have been generated. Comparative analysis <strong>of</strong> these CF models may help to understand how thepleiotropic functions <strong>of</strong> CFTR in multiple organs influence disease progression.Special NIH Program Session 21610:30 am to 12:30 pmRoom: Thurgood Marshall WestNIH Resources for <strong>Cell</strong> and <strong>Gene</strong> <strong>Therapy</strong> InvestigatorsModeratorCheryl L. McDonald, MDSpeakersDaniel Rosenblum, MDPThe National <strong>Gene</strong> Vector Biorepository and Coordinating Center (NGVBCC)The NGVBCC provides an array <strong>of</strong> support services for NIH-supported investigators engaged in research on <strong>Gene</strong> <strong>Therapy</strong>. The services include archiving <strong>of</strong> biologicalmaterials, insertional site analysis, pharmacology and toxicology resources, and a reagent repository. It has an open database and educational resource. The archiving andanalytical services are limited to investigators at academic and other not for pr<strong>of</strong>it institutions. We will review the scope <strong>of</strong> the services, methods for accessing them, ways<strong>of</strong> meeting eligibility for their use, website link information, specimen submission and electronic access, and other relevant information pertinent to use <strong>of</strong> the facility.How the Clinical and Translational Science Awards (CTSA) Program can Support <strong>Cell</strong> and <strong>Gene</strong> <strong>Therapy</strong> InvestigatorsThe CTSA program replaced the GCRC (<strong>Gene</strong>ral Clinical Research Center Program) with a much broader support for novel product and team science within and betweenacademic institutions and other related research support such as the NIH and industrial sponsors. The aim is to streamline project development, review, and implementation<strong>of</strong> translational and clinical research by providing support from a variety <strong>of</strong> services such as biostatistics, research design, ethics, clinical research management,informatics, and regulatory knowledge (including FDA). It also facilitates the creation <strong>of</strong> networks between academic centers and the recruitment <strong>of</strong> trial participants fromthe community. The presentation will review pertinent summary information about the CTSA program including web access to program information, access to services,potential support from CTSA-related key functions for development <strong>of</strong> clinical trials, and a brief description <strong>of</strong> concierge service (a team <strong>of</strong> experts who can provide supportin the creation and approval <strong>of</strong> a clinical trial).EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 47Program ScheduleCatherine McKeon, PhDPThe NIDDK Molecular <strong>Therapy</strong> Centers and <strong>Cell</strong> <strong>Therapy</strong> ProgramThis presentation will cover the NIDDK Molecular <strong>Therapy</strong> Centers and the <strong>Cell</strong> <strong>Therapy</strong> Program, as well as some other programs that <strong>of</strong>fer resources to investigators.David G. Badman, PhDThe NIH Rapid Access to Interventional Development Program (NIH-RAID)The NIH Roadmap has established the NIH-RAID Program (Rapid Access to Interventional Development), to make available, on a competitive basis, critical resourcesneeded for development <strong>of</strong> new small molecule or natural product-derived therapeutic agents. This program, part <strong>of</strong> the Translational Research component <strong>of</strong> Reengineeringthe Clinical Research Enterprise, uses resources <strong>of</strong> the National Cancer Institute’s (NCI) Developmental Therapeutics Program and the National Heart Lung and BloodInstitute’s (NHLBI) <strong>Gene</strong> <strong>Therapy</strong> Resource Program. Services provided depend upon the project and strength <strong>of</strong> the preliminary data. Services potentially available include:bulk supply, GMP manufacturing, formulation, assay development suitable for pharmacokinetic testing, and animal toxicology. Assistance also can be provided in the regulatoryprocess. Currently, animal efficacy studies are not supported; neither are syntheses nor formulation <strong>of</strong> recombinant proteins or monoclonal antibodies. However, theNIH-RAID Pilot will consider requests for services to support later-stage preclinical development <strong>of</strong> these entities. Manufacture <strong>of</strong> certain gene vectors (non-GMP viral andnon-viral gene vectors and GMP-grade adeno-associated virus and lentivirus vectors) can be requested, and preclinical studies <strong>of</strong> vectors may be supported. Additionally, theNIH-RAID Pilot will now consider requests for manufacture <strong>of</strong> small molecule or natural product material for any clinical study. Proposals must originate from academic ornon-pr<strong>of</strong>it investigators, as well as companies eligible for Small Business Innovation Research Program (SBIR). Collaboration with larger industry partners is encouraged.Roy S. Wu, PhDThe NCI Experimental Therapeutics Program (NExT) and the Chemical Biology Consortium (CBC)The NCI has recently consolidated their drug discovery and development efforts into a single pipeline for increased timeliness and efficiency and with an emphasis onunmet clinical needs. Researchers in academia, government or industry, either nationally or internationally, may apply. NCI is committed to moving high-priority discoveryand development projects through to pro<strong>of</strong>-<strong>of</strong>-concept clinical trials in a partnership relationship. NExT is not a grant or contract mechanism <strong>of</strong> support. Rather, projectswill be evaluated and ranked by a peer committee followed by internal committees that will assess clinical need, develop project budgets and milestones with go/no godecisions, and provide access to the NCI’s drug discovery and preclinical and clinical development resources at no cost to the applicant. The resources include activities suchas production <strong>of</strong> pharmaceutical-grade material, development <strong>of</strong> clinical formulations, and first-in-human studies. The discovery arm <strong>of</strong> NExT, known as the NCI ChemicalBiology Consortium or CBC, is a collaborative network <strong>of</strong> 12 Centers that provide access to high-throughput screens and chemistry to optimize compounds for human use.Applications for potential NExT projects are accepted online four times a year. Visit http://next.cancer.gov/ for application instructions and more information.Danilo A. Tagle, PhDThe NINDS Translational Research ProgramThe NINDS Translational Research Program is specifically focused on pre-clinical therapy development necessary to begin clinical testing <strong>of</strong> therapeutic candidates forneurological disorders. This program supports the pre-clinical development <strong>of</strong> drugs, biologics, and devices including activities that lead to Investigational New Drug (IND)or Investigational Device Exemptions (IDE) applications to the Food and Drug Administration (FDA). Supported projects can include lead optimization, pre-clinical efficacytesting, predictive ADMET (absorption, distribution, metabolism, excretion, and toxicology) testing, formulation, manufacture, pharmacology, toxicology, and IND or IDEsubmission. For entry to the program, projects must have one or more identified therapeutic leads and convincing pro<strong>of</strong>-<strong>of</strong>-principle on efficacy demonstrated in crediblemodels <strong>of</strong> the disease or against defined disease targets.Thursday, May 20 thLisbeth Welniak, PhDProduction Assistance for <strong>Cell</strong>ular Therapies (PACT)The PACT Group is an NHLBI-funded initiative that is committed to the advancement <strong>of</strong> effective cell therapies. This program was renewed in January, 2010 for a secondfive-year period <strong>of</strong> funding. PACT supports the development <strong>of</strong> novel somatic cell therapy products by providing production assistance to the cell therapy community, aswell as educational training via web seminars and at meetings. Production assistance is provided through the funded contract manufacturing <strong>of</strong> therapeutic cell products atPACT processing facilities. These facilities <strong>of</strong>fer scale-up capability and expertise in the translational development needed to produce cell therapy products under cGMP andcGTP regulations. PACT’s educational training focuses on three general areas: translational development/scale-up and manufacture <strong>of</strong> cell therapy products; and qualityassurance and regulatory issues. The objective <strong>of</strong> the PACT program is to continue to advance cellular therapy research in the areas <strong>of</strong> regeneration <strong>of</strong> damaged/diseasedtissues, organs, and biologic systems, and targeted treatments for serious diseases without effective therapies. The cell processing facilities will support translationalresearch in novel cell therapies to advance the field <strong>of</strong> cellular therapy and to enable scientifically meritorious basic science research to reach the clinical trial stage. Pleasevisit the PACT website at www.pactgroup.net for more information regarding the PACT program.


48<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleThursday, May 20 thSonia Skarlatos, PhDThe Center for Fetal Monkey <strong>Gene</strong> Transfer for Heart, Lung, and Blood DiseasesAnother unique resource that NHLBI <strong>of</strong>fers to gene therapy researchers is the Center for Fetal Monkey <strong>Gene</strong> Transfer for Heart, Lung, and Blood Diseases at the University<strong>of</strong> California, Davis (http://www.cfmgt.ucdavis.edu/). Established in 2001, this program provides NHLBI-funded investigators with essential expertise, services, andresources to evaluate the safety and efficacy <strong>of</strong> gene transfer strategies as they emerge.The NHLBI <strong>Gene</strong> <strong>Therapy</strong> Resource Program (GTRP)The NHLBI <strong>Gene</strong> <strong>Therapy</strong> Resource Program (GTRP) was initiated in March 2007 and since that time it has become a very active and valuable program. The program<strong>of</strong>fers several services at no cost to investigators researching gene therapy for heart, lung, and blood disorders. The GTRP includes four core labs to assist researchers withpreclinical vector production, pharmacology and toxicology testing, and production <strong>of</strong> clinical-grade AAV and lentiviral vectors. Additionally, there is a Clinical CoordinatingCenter to assist both investigators and the NHLBI in all aspects <strong>of</strong> the program.The Science Moving Towards Research Translation and <strong>Therapy</strong> (SMARTT) ProgramThe purpose <strong>of</strong> the SMARTT program is to assist translation to the clinic <strong>of</strong> novel synthetic, natural, or biologic therapeutic interventions arising in the scientific communityfor the treatment <strong>of</strong> heart, lung, and blood diseases. SMARTT will provide resources in the areas <strong>of</strong> preclinical and clinical-grade production and testing for biologics, nonbiologicsand small molecules in accordance with Good Manufacturing Practices (GMP), and in the conduct <strong>of</strong> pharmacology/toxicology studies. In addition, SMARTT willprovide regulatory assistance to investigators wishing to conduct early stage clinical trials.Ying Huang, PhDCommunication with FDA/CBER/OCTGT (Office <strong>of</strong> <strong>Cell</strong>ular, Tissue and <strong>Gene</strong> Therapies) Open Microphone Question and Answer PeriodThis talk will introduce the audience to the pathways that are currently available in CBER/OCTGT to facilitate communication between CBER and the sponsor <strong>of</strong> an investigationalcellular or gene therapy product to enable the conduct <strong>of</strong> safe clinical trials. This talk will briefly touch upon the various disciplines involved in the developmentprogram <strong>of</strong> these novel products, with a focus towards supporting translation from bench to bedside.Open Microphone Question and Answer PeriodLunch Break12:30 pm – 2:00 pmMeet-the-Investigator Lunch 22012:30 pm - 1:45 pmRoom: HardingMusculo-SkeletalSpeakersJeffrey Chamberlain, PhD & Paula Clemens, MDMeet-the-Investigator Lunch 22112:30 pm - 1:45 pmRoom: HooverCancer Stem <strong>Cell</strong>sSpeakersTimothy P. Cripe, MD, PhD & Samuel D. Rabkin, PhDEXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 49Program ScheduleOral Abstract Session 2302:00 pm - 4:00 pmRoom: Marriott Ballroom Salons 2 & 3AAV Vectors: Vector TraffickingCo-ChairsDavid V. Schaffer, PhD & Xiao Xiao, PhD(Abstracts 1-8; see page 86)Oral Abstract Session 2312:00 pm - 4:00 pmRoom: Maryland SuiteDNA Vectorology & <strong>Gene</strong> TargetingCo-ChairsPerry B. Hackett, PhD & Matthew H. Porteus, MD, PhD(Abstracts 9-16; see page 86)Oral Abstract Session 2322:00 pm - 4:00 pmRoom: Wilson ABCNeurologic & Ophthalmic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Co-ChairsMichael Kaplitt, MD, PhD & Deborah J. Watson, PhD(Abstracts 17-24; see page 87)Thursday, May 20 thOral Abstract Session 2332:00 pm - 4:00 pmRoom: Thurgood Marshall WCancer – Immunotherapy T <strong>Cell</strong> StudiesCo-ChairsChiara Bonini, MD & Dean A. Lee, MD, PhD(Abstracts 25-32; see pages 87-88)Oral Abstract Session 2342:00 pm - 4:00 pmRoom: Marriott Ballroom Salon 1Cancer-Oncolytic Viruses ICo-ChairsWinald Gerritsen, MD PhD & David H. Kirn, MD(Abstracts 33-40; see page 88)


50<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleOral Abstract Session 2352:00 pm - 4:00 pmRoom: Virginia SuiteImmunologic & Host Responses in <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Co-ChairsEvanthia Galanis, MD, DSc & Maciej S. Lesniak, MD, FACS(Abstracts 41-48; see pages 88-89)Thursday, May 20 thOral Abstract Session 2362:00 pm - 4:00 pmRoom: Delaware SuiteOligonucleotide & RNAi Therapeutics ICo-ChairsDieter C. Gruenert, PhD & Faye A. Rogers, PhD(Abstracts 49-56; see page 89)Outstanding New Investigator Symposium 2404:30 pm - 6:15 pmRoom: Marriott Ballroom Salons 1, 2 & 3Outstanding New Investigator SymposiumChairJohn J. Rossi, PhDSpeakersAlessandra Biffi, MDRendering Hematopoietic Stem <strong>Cell</strong> Transplantation Efficacious for the Treatment <strong>of</strong> Lysosomal DisordersTransplantation <strong>of</strong> hematopoietic stem cells (HSC) from healthy donors has been employed in Lysosomal Storage Disorders (LSD) for delivering a functional hydrolaseto the affected tissues. However, likely due to the slow kinetics <strong>of</strong> infiltration <strong>of</strong> the nervous system, the efficacy <strong>of</strong> the transplant is poor in LSD with severe neurologicmanifestations. The limited efficacy <strong>of</strong> the allogeneic procedure may be implemented by: i) understanding the mechanisms <strong>of</strong> myeloid infiltration into affectedtissues, and in particular into the brain, in order to favour and accelerate this phenomenon; ii) rendering the transplanted HSC and or their progeny more effectivethan normal donors’ cells in enzyme delivery by means <strong>of</strong> gene transfer to enhance the metabolic correction <strong>of</strong> the affected tissue. We have addressed these issuesattempting to develop an effective and safe therapeutic approach for different LSD with CNS involvement, for one <strong>of</strong> which we recently started clinical testing.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 51Program ScheduleKevin V. Morris, PhDUtilizing the Endogenous Long Non-Coding RNA Pathway in Human <strong>Cell</strong>s to Transcriptionally Modulate <strong>Gene</strong> ExpressionObservations over the last few years have demonstrated that non-coding RNAs can transcriptionally modulate gene expression in human cells. Recent data hasinferred long non-coding RNAs as endogenous effector molecules regulating gene transcription in human cells. Importantly, this non-coding RNA based mechanism canbe taken advantage <strong>of</strong> to either transcriptionally silence gene expression in a specific long-term manner or activate gene transcription by the targeted degradation <strong>of</strong>gene specific regulatory long antisense non-coding RNAs.Thursday, May 20 thBakhos A. Tannous, PhDEx-Vivo Monitoring <strong>of</strong> In Vivo <strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong>Secreted reporters are useful tools for the detection and non-invasive monitoring <strong>of</strong> different biological processes in experimental models. We have characterized thenaturally secreted Gaussia luciferase (Gluc) as a highly sensitive reporter for quantitative assessment <strong>of</strong> cells in vivo by measuring its activity in few microliters <strong>of</strong>blood. The Gluc blood level is linear with respect to cell number and therefore is a sensitive tool for monitoring tumor growth and response to therapy, gene transfer,viral infection and replication, transcription factor activation, as well as circulating cells survival and proliferation. We also applied the Gluc assay to monitor the effect<strong>of</strong> novel gene therapy based on the constitutively active mutant human brain sodium channel as well as tumor-specific nuclear factor kappa B inducible system drivingthe expression <strong>of</strong> a suicidal gene. The Gluc blood assay complements in vivo bioluminescence imaging which has the ability to localize the signal and provides amultifaceted assessment <strong>of</strong> biological processes in experimental disease and therapy models.Charles P. Venditti, MD, PhD<strong>Gene</strong> <strong>Therapy</strong> for Methylmalonic Acidemia (MMA)The hereditary methylmalonic acidemias are inborn errors <strong>of</strong> metabolism and are excellent candidates for gene and cell therapy. We have generated new insights frommouse models and patient investigations to instruct successful pre-clinical experiments. To facilitate the implementation <strong>of</strong> clinical gene therapy for MMA, we havedeveloped and validated a sensitive isotope ratio mass spectrometry technique to non-invasively monitor whole-body metabolic effects after gene delivery.


52<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleExhibit Hall Open & Networking Reception6:15 pm – 8:15 pmRoom: Exhibit Hall CThursday, May 20 thPoster Session I6:15 pm – 8:15 pmRoom: Exhibit Hall B South, CAAV Vectors I(Abstracts 57 through 72; see pages 89-90)Adenovirus and Other DNA Virus Vectors I(Abstracts 73 through 86; see pages 90-91)Targeting <strong>Gene</strong> Modification and Integration(Abstracts 87 through 99; see pages 91-92)Physical Methods <strong>of</strong> Delivery(Abstracts 100 through 116; see page 92)Chemical and Molecular Conjugates I(Abstracts 117 through 132; see page 93)<strong>Gene</strong>tic and Metabolic Diseases <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I(Abstracts 133 through 147; see pages 93-94)Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I(Abstracts 148 through 160; see pages 94-95)Infectious Diseases and Vaccines: Vaccine Related <strong>Gene</strong> Transfer Research(Abstracts 161 through 174; see pages 95-96)Cancer – Immunotherapy I(Abstracts 175 through 190; see pages 96-97)Cancer-Oncolytic Viruses I(Abstracts 192 through 204; Abstract 191 moved to Oral Session 413; see pages 97-98)Cancer-Targeted <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I(Abstracts 205 through 219; see pages 98-99)Hematologic and Immunologic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I(Abstracts 220 through 233 and Abstract 869 which has been moved from Poster Session III; see page 99)<strong>Gene</strong> Regulation(Abstracts 234 through 244; see page 100)Oligonucleotide & RNAi Therapeutics I(Abstracts 245 through 259; see pages 100-101)Stem <strong>Cell</strong> Biology(Abstracts 260 through 273; see pages 101-102)<strong>Cell</strong> Processing and Vector Production I(Abstracts 274 through 282; see pages 102)Late Breaking Science I(Abstracts 922 through 932; Abstract 926 withdrawn; see pages 102-103)EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 53Program ScheduleFoundation Symposium 2507:00 pm - 10:00 pmRoom: Thurgood Marshall WestExpanding Frontiers in Spinal Muscular Atrophy <strong>Gene</strong> <strong>Therapy</strong>ChairChristian L. Lorson, PhDSpeakersChristian L. Lorson, PhDIntroduction: Perspectives on SMA TherapeuticsC. Frank Bennett, PhDRNA Targeting Therapeutics for the Treatment <strong>of</strong> Motor Neuron DiseasesMarco Passini, PhD<strong>Gene</strong> and Antisense Therapies in SMA MiceBrian K. Kaspar, PhDRescue <strong>of</strong> a Spinal Muscular Atrophy Mouse Model by Early Postnatal Delivery <strong>of</strong> SMN with scAAV9 and Clinical Development PlansPetra Kaufman, MDSMA Clinical Trials – NINDS PerspectiveThursday, May 20 thFoundation Symposium 250 is supported by the following ASGCT partners:


54<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleFriday, May 21Friday, May 21 stPlenary Abstract Session 3008:00 am - 10:00 amRoom: Marriott Ballroom Salons 1, 2 & 3Top Oral Abstracts Plenary SessionCo-ChairsStanton L. Gerson, MD & John J. Rossi, PhDSpeakersMervin C. Yoder, MD IntroducesEirini P. Papapetrou, MD, PhDTherapeutic Transgene Expression in Patient-Specific iPS <strong>Cell</strong>s after Selection <strong>of</strong> “Safe Harbors” for Lentiviral Vector IntegrationJames Ellis, PhD IntroducesAxel Schambach, MD PhDA Novel Lentiviral Expression System Reveals Patchwork Colony Formation during Reprogramming to Induced Pluripotent Stem <strong>Cell</strong>sDieter C. Gruenert, PhD IntroducesErica B. Schleifman, BSTargeted Genomic Modification and Inactivation <strong>of</strong> the CCR5 <strong>Gene</strong> Using PNA-Induced Homologous RecombinationMatthew H. Porteus, MD, PhD IntroducesRichard Gabriel, Dipl.-Biol.Lentiviral Microarrays for High-Throughput and Real-Time Monitoring <strong>of</strong> <strong>Gene</strong> Expression DynamicsBruce Sullenger, PhD IntroducesJiehua Zhou, PhDSystemic Administration <strong>of</strong> Sendrimer- and Aptamer-siRNA Conjugates Efficiently Suppresses HIV-1 InfectionChiara Bonini, MD IntroducesAntonio Di Stasi, MDCASPALLO: Phase I Clinical Trial <strong>of</strong> Allodepleted T <strong>Cell</strong>s Transduced with Inducible Caspase 9 Suicide <strong>Gene</strong> after Haploidentical Stem <strong>Cell</strong> TransplantationBreak10:00 am – 10:30 amScientific Symposium 31010:30 am - 12:30 pmRoom: Virginia SuitePersonalized <strong>Cell</strong> and <strong>Gene</strong> <strong>Therapy</strong>ChairJohn J. Nemunaitis, MDSpeakersJohn J. Nemunaitis, MDSuccess <strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong> - A Personalized and Personal ApproachClinical use <strong>of</strong> molecular science is coming <strong>of</strong> age! Numerous targeted therapies, many related to gene based technology, in particular involving RNA interference platforms,are undergoing clinical track development. Mechanisms for cancer specific plasmid delivery are unfolding in the clinical arena. Furthermore, gene based vaccinesare showing improved promise. These dynamics combined with more efficient and economic quantitative proteogenomic technology development will enable a personalizedapproach to cancer therapy. Results <strong>of</strong> personalized gene and cell based therapies will be discussed.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 55Program ScheduleLilia Bi, PhDRecovery and Reduction Costs for <strong>Cell</strong>-based <strong>Gene</strong> <strong>Therapy</strong> ProductsThis presentation will discuss the recovery and reduction costs for cell-based gene therapy products from following aspects:What is the purpose <strong>of</strong> cost recovery and where does it apply?Cost recovery regulations and rulesWhat can be charged for under cost recovery?Process for requesting cost recovery and what information sponsors need to providePotential cost reduction by following the step-wise approach to implementing cGMP’s and product characterizationCarl H. June, MD<strong>Gene</strong>tically Engineered T <strong>Cell</strong>s: Can We Afford Not To Do This?A key concern with adoptive cell transfer therapy is a financial issue related to the potential costs <strong>of</strong> a lymphocyte infusions. Factors that relate to the eventual pricing <strong>of</strong>a personalized therapy consisting <strong>of</strong> autologous T cells are numerous and include issues such as the magnitude <strong>of</strong> efficacy, durability <strong>of</strong> responses, safety and tolerability.In this regard, it is instructive to consider allogeneic stem cell transplantation procedures that have potent efficacy and considerable toxicity, and whether adoptive T celltransfer therapy could supplant and extend this procedure.Jeffrey Schlom, PhDRecombinant Cancer Vaccines as Monotherapy and in Combination <strong>Therapy</strong>Recombinant poxviral vaccines have been developed that contain the transgenes for one or more tumor associated antigens and three T-cell costimulation molecules(TRICOM). Preclinical studies have shown that these vaccines have the ability to break tolerance and induce therapeutic activity in several models. Randomized clinicaltrials in patients with prostate cancer have shown statistical improvements in survival. Preclinical studies have shown that radiation and or chemotherapy can alter the phenotype<strong>of</strong> tumor cells and render them more susceptible to T-cell lysis; combination therapy studies with vaccine are ongoing. Recently results have shown that moleculesinvolved in the Epithelial to Mesenchymal Transition (EMT) metastatic process can be targeted by vaccine therapy.Scientific Symposium 31110:30 am - 12:30 pmRoom: Marriott Ballroom Salon 1Stem <strong>Cell</strong>s and MalignancyChairEvan Y. Snyder, MD, PhD, FAAPSpeakersInder M. Verma, PhDCancer Stem <strong>Cell</strong>s: Lessons from GlioblastomasWe have generated novel mouse models <strong>of</strong> human gliomas using lentiviral vectors containing oncogenes or shRNA to suppressor genes. The resulting tumors had stemcell properties. We will discuss the implications <strong>of</strong> these results.Roger J. Packer, MDChildhood Brain Tumors: Opportunities for Biologic-Based TherapiesBrain tumors are the most common solid malignancy <strong>of</strong> childhood and are the leading cancer related cause <strong>of</strong> morbidity and mortality in the pediatric-aged patients.Progress has been made in the management <strong>of</strong> childhood brain tumors, but lack a clear understanding <strong>of</strong> the molecular pathogenesis <strong>of</strong> these tumors and ways to exploitthis molecular pathogenesis for treatment has slowed progress. Over the past decade, there have been tremendous advances in the understanding <strong>of</strong> the molecularunderpinnings <strong>of</strong> a variety <strong>of</strong> different childhood brain tumors including the most common malignant brain tumor, the medulloblastoma, and the most common benigntumor, the low grade-glioma. Molecular targets have been identified and a host <strong>of</strong> biologic agents including antiangiogenesis drugs, tyrosine kinase growth inhibitors, andagents interfering with intracellular signaling are in clinical trials. To date, their use has not been associated with improvements in survival but there is great enthusiasm incontinuing their study with the hope <strong>of</strong> developing more effective and safer therapies.Friday, May 21 st


56<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleEvan Y. Snyder, MD, PhD, FAAP“Lineage Mapping” The Development <strong>of</strong> a Brain TumorCarl Bart Rountree, MDThe Role <strong>of</strong> Stem <strong>Cell</strong>s and Cancer Stem <strong>Cell</strong>s in Liver CancerHepatocellular carcinoma (HCC) ranks as the third most frequent cause <strong>of</strong> cancer-related mortality in the world; and over the last four decades, traditional cytotoxicchemotherapy has failed to improve patient survival in HCC. Typically, patients develop HCC after many years <strong>of</strong> chronic liver injury, during which time, there is a significantexpansion <strong>of</strong> the liver stem cell pool. Increasing evidence indicates that liver cancer stem cells (CSC) are the origin <strong>of</strong> a subset <strong>of</strong> HCC, and patients with a CSC phenotypeHCC have a significantly worse prognosis. Additionally, using stem cell surface markers such as CD133, purified liver CSC populations have been identified within humanHCC cell lines and mouse models that mimic human chronic liver injury and HCC development. These liver CSCs are resistant to traditional chemotherapy agents andligand-receptor mediated apoptosis, form tumors at very limited dilution, and represent a critical therapeutic target in future HCC treatments.Friday, May 21 stScientific Symposium 31210:30 am - 12:30 pmRoom: Thurgood Marshall WestCurrent Status <strong>of</strong> <strong>Gene</strong>tic VaccinesChairStephen Gottschalk, MDSpeakersSi-Yi Chen, MD, PhDDC-based Tumor Vaccine by Combining SOCS1 Silencing and TLR SignalingMichael A. Barry, PhD<strong>Gene</strong>-based Vaccines Against Mucosal PathogensThe vast majority <strong>of</strong> pathogens enter the body at mucosal surface. Yet, most vaccine strategies are delivered into the systemic immune system where induced immunologicalmemory may not efficiently “cross-over” to mucosal surfaces to provide barrier protection against these pathogens. To maximize protection, we will discuss comparisons<strong>of</strong> replication-competent and replication-defective first generation and helper-dependent adenoviral vaccines for their utility in systemic and mucosal infections by SHIV,influenza, and methicillin-resistant Staphylococcus Aureus (MRSA). We will also discuss “stealth” strategies during vaccination to evade pre-existing and vector-inducedimmunity against adenovirus including serotype-switching and PEGylation.Sattva S. Neelapu, MDTargeting Chemokine Receptors with Fusion DNA VaccinesTherapeutic vaccination with patient-specific tumor-derived idiotype protein conjugated to a carrier molecule, keyhole limpet hemocyanin and administered with granulocyte-monocytecolony-stimulating factor as an adjuvant was shown to induce tumor-specific immune responses and molecular remissions in follicular lymphoma patientsin phase II clinical trials. Results from a recently completed randomized controlled double-blind phase III trial suggested that administration <strong>of</strong> idiotype protein vaccine inthe setting <strong>of</strong> minimal residual disease improves disease-free survival in patients with follicular lymphoma as compared with control. However, a major limitation <strong>of</strong> thisstrategy is the requirement to generate a custom-made protein vaccine for each patient, by a process that is expensive, laborious, and time-consuming. DNA vaccines maybe produced more rapidly and provide an alternative to protein vaccines. Strategies to enhance the efficacy <strong>of</strong> DNA vaccines by fusing tumor antigens to chemokines andusing novel adjuvants will be discussed.Stephen Gottschalk, MDAdenoviral Vaccines for Enhancing T-cell Therapies for CancerAlthough the benefits <strong>of</strong> T-cell therapy for cancer can be increased by prior lymphodpeletion <strong>of</strong> the host, this process has usually required chemotherapy or radiation.Vaccination with tumor associated antigens to which the transferred T cells respond should be a less toxic means <strong>of</strong> promoting antitumor activity, but to date this hasproved ineffective, favoring instead the transition to a non-cytotoxic immune response. We have developed a new adenoviral based vaccine that contains antigen, atoll-like receptor 5 activator and an inhibitor <strong>of</strong> the antigen presenting attenuator A20. Administration <strong>of</strong> this vaccine prior to T-cell transfer induces a strong Th1-polarizingenvironment leading to potent antitumor effects even in the absence <strong>of</strong> lymphodepletion and even when vaccination or T-cell transfer alone are ineffectual. Such Th1-polarizing vaccines may be <strong>of</strong> value when enhancement and maintenance <strong>of</strong> a Th1 response is desirable.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 57Program ScheduleScientific Symposium 31310:30 am - 12:30 pmRoom: Maryland SuitePrecise <strong>Gene</strong>tic and Post-transcriptional Modification <strong>of</strong> Stem <strong>Cell</strong>sChairDieter C. Gruenert, PhDSpeakersPeter M. Glazer, MD, PhDTargeted <strong>Gene</strong> Modification in Hematopoietic Stem <strong>Cell</strong>s via Triple Helix FormationTriple helix-forming peptide nucleic acids (PNAs) can bind to polypurine regions in DNA in a sequence-specific manner. The resulting triplexes constitute an altered DNAstructure that can stimulate DNA repair and recombination in a site-specific manner in human cells. We have found that transfection <strong>of</strong> human hematopoietic stem cellswith triplex-forming PNAs plus short single-stranded donor DNAs can produce targeted modification <strong>of</strong> disease related genes. Site-directed modification <strong>of</strong> two key humangenes has been achieved: (1) the human beta-globin gene, mutations in which cause thalassemia and sickle cell anemia; and (2) the CCR5 gene, which encodes aco-receptor for HIV-1 entry into human cells. Triplex-modified hematopoietic stem cells have been transplanted into immune deficient mice, with full engraftment, normalpatterns <strong>of</strong> differentiation, and persistence <strong>of</strong> the targeted gene modification for up to four months.Linzhao Cheng, PhD<strong>Gene</strong> Targeting in Human Pluripotent Stem <strong>Cell</strong>sHuman induced pluripotent stem (iPS) cells reprogrammed from adult somatic cells resemble embryonic stem (ES) cells that can proliferate indefinitely in culture whilemaintain their full differentiation potential (pluripotency), therefore providing an unlimited resource for disease research and therapy. These self-renewable stem cells alsomake it feasible to achieve gene targeting (to correct or create DNA mutation) in the genome <strong>of</strong> normal human cells by homologous recombination (HR) that is naturalbut inefficient process for genome repair. To enhance HR-mediated gene targeting efficiency we also incorporate the strategy utilizing zinc finger nuclease (ZFN), whichis designed as a fusion protein with multiple zinc-finger DNA binding and the FokI endonuclease domains. Upon dimer formation, two ZFNs (co-delivered with a donorDNA template) can create a sequence-specific DNA double strand break to stimulate HR near the cut site. We found that ZFNs enhanced HR by >100-1,000 fold in eithercreating or correcting a mutation in human iPS and ES cells.Dieter C. Gruenert, PhDModification <strong>of</strong> the CF and Sickle <strong>Cell</strong> Anemia iPS cells by SFHRInduced pluripotent stem (iPS) cells have the potential <strong>of</strong> significantly advancing the development <strong>of</strong> stem cell-based therapies, especially as they pertain to inheriteddiseases. The potential to generate autologous pluripotent stem cells to repair and regenerate tissues and organs damaged by disease pathologies is a cornerstone <strong>of</strong>patient-specific therapy. Cystic fibrosis (CF), the most common inherited disease in the Caucasian population, and sickle cell anemia (SCA), the most prevalent hemoglobinopathycommonly found in individuals <strong>of</strong> African, Middle Eastern, Mediterranean, and Southeast Asian decent, have significant multiorgan damage that will benefit fromcellular repair. In this context, somatic cells from a CF patient with a ∆F508/∆F508 genotype and a SCA patient have been reprogrammed, characterized, and correctedby small fragment homologous replacement (SFHR).Robert Blelloch, MD, PhDMicroRNA Regulation <strong>of</strong> Embryonic Stem <strong>Cell</strong> Self-Renewal and DifferentiationFriday, May 21 st


58<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleFriday, May 21 stScientific Symposium 31410:30 am - 12:30 pmRoom: Delaware SuiteNew Physical <strong>Gene</strong> Delivery System and VectorologyChairShulin Li, PhDSpeakersChristian Plank, PhDNanomagnetic Nucleic Acid DeliveryPhysical force has been used with great success to enable, enhance or localize nucleic acid delivery. In magnet<strong>of</strong>ection, which is one such method, vectors for nucleic aciddelivery are associated with magnetic nanoparticles to accumulate or retain them at target sites with gradient magnetic fields. This concept <strong>of</strong> magnetic drug targetingenhances the efficiency <strong>of</strong> the delivery process over several orders <strong>of</strong> magnitude. This we have exploited to generate an integrated method <strong>of</strong> magnetic cell separation andgenetic modification, to boost the efficacy <strong>of</strong> oncolytic adenovirus and for establishing a tumor vaccine to treat feline fibrosarcoma. In other recent approaches we focus onextending the concept <strong>of</strong> magnet<strong>of</strong>ection with thermo-sensitive magnetic liposomes, magnetic microbubbles and magnetic aerosols for magnetically guided drug delivery.Declan M. Soden, PhDEndoscopic Electroporation: Targeted <strong>Gene</strong> Delivery to Intraluminal TissuesThe minimally invasive control <strong>of</strong> primary tumours is a critically important part in the treatment process as it relieves symptoms, forestalls complications, facilitates responsivenessto systemic therapies and is <strong>of</strong>ten curative. We have developed a device, which allows for an endoscopic cancer treatment, specifically targeting colorectal, gastricand oesophageal cancers by applying a brief electric pulse to the tumour. This greatly enhances tissue permeability maximizing the local/tumor absorption <strong>of</strong> therapeuticagents (drugs or DNA). Absorption occurs only in the area that has been treated with the electrical field and therefore is targeted to the tumor, leaving surrounding healthytissues unaffected. Our research has examined the efficacy <strong>of</strong> the device for targeted gene delivery to intraluminal tissues in large animals. We have compared the transfectionefficacy <strong>of</strong> a standard CMV driven reporter gene (b-galactosidase) in both murine and porcine tissues. In addition we have examined the efficacy <strong>of</strong> a DNA vectorcoding for a viral RNA polymerase encoded from the Semliki Forest Virus in comparison to the CMV driven vector.The treatment <strong>of</strong> spontaneous canine colorectal cancers has also been conducted using the device to facilitate local drug (bleomycin) absorption after tumor tissueelectroporation. The results have demonstrated the treatment to be effective and safe with complete tumor ablation noted in the two inoperable cases treated to date.All procedures were conducted as a simple colonoscopy type procedure with no adverse side effects recorded. A phase I clinical study with the device is due to commenceearly 2010 to treat patients with inoperable rectal cancer and electrochemotherapy.Kate E. Broderick, PhDDermal Electroporation Devices for Prophylactic DNA Vaccine DeliveryDuring the presentation, Dr. Broderick will discuss novel prototype dermal devices developed by Inovio and the pre-clinical Immunogenicity data generated from thesedevices in a number <strong>of</strong> animal models.Laurence JN Cooper, MD, PhDNon-viral <strong>Gene</strong> Transfer into Human T <strong>Cell</strong>sNon-viral approaches can be used to genetically modify T cells for therapy. We have combined electroporation with the Sleeping Beauty (SB) transposase/transposon DNAplasmid system to introduce desired transgenes. By improving the efficiency <strong>of</strong> electroporation with designer devices and improving the efficiency <strong>of</strong> integration by adaptingthe SB system for human translation, we seek to implement physical gene transfer systems for clinical trials.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 59Program ScheduleScientific Symposium 31510:30 am - 12:30 pmRoom: Marriott Ballroom Salons 2 & 3<strong>Gene</strong>tic Modification <strong>of</strong> Stem <strong>Cell</strong>s for Therapeutic ApplicationsChairFrederic D. Bushman, PhDSpeakersChrist<strong>of</strong> von Kalle, MD PhDThe Clonal Distribution <strong>of</strong> Insertion Site RepertoiresRetroviral vectors have been successfully used for the correction <strong>of</strong> inherited immune diseases in clinical trials. However, qualitative and quantitative in vivo monitoring <strong>of</strong>gene modified hematopoiesis in several clinical gene therapy trials revealed that vector induced side effects, ranging from subtle and not clinically obvious effects to clonaldominance and, rarely, leukemogenesis, have to be considered.Clonality data on oncoretroviral and lentiviral clinical gene therapy trials (i.e. CGD, SCID, WAS, ALD) show that integration site distribution is non-random and mayinfluence cellular gene expression, thus inducing changes in the clonality <strong>of</strong> the hematopoietic repopulation. Incorporation <strong>of</strong> next generation sequencing technologiessubstantially improve in depth analyses and monitoring <strong>of</strong> individual and global clonal contribution in a hitherto unexpected resolution. Prospective monitoring <strong>of</strong> vectorintegration sites in clinical gene therapy studies is feasible, can detect side effects <strong>of</strong> gene therapy at the clonal level, and may help to gain new insights into the basicmechanisms leading to specific clonal behaviour in vivo. Genome wide insertion studies will allow to improve the safety and performance <strong>of</strong> viral and non-viral integratingvector systems.Matthew H. Porteus, MD, PhD<strong>Gene</strong> Targeting with Zinc Finger Nucleases in Primary <strong>Cell</strong>s<strong>Gene</strong> targeting by homologous recombination has the potential to be both a safer and more efficacious approach to gene therapy. Therapeutically relevant rates <strong>of</strong> genetargeting have been achieved by using zinc finger nucleases in a variety <strong>of</strong> tissue culture cell lines and some primary human cells. We now demonstrate that therapeuticallyrelevant frequencies <strong>of</strong> gene targeting by homologous recombination can be achieved in primary cells and that these modified cells can be successfully transplantedback into a recipient. The demonstration that primary cells can be isolated, precisely modified ex vivo and then transplanted back into a recipient mimics the strategy weare working towards in humans as a cure for patients with monogenic diseases.Frederic D. Bushman, PhDHIV DNA Integration: Mechanism and ConsequencesThe lecture will cover integration by HIV and HIV-based vectors. Recent results from gene therapy trials will be presented. The lecture will also cover development <strong>of</strong>improved new methods for integration site isolation and analysis.Ko Mitani, PhDChromosomal Manipulation <strong>of</strong> Human Pluripotent Stem <strong>Cell</strong>s using Adenoviral VectorsIn order to fully utilize human induced pluripotent stem (iPS) cells and embryonic stem (ES) cells for basic research and clinical applications, it is essential to developefficient methods for homologous recombination (HR) in these cells. We have shown that, by delivering gene-targeting cassettes using helper-dependent adenoviralvectors (HDAdVs) into human ES/iPS cells, gene knock-in/-out has been efficiently achieved at both transcriptionally active and inactive loci. On average, one out <strong>of</strong>2 - 20 x 105 infected cells was homologous recombinant. The efficiencies <strong>of</strong> HR per chromosomal integration were 10 - 50% (30 - 90% with negative selection), whichwere ~100-fold higher than those obtained by other DNA delivery methods. These findings suggest that HDAdV-mediated HR is not only a highly efficient strategy forchromosomal manipulation <strong>of</strong> pluripotent stem cells but also a viable option for ex vivo gene repair therapy using iPS cells derived from patients <strong>of</strong> inherited diseases,which would overcome the risk <strong>of</strong> insertional mutagenesis.Friday, May 21 stLunch Break12:30 pm – 2:00 pm


60<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleOral Abstract Session 3202:00 pm - 4:00 pmRoom: Marriott Ballroom Salons 2 & 3Clinical <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Co-ChairsGuangping Gao, PhD & Matthew H. Porteus, MD, PhD(Abstracts 289-296; see pages 104-105)Oral Abstract Session 3212:00 pm - 4:00 pmRoom: Virginia SuiteRNA Virus VectorsCo-ChairsFabio Candotti, MD & Robert G. Hawley, PhD(Abstracts 297-304; see page 105)Oral Abstract Session 3222:00 pm - 4:00 pmRoom: Marriott Ballroom Salon 1AAV Vectors: Vector BiologyCo-ChairsDouglas M. McCarty, PhD & Matthew D. Weitzman, PhD(Abstracts 305-312; see pages 105-106)Friday, May 21 stOral Abstract Session 3232:00 pm - 4:00 pmRoom: Delaware Suite<strong>Gene</strong> <strong>Therapy</strong> for Lysosomal Storage Diseases and Other <strong>Gene</strong>tic DisordersCo-ChairsCary Harding, MD & Chester B. Whitley, PhD MD(Abstracts 313-320; see page 106)Oral Abstract Session 3242:00 pm - 4:00 pmRoom: Wilson ABCPhysical Methods <strong>of</strong> DeliveryCo-ChairsJeffrey Hughes, PharmD, PhD & Nancy Smyth Templeton, PhD(Abstracts 321-328; see pages 106-107)EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 61Program ScheduleOral Abstract Session 3252:00 pm - 4:00 pmRoom: Thurgood Marshall WCancer-Targeted <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Co-ChairsFrank Marini, PhD & Richard G. Vile, PhD(Abstracts 329-336; see page 107)Oral Abstract Session 3262:00 pm - 4:00 pmRoom: Maryland SuiteHematologic/Immunologic: BasicCo-ChairsDavid Klatzmann, MD, PhD & John F. Tisdale, MD(Abstracts 337-344; see pages 107-108)Break4:00 pm – 4:30 pmFriday, May 21 st


62<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program SchedulePresidential Symposium 3304:30 pm - 6:30 pmRoom: Marriott Ballroom Salons 1, 2 & 3Presidential SymposiumChairKenneth Cornetta, MDSpeakersFriday, May 21 stJohn J. Rossi, PhD Introduces Katherine A. High, MDKatherine A. High, MDOutstanding Achievement Award LectureEXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 63Program SchedulePresidential Symposium 330 — continuedJean Bennett, MD, PhDAn Aye for <strong>Gene</strong> <strong>Therapy</strong>: Lessons from a Clinical Trial for Congenital Blindness<strong>Gene</strong> therapy has the potential to reverse disease or prevent further deterioration <strong>of</strong> vision in patients with incurable inherited retinal degeneration. The recent demonstration<strong>of</strong> safe and stable recovery <strong>of</strong> retinal/visual function in 12 children and adults with Leber’s congenital amaurosis (LCA) due to RPE65 mutations in a trialbeing carried out at The Children’s Hospital <strong>of</strong> Philadelphia (CHOP), and encouraging results from two other clinical trials for LCA-RPE65 provide great hope for peoplewith other more common blinding diseases. This presentation will describe the latest safety and efficacy results in the LCA-RPE65 clinical trial being held at CHOP. Thisstudy is now >2.5 years past initiation. In addition, it will describe some <strong>of</strong> the challenges presented by the nature <strong>of</strong> the targeted disease itself, hurdles that havebeen navigated in order to conduct the study, and issues <strong>of</strong> importance for eventual approval <strong>of</strong> gene augmentation as a therapy for LCA and other blinding diseases.*This presentation involves contributions from the following additional individuals (listed alphabetically): Carmen Acerra, Defne Amado, Alberto Auricchio, SandroBanfi, Jeannette Bennicelli, Erin Bote, Yifeng Chen, Daniel Chung, Ann Fulton, Jeffrey Golden, Rebecca Grant, Bernd Hauck, Daniel Hui, Katherine A. High, BartLeRoy, Arkady Lyubarsky, Albert M. Maguire, Maureen G. Maguire, Kathleen Marshall, Federico Mingozzi, Jessica Morgan, Kristina Narfstrom, Stephen Orlin, EricA. Pierce, Settimio Rossi, Kenneth S. Shindler, Francesca Simonelli, Enrico M. Surace, Edwin Stone, Junwei Sun, Nasreen Syed, Francesco Testa, Nicholas J. Volpe,Jennifer Wellman, J. Fraser Wright, Gui-shuang Ying, Olga Zelenaia, Xiaosong (Sonia) ZhuFriday, May 21 stDavid A. Williams, MDNew Approaches to <strong>Gene</strong> <strong>Therapy</strong> for Fanconi AnemiaFA is a rare recessive syndrome characterized by bone marrow failure, congenital anomalies and a predisposition to malignancy. FA cells have a defect in DNA repairthat leads to increased spontaneous chromosomal breakage. Transgenic expression <strong>of</strong> FA genes in vitro corrects the increased chromosomal breakage and othercellular phenotypes <strong>of</strong> the disease. In a recent gene therapy trial, we have demonstrated that gene transfer per se is no longer a limitation to the effectiveness <strong>of</strong>gene therapy in this disease. Rather our clinical studies show that even early in the disease process there are significant deficiencies in the number and/or function<strong>of</strong> hematopoietic stem cells (HSCs) that can be collected and utilized as targets in gene transfer. One approach that could address this deficiency is the derivation<strong>of</strong> HSC from induced pluripotent stem (iPS) cells reprogrammed from non-blood somatic tissues. Important advantages <strong>of</strong> iPS cells for application <strong>of</strong> gene therapyare the apparent unlimited proliferative capacity <strong>of</strong> such cells (allowing production <strong>of</strong> large numbers <strong>of</strong> derived HSC) and the ability to clone such cells allowing bothsingle cell correction <strong>of</strong> genetic mutations and prospective molecular characterization <strong>of</strong> manipulated cells prior to therapeutic applications.


64<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleExhibit Hall Open & Networking Reception6:30 pm – 8:30 pmRoom: Exhibit Hall CFriday, May 21 stPoster Session II6:30 pm – 8:30 pmRoom: Exhibit Hall B South, CRNA Virus Vectors I(Abstracts 345 through 361; see pages 108-109)AAV Vectors II(Abstracts 362 through 377; see pages 109-110)Adenovirus and Other DNA Virus Vectors II(Abstracts 378 through 391; see page 110)Chemical and Molecular Conjugates II(Abstracts 392 through 407; see pages 110-111)<strong>Gene</strong>tic and Metabolic Diseases <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II(Abstracts 408 through 422 and Abstract 764 which has been moved from Poster Session III; see pages 111-112)Cardiovascular <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>(Abstracts 423 through 440; see pages 112-113)Ophthalmic Disorders and Technological Advances(Abstracts 441 through 453; see pages 113-114)Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II(Abstracts 454 through 468; see pages 114-115)Cancer – Immunotherapy II(Abstracts 469 through 487; see pages 115-116)Cancer-Oncolytic Viruses II(Abstracts 488 through 506; see pages 116-117)Cancer-Targeted <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II(Abstracts 507 through 520; Abstract 519 withdrawn; see page 117)Hematologic and Immunologic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II(Abstracts 521 through 534; see page 118)Immunologic & Host Responses in <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>(Abstracts 535 through 554; Abstract 541 withdrawn; see pages 118-119)Oligonucleotide & RNAi Therapeutics II(Abstracts 555 through 569; see pages 119-120)<strong>Cell</strong> Processing and Vector Production II(Abstracts 570 through 579; see pages 120-121)Late Breaking Science II(Abstracts 933 through 937; see page 121)EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 65Program ScheduleFoundation Symposium 3407:00 pm - 10:00 pmRoom: Marriott Ballroom Salons 2 & 3Progress and Prospects for <strong>Gene</strong> <strong>Therapy</strong> for CGD and other Primary ImmunodeficienciesSpeakersMary C. Dinauer, MD, PhD<strong>Gene</strong> <strong>Therapy</strong> for CGD - From Basic Discoveries to the Start <strong>of</strong> Clinical TrialsAlessandro Aiuti, MD, PhDConditioning Regimens for Haematopoietic <strong>Gene</strong> <strong>Therapy</strong>Harry L. Malech, MDUpdate on <strong>Gene</strong> <strong>Therapy</strong> Trials for CGDManuel Grez, PhDThe European Experience on GT for CGDAdrian J. Thrasher, MD, PhDUpdate on Clinical Trials for XC1-SCID, ADA and ALDFoundation Symposium 340 is supported by the following ASGCT partner:Friday, May 21 st


66<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleSaturday, May 22ASGCT Business Meeting8:00 am - 8:30 amRoom: Delaware SuiteA light continental breakfast will be served at 7:45 am.Scientific Symposium 4008:30 am - 10:30 amRoom: Maryland SuiteLate Stage Industry Clinical TrialsCo-ChairsDouglas J. Jolly, PhD & Thomas TillettSpeakersWilliam W. Li, MD<strong>Gene</strong> Transfer <strong>of</strong> FGF1 for Critical Limb Ischemia: A Phase III Clinical TrialRobert Sims, MDProgress in the Development <strong>of</strong> Sipuleucel-T for the Treatment <strong>of</strong> Men with Metastatic, Castrate Resistant Prostate CancerSipuleucel-T is an autologous cellular immunotherapy designed to elicit an antitumor response against prostate cancer. Sipuleucel-T is manufactured by incubating antigenpresenting cell precursors with a fusion protein composed <strong>of</strong> prostatic acid phosphatase linked to GM-CSF. Sipuleucel-T is then reinfused into the patient and the process isrepeated every 2 weeks for a total <strong>of</strong> 3 infusions. It has been evaluated in 3 randomized placebo-controlled Phase 3 studies in men with metastatic castrate-resistant prostatecancer, which have demonstrated clear evidence <strong>of</strong> prolongation <strong>of</strong> survival. The presentation will include an update on the regulatory status <strong>of</strong> sipuleucel-T currentclinical trials, as well as planned future trials.Ryuichi Morishita, MD, PhDDevelopment <strong>of</strong> HGF <strong>Gene</strong> <strong>Therapy</strong> Drug (Collategen)As HGF is a most potent mitogen to stimulate angiogenesis, a multi-center randomized, placebo-controlled, double blind study for critical limb ischemia (CLI) using HGFplasmid DNA was conducted, based on phase I/IIa open label study at Osaka University. Improvement rate was 70.4 % in HGF and 30.8% in placebo in Fontaine III/Rutherford 4 patients (P=0.014). HGF gene therapy achieved a significantly higher improvement rate <strong>of</strong> ischemic ulcers compared with placebo (100% vs. 40%,P=0.018). No major safety problems were observed. Based on phase III data, NDA was submitted to Japan Regulatory Agency (MHWL) on 2008. In USA, phase IIIstudy using HGF gene therapy will be started in 2010, since phase III USA study obtained SPA from FDA.Sander van Deventer, MD, PhDGlybera for Lipoprotein Lipase DeficiencySaturday, May 22 ndEXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 67Program ScheduleScientific Symposium 4018:30 am - 10:30 amRoom: Virginia SuiteLocal Delivery <strong>of</strong> Chemical <strong>Gene</strong> <strong>Therapy</strong> – Mechanisms, Quantitative Understanding and Applications <strong>of</strong> Biodegradable CarriersChairErnst Wagner, PhDSpeakersLonnie D. Shea, PhDLocalized <strong>Gene</strong> Delivery by Biomaterial ImmobilizationBiomaterials are combined with gene delivery to provide a structure that guides cell growth, while also inducing the expression <strong>of</strong> inductive factors that drives the cellularprocesses leading to functional tissues. We have investigated immobilization <strong>of</strong> gene therapy vectors to biomaterials as a means to maintain the vectors locally, co-localizethe vectors to the surface for cell adhesion, and stabilize the vectors against degradation. The design principles we have developed for hydrogels and microporous scaffoldswill be presented, as well as their use with in vitro tissue models and in vivo regeneration. These biomaterial scaffolds provide opportunities to enhance or control genetransfer relative to more traditional delivery methods (e.g., injection), and provide an enabling technology to molecularly dissect tissue formation or to promote functionaltissue regeneration.Aliasger K. Salem, PhDPulsatile Release <strong>of</strong> Biomolecules from Polydimethylsiloxane ChipsCytosine-phosphorothioate-guanine oligodeoxynucleotides (CpG ODN) is a recent class <strong>of</strong> immunostimulatory adjuvants that includes unmethylated CpG dinucleotidesequences similar to those commonly found in bacterial DNA. CpG ODN specifically triggers toll like receptor 9 (TLR9), which is found within phagoendosomes <strong>of</strong> antigenpresenting cells (APCs) such as dendritic cells (DCs). CpG ODN triggers activation and maturation <strong>of</strong> DCs and helps to increase expression <strong>of</strong> antigens. CpG ODN can beused to induce polarized Th1 type immune responses. We have shown that antigens and CpG ODN must be co-delivered to generate the most potent therapeutic antigenspecificimmune responses. We have developed a series <strong>of</strong> delivery vehicles to ensure co-delivery <strong>of</strong> antigens and CpG ODN to the same APCs and to significantly increaseuptake by APCs. These strategies can result in antigen-specific immune responses that are 5 to 500-fold greater than administration <strong>of</strong> antigen alone. The approaches wehave developed to achieve this include the utilization <strong>of</strong> conjugate molecules, multicomponent nanorods, biodegradable microparticles, cell-microparticle hybrids and finallypolydimethylsiloxane based pulsatile release chips.David M. Lynn, PhDA ‘Multilayered’ Approach to the Delivery <strong>of</strong> DNA: Exploiting the Structure <strong>of</strong> Polyelectrolyte Multilayers to Promote Surface-MediatedTransfection and Tunable Multi-Agent DeliveryMethods for the layer-by-layer deposition <strong>of</strong> oppositely charged polymers on surfaces can be used to fabricate ultrathin multilayered films (or ‘polyelectrolyte multilayers’)using a broad range <strong>of</strong> natural and synthetic materials, including DNA. If the individual components <strong>of</strong> these assemblies are designed appropriately, these methods alsoprovide opportunities to design thin films that provide control over the release <strong>of</strong> DNA and other agents from surfaces. This talk will describe recent work from our laboratoryon the design <strong>of</strong> new polymers and the fabrication <strong>of</strong> thin films and coatings that provide spatial and temporal control over the release <strong>of</strong> DNA from surfaces in vitroand in vivo. These methods provide tunable control over the surface-mediated release <strong>of</strong> DNA over periods ranging from several hours to several days, weeks, or months,and can also be used to fabricate nanostructured films with hierarchical structures that permit control over the release <strong>of</strong> two or more DNA constructs with separate,distinct, and predictable release pr<strong>of</strong>iles (e.g., rapid release <strong>of</strong> one DNA construct followed by the slower, sustained release <strong>of</strong> a second DNA construct). With furtherdevelopment, these approaches could contribute to the design <strong>of</strong> thin films and coatings capable <strong>of</strong> delivering precise and well-defined quantities <strong>of</strong> multiple different DNAconstructs (or combinations <strong>of</strong> DNA and other therapeutic agents) in a broad range <strong>of</strong> fundamental and applied contexts.Daniel G. Anderson, PhDCombinatorial Development <strong>of</strong> Biomaterials for <strong>Gene</strong> DeliveryHigh throughput, combinatorial approaches have revolutionized small molecule drug discovery. Here we describe our work on high throughput methods for developing andcharacterizing biomaterials for genetic therapy and cell therapy. We have developed automated methods allowing for rapid synthesis <strong>of</strong> 1000’s <strong>of</strong> biomaterials, as wellas the testing <strong>of</strong> their chemical, material, and biological properties. These methods have also been applied towards the development <strong>of</strong> new methods to control stem cellbehavior, as well as vehicles for drug delivery. In particular, these combinatorial libraries <strong>of</strong> different biomaterials have enabled new methods for non-viral gene therapyand siRNA delivery.Saturday, May 22 nd


68<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleScientific Symposium 4028:30 am - 10:30 amRoom: Delaware SuiteEngineered T <strong>Cell</strong>s in the Clinic: Emerging Clinical and Regulatory IssuesChairJacqueline Corrigan-Curay, MD, JDSpeakersDaniel M. Takefman, PhD<strong>Gene</strong> Modified <strong>Cell</strong> Based Therapies: FDA PerspectivesThe purpose <strong>of</strong> this presentation is to provide FDA perspectives on gene modified cell based therapies. This presentation will outline some key regulatory considerationsfor the clinical use <strong>of</strong> patient specific gene therapies, such as engineered T-cells. The emphasis with be on manufacturing considerations as unique issues arise whenmanufacturing these types <strong>of</strong> products for clinical use.Joy Cavagnaro, PhD, DABTLimitations <strong>of</strong> Preclinical Studies for Predicting Safety <strong>of</strong> Uniquely Human Specific TherapiesCarl H. June, MDProlonged Engraftment and Decade Long Safety Record <strong>of</strong> Engineered T <strong>Cell</strong>sIncreasing data from pre-clinical experiments and pilot clinical trials illustrate the potential <strong>of</strong> engineered T cells for therapy <strong>of</strong> cancer, chronic infection and autoimmunity.A key issue facing the field has been limited persistence <strong>of</strong> engineered T cells in most trials. Our clinical data indicates that T cells engineered to express a chimeric antigenreceptors can exhibit long term survival, and that in HIV infected patients, engineered T cells have a considerable safety record.PanelHelen E. Heslop, MDMichel Sadelain, MD, PhDSteven A. Rosenberg, MD, PhDSaturday, May 22 ndScientific Symposium 4038:30 am - 10:30 amRoom: Marriott Ballroom Salon 1Mechanisms <strong>of</strong> Innate Immunity Influencing <strong>Gene</strong> <strong>Therapy</strong>ChairDavid B. Weiner, PhDSpeakersValder Arruda, MD, PhD<strong>Gene</strong> <strong>Therapy</strong> for Tolerance Induction in Large Animal ModelsThe formation <strong>of</strong> neutralizing antibodies (inhibitors) to clotting Factor VIII or FIX is a major safety concern on the development <strong>of</strong> novel therapies for hemophilia. Inhibitorformation renders the protein replacement ineffective resulting in both high morbidity and mortality. Therefore the prevention or eradication <strong>of</strong> inhibitors is <strong>of</strong> fundamentalimportance. To eliminate the presence <strong>of</strong> inhibitors in hemophilia, an immune tolerance induction strategy consisting <strong>of</strong> large amounts <strong>of</strong> FVIII/FIX injected on a dailybasis for long periods <strong>of</strong> time (months to years) results in variable success rates. We will review gene-based strategies to prevent inhibitor formation in inhibitor-pronedogs. Notably, recent evidence demonstrates the possibility <strong>of</strong> the use <strong>of</strong> gene-based therapy for the eradication <strong>of</strong> preexisting inhibitors to FVIII from studies in hemophiliaA dogs. Emerging evidence on the role <strong>of</strong> T regulatory cells in modulating immune responses to the therapeutic protein now endogenously expressed by gene therapyopens a novel potential application <strong>of</strong> gene therapy, not only to prevent, but also to eradicate inhibitors in hemophilia and other diseases.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 69Program ScheduleYiping Yang, MD, PhDInnate Immunity in AAV-mediated <strong>Gene</strong> <strong>Therapy</strong>Recombinant adeno-associated viruses (AAVs) have been used widely for in vivo gene therapy. However, adaptive immune responses to AAV have posed a significanthurdle in clinical application <strong>of</strong> AAV vectors. Recent advances have suggested a crucial role for innate immunity in shaping adaptive immune responses. Here I will discusshow AAV activates the innate immune system and the potential implications for improving the outcome <strong>of</strong> AAV-mediated gene therapy.David B. Weiner, PhDHigh Immune Potency <strong>of</strong> Highly Optimized EP DNA Vaccines in Animal Models and HumansDNA vaccines are an important emerging vaccine technology, however the immune responses induced by past generations <strong>of</strong> DNA vaccines were poor in larger animalsincluding nonhuman primates and importantly humans. We have recently utilized a combination <strong>of</strong> technologies for plasmid optimization, combined with enhanced EPdelivery to dramatically improve the immune potency <strong>of</strong> this platform. These new EP systems also <strong>of</strong>fer improved tolerability and effectiveness. We will present data inanimal models and humans illustrating immune aspects <strong>of</strong> the technology for application to human infectious diseases.Michael T. Lotze, MDMicroRNAs in Immune Regulation-Opportunities for Cancer <strong>Gene</strong> <strong>Therapy</strong>Initially considered as means to regulate tissue differentiation and disordered in cancer, miRNAs [miR] have now been identified that are induced in response to hypoxia,oncogenic signaling, pathogens and tissue damage or injury. Each miR can regulate the translation <strong>of</strong> up to 100 separate mRNAs. Means to either express or knockdownmiRNA expression [so-called antagomiRs] have been operationalized in murine models and represent exciting novel targets for modifying immunity.Scientific Symposium 4048:30 am - 10:30 amRoom: Wilson ABCMicroRNA and siRNA Roles in Disease Progression and Development <strong>of</strong> Novel Therapeutic TargetsChairMaria G. Castro, PhDSpeakersAnna M. Krichevsky, PhDMicroRNAs as Targets for Glioma <strong>Therapy</strong>MicroRNA (miRNA) is a class <strong>of</strong> small RNA molecules involved in the regulation <strong>of</strong> expression <strong>of</strong> at least 30% <strong>of</strong> human genes and, therefore, it controls all principalcellular processes, including cell division and cell death. In my talk, I will describe our approach to reveal key miRNA regulators in human diseases and focus on miRNAfunctions in the development <strong>of</strong> human brain tumors. Our recent work presents pro<strong>of</strong>-<strong>of</strong>-principles that specific inhibition <strong>of</strong> a single oncogenic miRNA could provide a noveltherapeutic approach for physiological modulation <strong>of</strong> multiple proteins whose expression and activity is de-regulated in cancer. It also suggests a diagnostic potential forthis novel class <strong>of</strong> molecules.M. Maral Mouradian, MDMicroRNAs in Parkinson’s Disease and Other Neurodegenerative Disordersalpha-Synuclein is a key protein that accumulates in the brains <strong>of</strong> patients with Parkinson’s disease and related neurodegenerative disorders. Evidence from clinical geneticstudies, animal models and cell biologic experiments indicate that elevated levels <strong>of</strong> this protein are toxic. This presentation will discuss down-regulation <strong>of</strong> alpha-synucleinexpression by microRNA leading to neuroprotection.Sean E. Lawler, PhDMicroRNA Alterations and Functions in GlioblastomaMicroRNAs have recently emerged as important regulators <strong>of</strong> gene expression with potential therapeutic applications in many human diseases. We have identified extensivealterations in microRNA expression in the malignant brain tumor glioblastoma multiforme. We have shown that altered microRNAs play roles in proliferation, stemcell-like behavior, and glioma cell migration. This talk will focus on the underlying mechanisms <strong>of</strong> microRNA action in glioblastoma, and their therapeutic potential.Saturday, May 22 nd


70<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleAl Charest, PhDNanoparticle-Mediated Delivery <strong>of</strong> siRNA in a Pre-Clinical Mouse Model <strong>of</strong> Glioblastoma MultiformeSmall interfering RNAs mediate cleavage <strong>of</strong> specific, complementary mRNA sequences and thus regulate gene expression. Not surprisingly, their use for treatment <strong>of</strong>diseases that are rooted in aberrant gene expression, such as cancer, has become a paradigm that has gained wide interest. We utilized dendrimer-conjugated magnet<strong>of</strong>luorescentnanoparticles to deliver anti-EGFR siRNA in vivo in an EGFR-driven transgenic model <strong>of</strong> glioblastoma. These nanoparticles were well tolerated in the CNS anddemonstrated specific and significant suppression <strong>of</strong> EGFR expression by convection-enhanced delivery to the mouse brain. Our data demonstrate the feasibility <strong>of</strong> siRNAdelivery in animals using a nanoplatform to efficiently knockdown target proteins.Scientific Symposium 4058:30 am - 10:30 amRoom: Marriott Ballroom Salons 2 & 3Special Considerations with Using Viral Vectors for <strong>Gene</strong> Modification <strong>of</strong> Stem <strong>Cell</strong>sChairPhilip D. Gregory, PhDSpeakersMatthew Hirsch, PhDThe AAV Induced DNA Damage Response in Human Pluripotent <strong>Cell</strong>sThe ability to genetically engineer stem cells ex vivo, then re-introduce them into a host for therapeutic purposes is an attractive approach for a variety <strong>of</strong> human geneticdiseases. Critical in this process is the ability to efficiently deliver transgenic DNA into the nucleus <strong>of</strong> stem cells in a manner that is well tolerated by the cell and withoutextensive host chromosome integration. A concern at the cellular level is the characterization <strong>of</strong> the cell type modified, and in particular, understanding the relationshipbetween “stemness” and oncogenesis. For example, the DNA damage response <strong>of</strong> pluripotent human embryonic stem cells (hESCs), in general, differs from that <strong>of</strong>differentiated cells in that checkpoint functions are impaired and that these cells appear primed for rapid apoptosis. In this work, incidentally, we demonstrate that theadeno-associated virus (AAV) origin <strong>of</strong> replication, found ubiquitous in nature and currently used in human clinical trials for DNA delivery, induces a rapid, specific, andcomplete apoptotic response following transduction <strong>of</strong> non-differentiated hESCs. <strong>Cell</strong> cycle analysis by flow cytometry suggested that AAV-infected hESCs fail to induce aG1 checkpoint response and display an accumulation <strong>of</strong> cells in early S phase followed by apoptosis, suggesting that the AAV ITRs mimic stalled replication forks and/orperhaps uncapped telomeres. In contrast to reports <strong>of</strong> AAV-induced apoptosis in p53-deficient cancers, hESCs depleted <strong>of</strong> p53 survived significantly longer following AAVinfection than the parental control. Collectively, these data demonstrate that the AAV origin <strong>of</strong> replication (present on all AAV vectors) induces a lethal signaling cascadeduring S-phase in hESCs and a subsequent p53-dependent apoptotic response. This unexpected phenomenon is the first report <strong>of</strong> AAV vector toxicity in a wild type humancell and is reminiscent <strong>of</strong> previous reports <strong>of</strong> toxicity in p53-deficient cancers cells following AAV transduction. Understanding such unique DNA damage responses in stemcells and cancers following DNA delivery is necessary for optimization <strong>of</strong> conditions for efficient genetic engineering and safety in cell replacement therapies.Saturday, May 22 ndPhilip D. Gregory, hDStem <strong>Cell</strong> Modification with Zinc Finger NucleasesFrederic Paques, PhDMeganucleases and Viral Vectors: Alternative or Complementary ApproachesTargeted approaches have emerged today as an alternative to current random insertion strategies for gene therapy. Meganucleases, the most specific natural endonucleasesrepresent ideal tools for targeted genome editing, or “genome surgery”. Natural as well as engineered can be used to induce up to 20% <strong>of</strong> gene insertion into chosenhuman genes. The introduction <strong>of</strong> meganucleases into different cell types can be achieved by various means, including viral and non viral vectorization.Andrew M. Scharenberg, MD<strong>Gene</strong> Editing Using Lentiviral Expression <strong>of</strong> Homing Endonucleases<strong>Gene</strong> editing and targeted integration approaches to gene therapy <strong>of</strong>fer the potential to avoid idiosyncratic side effects associated with the random integration <strong>of</strong>promoter/gene cassettes in traditional gene therapy. The Northwest Genome Engineering Consortium is exploring the development <strong>of</strong> lentiviral vector-mediated expression<strong>of</strong> homing endonucleases for gene editing and targeted integration in hematopoietic cells. Preliminary data on the efficacy <strong>of</strong> this approach in cultured and primaryhematopoietic cell models will be presented, and challenges and future directions in the field will be discussed.Break10:30 am – 11:00 amEXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 71Program ScheduleOral Abstract Session 41011:00 am - 1:00 pmRoom: Delaware SuiteMusculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Co-ChairsJeffrey Chamberlain, PhD & Paula Clemens, MD(Abstracts 580-587; see page 122)Oral Abstract Session 41111:00 am - 1:00 pmRoom: Virginia SuiteChemical and Molecular ConjugatesCo-ChairsHideyoshi Harashima, PhD & Millicent O. Sullivan, PhD(Abstracts 588-595; see page 122)Oral Abstract Session 41211:00 am - 1:00 pmRoom: Wilson ABCCardiovascular <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Co-ChairsRoger J. Hajjar, MD & Xiao Xiao, PhD(Abstracts 596-603; see page 123)Oral Abstract Session 41311:00 am - 1:00 pmRoom: Marriott Ballroom Salons 2 & 3Cancer-Oncolytic Viruses IICo-ChairsRonald Rodriguez, MD, PhD & Masato Yamamoto, MD, PhD(Abstracts 604-611; Abstract 605 withdrawn; Abstract 191 moved from Poster Session I; see page 123)Oral Abstract Session 41411:00 am - 1:00 pmRoom: Maryland SuiteHematologic/Immunologic: Clinical, Preclinical and SafetyCo-ChairsGay Crooks, MD & Adrian J. Thrasher, MD, PhD(Abstracts 612-619; see page 124)Saturday, May 22 nd


72<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleOral Abstract Session 41511:00 am - 12:00 pmRoom: Thurgood Marshall WLung and Respiratory Disease <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Co-ChairsTerence R. Flotte, MD & Maria Limberis, PhD(Abstracts 620-623; see page 124)Oral Abstract Session 41612:15 pm - 1:15 pmRoom: Thurgood Marshall W<strong>Gene</strong> RegulationCo-ChairsOlivier Danos, PhD & Giuliana Ferrari, PhD(Abstracts 624-627; see pages 124-125)Oral Abstract Session 41711:00 am - 1:00 pmRoom: Marriott Ballroom Salon 1Stem <strong>Cell</strong> TherapiesCo-ChairsCarolyn Lutzko, PhD & Alice F. Tarantal, PhD(Abstracts 628-635; see page 125)Lunch Break1:00 pm – 3:00 pmMeet-the-Investigator Lunch 4201:00 pm - 2:15 pmRoom: Balcony AStem <strong>Cell</strong>sSpeakersJames Ellis, PhD & Carolyn Lutzko, PhDSaturday, May 22 ndMeet-the-Investigator Lunch 4211:00 pm - 2:15 pmRoom: Balcony BRoles <strong>of</strong> miRNAsSpeakersMark A. Kay, MD, PhD & Luigi M. Naldini, MD, PhDEXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 73Program ScheduleOral Abstract Session 4303:00 pm - 5:00 pmRoom: Maryland SuiteAdenovirus and Other DNA Virus VectorsCo-ChairsAnja Ehrhardt, PhD & Florian Kreppel, PhD(Abstracts 636-643; see pages 125-126)Oral Abstract Session 4313:00 pm - 5:00 pmRoom: Delaware SuiteInfectious Diseases and VaccinesCo-ChairsAnn Leen, PhD & Bruce E. Torbett, PhD, MSPH(Abstracts 644-651; see page 126)Oral Abstract Session 4323:00 pm - 5:00 pmRoom: Marriott Ballroom Salons 2 & 3Cancer – Immunotherapy IICo-ChairsStephen Gottschalk, MD & David B. Weiner, PhD(Abstracts 652-659; see pages 126-127 )Oral Abstract Session 4333:00 pm - 5:00 pmRoom: Thurgood Marshall WOligonucleotide & RNAi Therapeutics IICo-ChairsPaloma H. Giangrande, PhD & Peter M. Glazer, MD, PhD(Abstracts 660-687; see page 127)Oral Abstract Session 4343:00 pm - 5:00 pmRoom: Marriott Ballroom Salon 1<strong>Cell</strong> Processing and Vector ProductionCo-ChairsStephen M. Kaminsky, PhD & J. Fraser Wright, PhD(Abstracts 668-675; see pages 127-128)Saturday, May 22 nd


74<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Program ScheduleOral Abstract Session 4353:00 pm - 5:00 pmRoom: Wilson ABC<strong>Gene</strong> <strong>Therapy</strong> for Inborn Errors <strong>of</strong> MetabolismCo-ChairsDwight Koeberl, MD, PhD & Brendan Lee, MD, PhD(Abstracts 676-683; see page 128)Oral Abstract Session 4363:00 pm - 5:00 pmRoom: Virginia SuiteLate Breaking SessionCo-ChairsGuangping Gao, PhD & Stanton L. Gerson, MD(Abstracts 914-921; see pages 128-129)Poster Hall Social (use your drink ticket at tonight's social)5:00 pm – 6:00 pmRoom: AtriumSaturday, May 22 ndPoster Session III6:00 pm – 8:00 pmRoom: Exhibit Hall CRNA Virus Vectors II(Abstracts 684 through 696; see page 129)AAV Vectors III(Abstracts 697 through 711; see page 130)Adenovirus and Other DNA Virus Vectors III(Abstracts 712 through 725; see pages 130-131)DNA Vectorology & <strong>Gene</strong> Targeting(Abstracts 726 through 741; see pages 131-132)Chemical and Molecular Conjugates III(Abstracts 742 through 757; see pages 132-133)<strong>Gene</strong>tic and Metabolic Diseases <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> III(Abstracts 758 through 772; Abstract 764 has been moved to Poster Session II after Abstract 422; see page 133)Neurodegenerative Disorders, Spinal Cord and Trauma(Abstracts 773 through 783; see page 134)Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> III(Abstracts 784 through 796; see pages 134-135)Infectious Diseases and Vaccines: Non-Vaccine Related <strong>Gene</strong> Transfer Research(Abstracts 797 through 808; see pages 135-136)Cancer – Immunotherapy III(Abstracts 809 through 827; see pages 136-137)EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 75Program ScheduleCancer-Oncolytic Viruses III(Abstracts 828 through 842; Abstract 838 withdrawn; see page 137)Cancer-Targeted <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> III(Abstracts 843 through 856; see pages 137-138)Hematologic and Immunologic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> III(Abstracts 857 through 871; Abstract 869 has been move to Poster Session I after Abstract 233; see pages 138-139)Lung and Respiratory Disease <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>(Abstracts 872 through 881; see pages 139-140)Oligonucleotide & RNAi Therapeutics III(Abstracts 882 through 897; see pages 140-141)Stem <strong>Cell</strong> Therapies(Abstracts 898 through 913; see page 141)Late Breaking Science III(Abstracts 938 through 947; see page 142)Annual Meeting ConcludesSaturday, May 22 nd


76<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Exhibit Hall Floor PlanASGCT 2010 Annual MeetingMay 19-22, 2010 • Marriott Wardman Park Hotel • Washington, DCSpeaker Ready RoomRegistrationEXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 77Exhibitor DescriptionsAldevron3233 15 th Street, SouthFargo, ND 58104Telephone: (701) 297-9256Fax: (701) 280-1642E-mail: miller@aldevron.comWebsite: www.aldevron.comBooth Number: 506Aldevron is a leading contract manufacturing organization specializing in plasmidDNA, protein, antibody, DNA vaccine screening and genetic immunization(GIA) services. We manufacture biologics for research, clinical, and diagnosticapplications including ready-to-use AAV helper, cytokine, and reporter plasmids.Aldevron’s capacity and throughput capabilities support any project in bacterial,insect, and mammalian cell systems. By combining innovative technologies withbest-in-class project management, our team ensures that we consistently exceedyour expectations.<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>555 East Wells StreetSuite 1100Milwaukee, WI 53202Telephone: (414) 278-1341Fax: (414) 276-3349E-mail: info@asgct.orgWebsite: www.asgct.orgBooth Number: 609 & 611Stop by the ASGCT membership booth to pick-up your Abstracts-on-Disc and theEducation Session CD. Find out where the 2011 Annual Meeting will be heldand other <strong>Society</strong> information.Biologics Consulting Group, Inc.1317 King StreetAlexandria, VA 22314Telephone: (703) 739-5695Fax: (703) 548-7457E-mail: mhawkins@bcg-usa.comWebsite: www.bcg-usa.comBooth Number: 401Biologics Consulting Group, Inc., is an international firm providing regulatoryand product development advice on the development and commercialization <strong>of</strong>biologicals, drugs, devices, diagnostics, and combination products. Since 1993,we have helped hundreds <strong>of</strong> products reach the marketplace. Our consultantscome from the executive/senior tiers <strong>of</strong> FDA and industry.BioSpherix, Ltd.19 Demott St.Lacona, NY 13083Telephone: (315) 387-3414Fax: (315) 387-3415E-mail: kmurray@biospherix.comWebsite: www.biospherix.comBooth Number: 210Closed-incubation-system for clinical cell cultures. Multiple, independent, highperformance, O2/CO2 incubators open into aseptic isolation modules with identicalcontrolled atmospheres for uninterrupted conditions during cell handling. Can bea cGMP compliant mini-facility, <strong>of</strong>fering cell and gene therapists an economical andpractical alternative to expensive brick-and-mortar walk-in clean rooms.Caliper Life Sciences68 Elm StreetHopkinton, MA 01748Telephone: (508) 435-9500Fax: (508) 435-3439Website: www.caliperls.comBooth Number: 514Caliper Life Sciences is a leading supplier <strong>of</strong> tools and services for life scienceresearch including IVIS in vivo imaging systems. IVIS is the most publishedplatform for sensitive fluorescence and bioluminescence imaging in both 2D and3D providing the insight to view complex biological interactions such as geneexpression or stem cell engraftment in longitudinal studies <strong>of</strong> whole animals.Center for <strong>Cell</strong>ular and Molecular Therapeutics at theChildren’s Hospital <strong>of</strong> PhiladelphiaRoom 5000Colket Translational Research Building3501 Civic Center Blvd.Philadelphia, PA 19104Telephone: (267) 426-5256Fax: (215) 590-3660E-mail: sunj@email.chop.eduBooth Number: 313


78<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Exhibitor DescriptionsData Unlimited International, Inc.15881-B Crabbs Branch WayRockville, MD 20855Telephone: (240) 631-7933Fax: (240) 631-7937E-mail: contact@duii.comWebsite: www.duii.comBooth Number: 211Starfruit Technologies® is s<strong>of</strong>tware/database/computer/barcode for DNAdiagnostic, genome/cancer research, immunogenetic labs – enables sample,patient, biorepository, billing databases; mutation, variant, disease, genotypedatabases, SNP databank; instrument interface, barcode, electronic signature,QA/QC; worksheet, workflow, inventory, protocol, image; epidemiologicalfrequency, trend, geographical distribution, disease/disorder susceptibility,reporting; comply with 21CFR11, CAP, CLIA, HIPPA, PHI.Elsevier - Academic Press15807 Quince Orchard RoadN. Potomac, MD 20878Telephone: (240) 477-8564Fax: (240) 477-8571E-mail: b.karafin@elsevier.comBooth Number: 406As the world’s leading publisher <strong>of</strong> science and health information, Elsevierserves more than 30 million scientists, students, and health and informationpr<strong>of</strong>essionals worldwide. Visit Elsevier for the latest clinical trial resources, suchas: Clinical and Translational Science: Principles <strong>of</strong> Human Research by DavidRobertson and Gordon H. Williams.Exemplar <strong>Gene</strong>tics958 N. MainSioux Center, IA 51250Telephone: (712) 722-2767Fax: (712) 722-2126E-mail: troy.arends@exemplargenetics.comWebsite: www.exemplargenetics.comBooth Number: 408Exemplar <strong>Gene</strong>tics is enabling discovery through the creation <strong>of</strong> large animalmodels <strong>of</strong> human disease. Through proprietary techniques and licensedprocesses, we have been successful in creating the only commercially availablelarge animal model that replicates human disease. These techniques can beutilized to create models <strong>of</strong> interest in any field.Institutional Biosafety Committee Services (IBC Services)2103 Harrison Av NWPMB 2436Olympia, WA 98502-2607Telephone: (360) 252-2850Fax: (360) 252-2820E-mail: ibcs@wirb.comWebsite: www.ibcservicepoint.comBooth Number: 505IBC Services establishes and provides administrative support for local IBCsreviewing gene transfer clinical trials and related rDNA research. We have servedmedical practices, hospitals and health systems, and VA and university settingsin 46 states and abroad. Broad experience from first-in-human and single-subjectprotocols to multicenter late-phase trials.Lovelace Respiratory Research Institute2425 Ridgecrest Dr. SEAlbuquerque, NM 87108Telephone: (505) 348-9457/9557Fax: (505) 348-4983E-mail: JBenson@LRRI.orgWebsite: www.lrri.orgBooth Number: 307The Lovelace Respiratory Research Institute is a private not-for-pr<strong>of</strong>it biomedicalresearch organization. Lovelace <strong>of</strong>fers animal models <strong>of</strong> disease, evaluation <strong>of</strong>efficacy <strong>of</strong> potential new therapeutics, and pre-clinical assessments includingevaluation <strong>of</strong> pharmacokinetics/biodistribution in large and small animalmodels. Studies are routinely performed under GLP guidelines.Mary Ann Liebert, Inc.140 Huguenot StreetNew Rochelle, NY 10801Telephone: (914) 740-2100Fax: (914) 740-2101E-mail: info@liebertpub.comWebsite: www.liebertpub.comBooth Number: 510Mary Ann Liebert, Inc., publishes authoritative peer-reviewed journals in newand promising areas <strong>of</strong> science and biomedical research including Human <strong>Gene</strong><strong>Therapy</strong>, (www.liebertpub.com/hum), named one <strong>of</strong> the 100 most influentialJournals in Biology & Medicine over the last 100 years! Visit us @ booth#510.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 79Exhibitor DescriptionsMeridian Life Science, Inc.3471 River Hills DriveCincinnati, OH 45244Telephone: (513) 271-3700Fax: (513) 271-3762E-mail: Shirley.Torrence@meridianbioscience.comWebsite: www.meridianlifescience.comBooth Number: 208MLS provides contract R&D, process development, and clinical cGMPbiomanufacturing for vaccines, viral challenge materials, vectored genetherapies, VLPs, and r-proteins from BEVS. With over 25 yrs. <strong>of</strong> cell culture, virusproduction, protein purification, assay development and regulatory experience,MLS is your trusted partner for preclinical to Phase I/II manufacturing.Molmed S.p.AVia Olgettina, 58Milan, Italy 20132Telephone: +39 0221277257Fax: +39 0221277325E-mail: simona.laseta@molmed.comWebsite: www.molmed.comBooth Number: 209MolMed is a medical biotechnology company with a primary focus on developingnovel cell/gene therapies for treating cancer. The in-house GMP facility allowedMolMed to be involved in projects as GMP manufacturer. We are glad to <strong>of</strong>feryou our solutions for GMP cell/gene therapy, from early stage to advancedclinical development.Nanosight6660 N. High Street, Suite 2AWorthington, OH 43085Telephone: (614) 888-0023Fax: (614) 987-0045E-mail: jim.munhall@nanosight.comWebsite: www.nanosight.comBooth Number: 405Nanosight develops and manufactures instruments for Counting, Sizing andVisualizing Nanoparticles from 10 nm to 2.0 µm. Our unique NanoparticleTracking Analysis (NTA) technology allows for the measurement <strong>of</strong> DiffusionCoefficients <strong>of</strong> individual nanoparticles or viruses and determines the Count,Size & Concentration <strong>of</strong> the particle dispersions. Polydispersed and multi-modalsamples are quickly identified and resolved. Direct Visualization <strong>of</strong> the dispersionbeing measured is displayed and a video <strong>of</strong> the particles being measured iscreated and stored along with the size distribution data. Options are available forfluorescence measurements.National <strong>Gene</strong> Vector Biorepository980 West Walnut Street, R3 C602Indianapolis, IN 46202Telephone: (317) 274-4519Fax: (317) 278-3327E-mail: lrubin@iupui.eduWebsite: https://www.ngvbcc.orgBooth Number: 512The National <strong>Gene</strong> Vector Biorepository provides support for gene therapy researchby <strong>of</strong>fering several services. Check out the reagent repository comprising celllines, plasmids and other tools used in gene therapy research. There are archivingservices to meet FDA requirements. The NGVB will store clinical post-trial samples,final product reserves, and master cell banks as well as pharmacology/toxicologysamples. A pharm/tox database containing gene therapy tox studies is a valuableresource. We <strong>of</strong>fer assistance with insertional mutagenesis analysis through theSeqMap Bioinformatic Tool as well as LAM-PCR and other testing services. Go tohttps://www.ngvbcc.org or come and see us in Booth 512.Nature Publishing Group75 Varick Street, 9th floorNew York, NY 10013Telephone: (212) 726-9200Fax: (212) 696-9006E-mail: n.neysmith@us.nature.comWebsite: www.nature.comBooth Number: 206Nature Publishing Group brings leading scientific and medical research to yourdesk top. The NPG portfolio combines the continued excellence <strong>of</strong> Nature, itsassociated research and review journals, and over 45 leading academic andsociety journals in the life, physical and clinical sciences. Visit Booth Number 206for free sample copies.Nature Technology Corporation4701 Innovation Dr.Lincoln, NE 68521Telephone: (402) 472-6530Fax: (402) 472-6532E-mail: natx@natx.comWebsite: www.natx.comBooth Number: 400Nature Technology Corporation is a discovery-based bioengineering technologycompany, providing industry solutions for DNA-based gene therapeutics and DNAvaccines development, including: design, manufacturing, testing, and tech transfer<strong>of</strong> best-in-class vectors for in vivo gene expression.


80<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Exhibitor DescriptionsNexcelom Bioscience360 Merrimack StreetBldg 9Lawrence, MA 01843Telephone: (978) 327-5340Fax: (978) 327-5341E-mail: ddemers@nexcelom.comWebsite: www.nexcelom.comBooth Number: 414Nexcelom’s <strong>Cell</strong>ometer line <strong>of</strong> simple-to-use cell counters automate manual cellcounting procedures by obtaining accurate counts, viability, and cell sizes in lessthan 30 seconds & only 20uL <strong>of</strong> sample. Fluorescence detection capabilitiesenable fast & simple determination <strong>of</strong> GFP transfection rates, PI-viability, &direct counting <strong>of</strong> WBCs without lysing.NHLBI <strong>Gene</strong> <strong>Therapy</strong> Resource ProgramNational Heart, Lung, and Blood Institute, NIH2 Rockledge Center – 6701 Rockledge DriveBethesda, MD 20892-7940Telephone: (301) 435-0477Fax: (301) 480-7971E-mail: gtrpccc@s-3.comWebsite: www.gtrp.orgBooth Number: 311The NHLBI <strong>Gene</strong> <strong>Therapy</strong> Resource Program (GTRP) facilitates the translation<strong>of</strong> gene therapy research into clinical applications primarily in heart, lung, andblood diseases. Services provided by the program include preclinical and clinicalgradevector production, pharmacology/toxicology testing, immunology testing,regulatory support, and clinical trial funding assistance.NIH Office <strong>of</strong> Biotechnology Activities6705 Rockledge DriveSuite 750Bethesda, MD 20892Telephone: (301) 496-9838Fax: (301) 496-9839E-mail: oba@od.nih.govWebsite: http:\\oba.od.nih.govBooth Number: 508The NIH Office <strong>of</strong> Biotechnology Activities (OBA) promotes science, safety, andethics in biotechnology through advancement <strong>of</strong> knowledge, enhancement<strong>of</strong> public understanding, and development <strong>of</strong> sound public policies. OBAaccomplishes its mission through analysis, deliberation, and communication <strong>of</strong>scientific, medical, ethical, legal, and social issues.NIH-RAID6001 Executive Blvd, Room 2141Bethesda, MD 20892Telephone: (301) 594-4660Fax: (301) 402-1501E-mail: nih-raid@mail.nih.govWebsite: http://nihroadmap.nih.gov/raid/Booth Number: 305NIH-RAID makes available, at no cost, certain resources needed for thedevelopment <strong>of</strong> new therapeutic agents. Available services include: production,bulk supply, GMP manufacturing, formulation, development <strong>of</strong> an assay suitablefor pharmacokinetic testing, and animal toxicology. NIH-RAID is not a grantprogram; successful projects gain access to government contract resources.Omnia Biologics, Inc.12111 Parklawn DriveRockville, MD 20852Telephone: (301) 984-5928Fax: (301) 984-2515E-mail: scardak@omniabiologics.comWebsite: www.omniabiologics.comBooth Number: 212Omnia Biologics provides cost effective outsourcing <strong>of</strong> product developmentservices to the biotechnology, pharmaceutical, and academic biologic drugdevelopers. These services include process development, cGMP cell and virusbanking, comprehensive cGMP manufacturing services, BSL-3 containment,assay development, formulation and fill/finish. Omnia has experience with avariety <strong>of</strong> vaccine, gene therapy and protein therapeutic platforms.Penn Vector Core125 South 31 st Street, Suite 2000Philadelphia, PA 19104-3403Telephone: (215) 573-0633Fax: (215) 573-0300E-mail: vector@mail.med.upenn.eduWebsite: http://www.med.upenn.edu/gtp/vector_core.shtmlBooth Number: 309Penn Vector Core is a state-<strong>of</strong>-the-art vector production facility for investigatorsboth within and external to the University <strong>of</strong> Pennsylvania. The main objective <strong>of</strong>the Core is to make various types <strong>of</strong> high quality viral vectors for gene transfer/Vaccine studies at the preclinical and basic research level.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 81Exhibitor DescriptionsPhilips Research345 Scarborough RoadBriarcliff Manor, NY 10510Telephone: (914) 945-6188Fax: (914) 945-6580E-mail: ralf.seip@philips.comWebsite: www.research.philips.com/TIPSBooth Number: 501Philips Research has developed the <strong>Therapy</strong> Imaging Probe System (TIPS)device. TIPS is a turnkey solution for all pre-clinical research needs using focusedultrasound for non-invasive gene delivery/transfection and site-targeted drugdelivery using ultrasound and microbubble agents.Polyplus-transfectionBioparc Bd S. BrantIllkirch, Alsace67401FranceTelephone: +33390406180Fax: +33390406181E-mail: support@polyplus-transfection.comWebsite: www.polyplus-transfection.comBooth Number: 507Polyplus-transfection is a Biotech company dedicated to the R & D andmarketing <strong>of</strong> transfection reagents. Polyplus provides state-<strong>of</strong>-the-art qualityreagents for standard in vitro DNA transfection to human gene therapy, aswell as for protein production in synthetic medium, siRNA delivery and proteindelivery in living cells.Puresyn, Inc.87 Great Valley ParkwayMalvern, PA 19355Telephone: (610) 640-0800Fax: (610) 640-0808E-mail: cflynn@puresyn.comWebsite: www.puresyn.comBooth Number: 308Puresyn, Inc., is a Separations and Purification Technology Company thatdevelops, manufactures and markets state-<strong>of</strong>-the-art products and contractmanufacturing services for nucleic acids and other biological molecules utilizingour proprietary polymer technology, PolyFlo ® for production and purification <strong>of</strong>gene delivery products including plasmids and recombinant viruses at scales andcustomized specifications.ReGenX Biosciences750 17th St. NWSuite 1100Washington, DC 20006Telephone: (202) 785-7438Fax: (202) 785-7439E-mail: sales@regenxbio.comWebsite: www.regenxbio.comBooth Number: 407ReGenX is a research and development stage biopharmaceutical companydeveloping novel therapeutics and research tools based on adeno-associated viralvectors, or AAV vectors.Sartorius Stedim North America Inc.5 Orville DriveBohemia, NY 11716Telephone: (800) 368-7178Fax: (631) 254-2453E-mail: btcs@sartorius-stedim.comWebsite: www.sartorius-stedim.comBooth Number: 306Sartorius Stedim Biotech is a leading provider <strong>of</strong> equipment and servicesfor the development, quality assurance and production processes <strong>of</strong> thebiopharmaceutical industry as well as the public sector. With integrated solutionscovering fermentation, filtration, purification, fluid management, and labtechnologies, Sartorius Stedim Biotech focuses on single-use technologies andvalue-added services to meet requirements <strong>of</strong> the industries it serves.Seattle’s Convention and Visitors Bureau701 Pike Street, Suite 800Seattle, WA 98101Telephone: (206) 461-5800Fax: (206) 461-5855E-mail: visinfo@visitseattle.orgWebsite: www.visitseattle.orgBooth Number: 500Seattle is two cities in one. It’s a world-class metropolis set within, beautiful naturalsurroundings, <strong>of</strong>fering the best <strong>of</strong> urban lifestyle while embracing the outdoors.Many <strong>of</strong> the city’s top visitor attractions are within mere blocks <strong>of</strong> the WashingtonState Convention Center, including Pike Place Market. Seattle is metronatural.


82<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Exhibitor DescriptionsSt. Jude Children’s Research Hospital262 Danny Thomas PlaceMemphis, TN 38105Telephone: (901) 595-2750Fax: (901) 595-5376E-mail: fellowships@stjude.orgWebsite: www.stjude.orgBooth Number: 207St. Jude Children’s Research Hospital is a non-pr<strong>of</strong>it biomedical researchinstitution located in Memphis, TN, in the United States where 190 basic scienceand clinical researchers investigate the molecular basis <strong>of</strong> both normal cellularand diseased processes. Visit our booth to discuss and apply for PostdoctoralFellowship positions for PhD, MD, PharmD, and DVM students.Techulon, Inc.2200 Kraft DriveSuite 2475Blacksburg, VA 24060Telephone: (415) 602-0481Fax: (206) 202-0418E-mail: frank.akers@techulon.comWebsite: www.techulon.comBooth Number: 613Techulon develops and markets biodegradable transfection reagents that exhibithigh efficacy and very low cytotoxicity, especially in sensitive primary cells.Techulon welcomes inquiries from researchers and from companies interested indeveloping therapeutic applications.Thermo Scientific Genomics601 Genome WaySuite 2100Huntsville, AL 35806Telephone: (256) 704-4848Fax: (256) 704-4849E-mail: Info.account@therm<strong>of</strong>isher.comWebsite: www.thermo.com/genBooth Number: 213The Thermo Scientific genomics portfolio, which includes Dharmacon and OpenBiosystems RNAi and gene expression technologies and ABgene PCR products,provides novel tools to help our customers unlock the underlying molecularmechanisms <strong>of</strong> disease from cancer, to diabetes to viral infection and developnew therapies to fight it.TriLink BioTechnologies, Inc.9955 Mesa Rim RoadSan Diego, CA 92121Telephone: (858) 546-0004Fax: (858) 546-0020E-mail: info@trilinkbiotech.comWebsite: www.trilinkbiotech.comBooth Number: 314TriLink BioTechnologies manufactures custom oligonucleotides, modifiednucleoside triphosphates and CleanAmp PCR products. In addition, customchemistry, contract research services and ISO/QSR compliant cGMP productionfacilities are <strong>of</strong>fered. TriLink’s strong pr<strong>of</strong>iciency in nucleoside and oligonucleotidechemistry means high purity, modified compounds and expert technical support.For more information visit www.trilinkbiotech.com.Vandalia ResearchPO Box 2705Huntington, WV 25726-2705Telephone: (304) 529-0803Fax: (888) 550-8220E-mail: hughes@vandaliaresearch.comWebsite: www.vandaliaresearch.comBooth Number: 513VGXI, Inc.2700 Research Forest DriveSuite 180The Woodlands, TX 77381Telephone: (281) 296-7300E-mail: hhebel@vgxii.comWebsite: www.vgxii.comBooth Number: 607VGXI, Inc. has over 13 years experience manufacturing injectable-gradeDNA plasmids for gene therapy research. A leading contract manufacturingorganization (CMO), our fermentation services are designed to meet the uniqueneeds <strong>of</strong> gene therapy researchers at every stage <strong>of</strong> product development –from research to commercial supply. This comprehensive <strong>of</strong>fering lets researchersavoid the risks associated with switching suppliers just prior to clinical trials. Ourmission is to support biomedical researchers in their effort to rid the world <strong>of</strong>deadly and debilitating diseases. We do this by leveraging our experience andpatented technologies to maximize our clients’ chances <strong>of</strong> clinical success.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 83Exhibitor DescriptionsWaisman Clinical Biomanufacturing Facility1500 Highland AvenueMadison, WI 53705Telephone: (608) 263-8940E-mail: keach@waisman.wisc.eduWebsite: www.GMPbio.orgBooth Number: 214The Waisman Clinical Biomanufacturing Facility (WCBF) is a dedicated manufacturingfacility that provides cGMP manufacturing services for producing biotherapeuticsfor early-stage human clinical trials. Services that are provided by the WCBF rangefrom process development to clinical production and product fill and finish, all withfull quality system support.Wiley-Blackwell350 Main St.Malden, MA 02148Telephone: (800) 532-5954Fax: (781) 338-8552E-mail: jo.wright@wiley.comWebsite: www.wiley.comBooth Number: 413Wiley-Blackwell publishes quality journals in gene and cell therapy includingStem <strong>Cell</strong>s, The Journal <strong>of</strong> <strong>Gene</strong> Medicine, and Journal <strong>of</strong> Tissue Engineeringand Regenerative Medicine, and must-read books including Immunology <strong>of</strong><strong>Gene</strong> <strong>Therapy</strong>, Delivery Technologies for Biopharmaceuticals and Viral <strong>Therapy</strong> <strong>of</strong>Cancer. See our full collection at: www.wileyblackwell.com.


84<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Exhibitor Information<strong>Gene</strong>ral InformationThe Exhibit Hall is located in Exhibit Hall C <strong>of</strong> the Marriott Wardman Park Hotel.All participants are urged to allow adequate time in your daily schedule to visitthe exhibits. The companies, organizations and institutions exhibiting at theASGCT 13th Annual Meeting will be providing you with the latest informationon products and services directly related to your pr<strong>of</strong>essional needs, as well asrecruiting for open positions.Exhibit DatesExhibits will be open Wednesday, May 19 through Friday, May 21, 2010.Exhibitor RegistrationThe Exhibitor Registration Desk is located in the atrium, right outside Exhibit Hall C.Exhibitor Registration Desk HoursWednesday, May 19 9:00 am – 7:00 pmThursday, May 207:00 am – 5:00 pmFriday, May 217:00 am – 5:00 pmExhibitor Badge PolicyAdmission to the Exhibit Hall will be by name badge only. Security guards willmonitor exhibit hall entrances for proper I.D. Exhibit booth personnel must showan <strong>of</strong>ficial ASGCT exhibitor name badge in order to gain access to the exhibithall during installation, show, or dismantlement hours. Independent contractorpersonnel, hired by an exhibitor to install and dismantle their display, do notneed to show exhibitor badges, but should possess identification that they areemployed by the independent contractor. If independent contractors do notpossess proper identification, they should obtain a name badge at the on-siteexhibitor registration desk.Endorsement DisclaimerProducts and services displayed in the Exhibit Hall or advertised in this programoccur by contractual business arrangements between ASGCT and participatingcompanies and organizations. These arrangements do not constitute or imply anendorsement by ASGCT <strong>of</strong> these products and services.Exhibit InstallationWednesday, May 19Exhibit Hall HoursWednesday, May 19Thursday, May 20Thursday, May 20Friday, May 219:00 am – 5:00 pm7:00 pm – 9:00 pm9:15 am – 10:30 am6:15 pm – 8:15 pm6:30 pm – 8:30 pmDismantlementFriday, May 218:30 pm – 10:30 pmNote: All exhibit booths must be staffed during hall hours. Packing <strong>of</strong>equipment/literature and the dismantling <strong>of</strong> exhibits will not be permitted untilafter 8:30 pm on Friday, May 21, 2010.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 85Washington, DC Fun FactsIn Washington, DC, you’ll enjoy access to fascinating, FREE attractions andhistoric sights. Touch a moon rock, marvel at the Hope Diamond, view Dorothy’sRuby Red slippers or explore Native <strong>American</strong> culture at the SmithsonianInstitution’s fifteen Washington, DC area facilities. Discover treasures like theGutenberg Bible at the Library <strong>of</strong> Congress, the only da Vinci painting in NorthAmerica at the National Gallery <strong>of</strong> Art and historic documents like the Declaration<strong>of</strong> Independence at the National Archives. The National Zoo is also located withinwalking distance <strong>of</strong> the Marriott Wardman Park Hotel.Away from these celebrated federal sites, Washington, D.C. unwinds into afascinating network <strong>of</strong> neighborhoods where visitors discover trendy boutiques,hip bars and restaurants, plus art galleries, historic homes and lush parks andgardens. Shoppers love the store-lined streets <strong>of</strong> Georgetown, while jazz musicfans won’t want to miss a trip to U Street, where Duke Ellington played his firstnotes. The city’s international character shines through in its Adams Morganand Dupont Circle neighborhoods, two prime destinations for eclectic dining andnightlife and the historic center <strong>of</strong> the city’s embassy community.The arrival <strong>of</strong> several new eateries has made the nation’s capital a primedestination for dining out, with many <strong>of</strong> the city’s top tables located in thedowntown Penn Quarter neighborhood. DC is also earning new recognition as athriving performing arts town, with 65 pr<strong>of</strong>essional theatre companies based inthe metropolitan area presenting edgy world premieres and celebrated Broadwaymusicals throughout the year.It’s easy to get around town thanksto DC’s pedestrian-friendly streets andits safe, efficient public transportationsystem—including Metrorail and the newCirculator bus—it’s easy to get from yourhotel to Washington, DC’s attractions. Formore information, visit www.washington.org.Hotel InformationMarriott Wardman Park Hotel2660 Woodly Road NWWashington, DCPhone: (202) 328-2000The hotel has several places you can grab a bit to eat, which include:Harry’s Pub:This Washington DC restaurant features an Old English menu. Enjoysandwiches, soups, pizzas and microbrews. Open for lunch and dinner.Illy’sGrab a bite to go at this c<strong>of</strong>feehouse featuring specialty c<strong>of</strong>fees, teas andfresh baked goods.Stone’s Throw Restaurant & Bar:A steakhouse <strong>of</strong>fering steaks, chops and seafood dishes that is open forbreakfast, lunch and dinnerWoodley’s MarketThis gourmet deli serves breakfast items, snacks and gourmet takeoutsandwiches and meals.Or walk through the neighborhood and enjoy over 20 eatery options.


86<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryThursday, May 20 thThursday, May 20, 2010Oral Abstract Session 230AAV Vectors: Vector Trafficking2:00 PM - 4:00 PMRoom: Marriott Ballroom: Salons 2 & 31 2:00 PMEngineering AAV for Improved In Vitro Infectivity, In Vivo Tropismand Onset <strong>of</strong> ExpressionRu Xiao, Sadik H. Kassim, Lauren E. Mays, Deirdre McMenamin, Regina Munden, JamesM. Wilson, Luk H. Vandenberghe.2 2:15 PMTargeting Directed Evolution <strong>of</strong> AAV Capsids for Specific TherapeuticApplicationsSteven J. Gray, Bonita L. Blake, Matthew Hirsch, Hugh E. Criswell, Sarah Nicolson,Wuping Li, Thomas J. McCown, R. Jude Samulski.3 2:30 PMEnhanced Hepatic Transgene Expression Following IntravenousDelivery <strong>of</strong> Novel Stem <strong>Cell</strong>-Derived Recombinant AAV VectorsLaura J. Smith, Ariel Kauss, Kamehameha K. Wong, Saswati Chatterjee.4 2:45 PMAAV Capsid Sequence Significantly Influences Intracellular ViralTrafficking in Polarized Human Airway Epithelial <strong>Cell</strong>sNicholas W. Keiser, Ziying Yan, Diana Lei-Butters, John F. Engelhardt.5 3:00 PM<strong>Cell</strong> Entry Pathway Defines Efficiency and Specificity <strong>of</strong> rAAVTargeting VectorsSilke Uhrig, Jessica Sallach, David Gomez Almarza, Luca Perabo, Sabine Eming,Fernando Laguzzi Larcher, Michael Hallek, Hildegard Büning.6 3:15 PMNovel Cardiac-Tropic AAV Vectors by DNA Shuffling and In VivoScreeningZhenhua Yuan, Lin Yang, Chunping Qiao, Juan Li, Xiao Xiao.7 3:30 PMIntravenously Administered rAAV9 Vector Efficiently TransducesTissues In Vivo Via Caveolin-1-Independent Transport across theCapillary Endothelial <strong>Cell</strong> BarrierKei Adachi, Nicole M. Kotchey, Maliha Zahid, Hiroyuki Nakai.8 3:45 PMDirected Evolution <strong>of</strong> Adeno-Associated Virus Variants That EvadeHuman Neutralizing AntibodiesDaniel Stone, James T. Koerber, Federico Mingozzi, Gregory M. Podsak<strong>of</strong>f, KatherineHigh, David V. Schaffer.Oral Abstract Session 231DNA Vectorology & <strong>Gene</strong> Targeting2:00 PM - 4:00 PMRoom: Maryland Suite9 2:00 PMA Simplified Approach for Robust Minicircle DNA Vector ProductionUsing a New <strong>Gene</strong>tically-Modified E. Coli Producer StrainZYCY10P3S2TZhi-Ying Chen, Cheng-Yi He, Mark A. Kay.10 2:15 PMTargeted Transgene Integration in Epidermal Stem <strong>Cell</strong>s by Zinc FingerNucleases and Integrase-Defective Lentiviral VectorsGiulietta Maruggi, Angelo Lombardo, Francesca Miselli, Pietro Genovese, Michael C.Holmes, Philip D. Gregory, Alessandra Recchia, Fulvio Mavilio, Luigi Naldini.11 2:30 PMZinc Finger Nucleases Drive Efficient <strong>Gene</strong> Disruption and Site-Specific <strong>Gene</strong> Addition in Human HSCNathalia G. Holt, Kevin G. Haworth, Kathleen C. Burke, Jianbin Wang, Kenneth Kim,Ge<strong>of</strong>frey Friedman, Philip D. Gregory, Michael C. Holmes, Paula M. Cannon.12 2:45 PMManipulating piggyBacTransposon Chromosomal Integration Site-Selection in Human <strong>Cell</strong>sClaudia Kettlun, Daniel Galvan, Aparna Kaja, Edward Rebar, Matthew H. Wilson.13 3:00 PMOptimized Recovery <strong>of</strong> Integration Sites from <strong>Gene</strong>-Modified <strong>Cell</strong>sUsing Mu Transposition In VitroTroy L. Brady, Nirav Malani, Gary P. Wang, Charles C. Berry, Philippe Leboulch, SalimaHacein-Bey-Abina, Marina Cavazzana-Calvo, Harri Savilahti, Frederic D. Bushman.14 3:15 PMAn In Vitro-Identified High Affinity Nucleosome Positioning Signal IsCapable <strong>of</strong> Transiently Positioning a Nucleosome In VivoLia E. Gracey, Zhi-Ying Chen, Jay M. Maniar, Anton Valouev, Arend Sidow, Andrew Z.Fire, Mark A. Kay.15 3:30 PMHyperactive piggyBac-Mediated <strong>Gene</strong> Transfer in Somatic <strong>Cell</strong>s InVivoJoseph E. Doherty, Liqin Zhou, Kosuke Yusa, Allan Bradley, Nancy L. Craig, Matthew H.Wilson.16 3:45 PMHoming Endonucleases for <strong>Gene</strong> <strong>Therapy</strong>Adi Barzel, Hilla Giladi, Einat Sachar, Ludmila Rivkin, Eyal Privman, Mor Paldor, TalPupko, Toni Cathomen, Eithan Galun, Martin Kupiec.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 87Abstract DirectoryThursday, May 20, 2010Oral Abstract Session 232Neurologic & Ophthalmic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>2:00 PM - 4:00 PMRoom: Wilson ABC17 2:00 PMAAV-Mediated <strong>Gene</strong> <strong>Therapy</strong> Restores Visual Function and Behaviorto a Mouse Model <strong>of</strong> Leber Congential Amaurosis-1 (LCA1)Shannon E. Boye, Sanford L. Boye, Jijing Pang, Renee C. Ryals, Andy W. Neeley, DrewEverhart, Yumiko Umino, Joseph C. Besharse, Robert Barlow, William W. Hauswirth.18 2:15 PMAdeno-Associated Virus Vector Expressing an Amyloid beta 1-42Intrabody Rescues White Matter Pathology during Early Alzheimer’sDiseaseMaya K. Desai, Michael A. Mastrangelo, Deborah A. Ryan, Kelly L. Sudol, Wade C.Narrow, William J. Bowers.19 2:30 PMBrain <strong>Gene</strong> <strong>Therapy</strong> for Metachromatic Leucodystrophy: TowardsClinical TrialCaroline Sevin, Dolan Sondhi, Françoise Piguet, Marie-Anne Colle, Sylvie Raoul, ThomasRoujeau, Monique Piraud, Ornella Ahouansou, Celine Bouquet, Marie-Thérèse Vanier,Philippe Moullier, Yan Cherel, Neil R. Hackett, Michel Zerah, Patrick Aubourg, Ronald G.Crystal, Nathalie Cartier.20 2:45 PMNeonatal Intracerebroventricular Infusion <strong>of</strong> AAV2/8 Vector Leadsto High Levels <strong>of</strong> Human a-L-Iduronidase Expression in the Brainand Complete Neurophenotypic Correction in a Murine Model <strong>of</strong>Mucopolysaccharidosis Type IDaniel A. Wolf, Andrew W. Lenander, Zhenhong Nan, Lalitha R. Belur, Debra Swanson,Chester B. Whitley, Walter C. Low, R. Scott McIvor.21 3:00 PMCircuit-Specific Expression <strong>of</strong> Channelrhodopsin Restores VisualFunction in Blind rd1, rd16, and rho -/- MiceAlan Horsager, Jian Wen Liu, Edward S. Boyden, A. Cyrus Arman, Kimberly A. Silka,Mehdi Doroudchi, Shannon E. Boye, Benjamin C. Matteo, Alapakkam P. Sampath,William W. Hauswirth.22 3:15 PMCombination <strong>Therapy</strong> for the Treatment <strong>of</strong> Infantile Neuronal CeroidLip<strong>of</strong>uscinosis (INCL)Shannon L. Macauley-Rambach, Marie S. Roberts, Mark S. Sands.23 3:30 PMrAAV Delivery <strong>of</strong> a-Synuclein-Specific Single Chain AntibodiesAttenuates Dopamine Loss in a Mouse Model <strong>of</strong> Parkinson’s DiseaseKathleen Maguire-Zeuss, William J. Bowers, Wade Narrow, Eric Yehling, Howard J.Feder<strong>of</strong>f.24 3:45 PMPatterns <strong>of</strong> Motor Function Recovery in a Murine Model <strong>of</strong> SevereMenkes Disease Rescued by Brain-Directed AAV5 <strong>Gene</strong> <strong>Therapy</strong> PlusCopperAnthony Donsante, Stephen G. Kaler.Oral Abstract Session 233Cancer – Immunotherapy T <strong>Cell</strong> Studies2:00 PM - 4:00 PMRoom: Thurgood Marshall W25 2:00 PMAdoptive Transfer <strong>of</strong> Peripheral Blood Lymphocytes <strong>Gene</strong>ticallyModified To Express a T <strong>Cell</strong> Receptor Recognizing CarcinoembryonicAntigen into Patients with Metastatic Colorectal Cancer InducedInflammatory Colitis without Mediating Anti-Tumor EffectsMaria R. Parkhurst, James C. Yang, Russell C. Langan, Steven A. Feldman, Mark E.Dudley, Paul F. Robbins, Steven A. Rosenberg.26 2:15 PMAdenovirus Mediated Expression <strong>of</strong> Flt3L and HSV1-TK within theBrain Tumor Microenvironment Elicits Higher Therapeutic EfficacyThan Dendritic <strong>Cell</strong>s’ VaccinationYohei Mineharu, Gwendalyn D. King, Marianela Candolfi, Weidong Xiong, Hikmat Assi,Kader Yagiz, Kurt M. Kroeger, Patricia Lin, B. Serguei, Pedro R. Lowenstein, Maria G.Castro.27 2:30 PMPharmacokinetics <strong>of</strong> Anti-PSMA <strong>Gene</strong>-Modified T <strong>Cell</strong>s Infused afterNon-Myeloablative Conditioning in Prostate CancerAnthony J. Bais, Qiangzhong Ma, Hannah Boss, Esther L. Beaudoin, Richard P. Junghans.28 2:45 PMBinary Control <strong>of</strong> Tumor-Specific Cytotoxic T Lymphocytes byTransgenic IL7 and IL7r ExpressionJuan F. Vera, Ceidy Sanchez, Sujatha Muralidharan, Helen E. Heslop, Cliona M. Rooney,Gianpietro Dotti, Malcolm K. Brenner.29 3:00 PMMulti-Tumor-Antigen-Specific Cytotoxic T Lymphocytes for CancerImmunotherapyUlrike Gerdemann, Juan F. Vera, Anne S. Christin, Russell C. Cruz, Helen E. Heslop,Malcolm K. Brenner, Catherine M. Bollard, Cliona M. Rooney, Ann M. Leen.30 3:15 PMSimultaneous Comparison <strong>of</strong> T <strong>Cell</strong>s Expressing 1 st or 2 nd <strong>Gene</strong>rationCARs in Human Subjects with B-<strong>Cell</strong> Malignancies: Contribution <strong>of</strong>Costimulatory EndodomainsB. Savoldo, C. Ramos, C. Bollard, E. Liu, M. Mims, M. Keating, A. Gee, Z. Mei, C.Rooney, H. Heslop, M. Brenner, G. Dotti.Thursday, May 20 th


88<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryThursday, May 20 thThursday, May 20, 2010Oral Abstract Session 233 — continuedCancer – Immunotherapy T <strong>Cell</strong> Studies2:00 PM - 4:00 PMRoom: Thurgood Marshall W31 3:30 PMProlonged Regression <strong>of</strong> Lymphoma and Elimination <strong>of</strong> CD19 + <strong>Cell</strong>sin a Patient That Received Anti-CD19 Chimeric Antigen Receptor-Transduced T <strong>Cell</strong>sJ. N. Kochenderfer, M. E. Dudley, R. A. Morgan, I. Maric, M. Raffeld, M. Stetler-Stevenson, J. E. Janik, W. H. Wilson, C. M. Yuan, S. A. Rosenberg.32 3:45 PMMarked Regression <strong>of</strong> Adenopathy Following Infusion <strong>of</strong> AutologousT <strong>Cell</strong>s <strong>Gene</strong>tically Targeted to the CD19 Antigen in a Patient withBulky CLLRenier J. Brentjens, Isabelle Riviere, Mark Frattini, Xiuyan Wang, Clare Taylor, MalgorzataOlszewska, Oriana Borquez-Ojeda, Shirley Bartido, Jolanta Stefanski, Raymond Yeh,Michel Sadelain.37 3:00 PMInsect <strong>Cell</strong> Carriers for Systemic Delivery <strong>of</strong> Oncolytic VirusesDominic G. Roy, Anthony T. Power, Fabrice Le Boeuf, John C. Bell.38 3:15 PMClinical Translational Development <strong>of</strong> rVSV(MD51)-M3:Biodistribution and Pharm/Tox Studies in Rats and MonkeysMarcia Meseck, Nelson Humala, Boxun Xie, Swan Thung, Susan Morgello, GreggGoldschlager, Savio Woo.39 3:30 PMA High-Throughput Pharmaco-Viral Approach Identifies OncolyticNovel Virus SensitizersJean-Simon Diallo, Fabrice Le Boeuf, Frances Lai, Julie Cox, Markus Vaha-Koskela,Hesham Abdelbary, Heather MacTavish, Katherine Waite, Theresa Falls, Jenny Wang,Ryan Brown, Jan E. Blanchard, Eric D. Brown, David H. Kirn, John Hiscott, Harry Atkins,Brian D. Lichty, John C. Bell.40 3:45 PMIdentification <strong>of</strong> Adenovirus Modulator <strong>Gene</strong>s in Human Cancer <strong>Cell</strong>sby Whole Genome RNAi Library ScreeningChristie Vermeulen, Nikki Tol, Ida van der Meulen-Muileman, Winald R. Gerritsen, VictorW. van Beusechem.Oral Abstract Session 234Cancer-Oncolytic Viruses I2:00 PM - 4:00 PMRoom: Marriott Ballroom Salon 133 2:00 PMDemonstration <strong>of</strong> Delivery and Antitumoral Activity <strong>of</strong> JX-594, aTargeted Multi-Mechanistic Oncolytic Poxvirus, Following a SingleIntravenous Infusion in Patients with Refractory Metastatic CancersJames Burke, Joe Stephenson, Laura Chow, Derek Jonker, Andrew Haas, Jorge Nieva,Kara Sabourin, Adina Pelusio, Caroline Breitbach, Taeho Hwang, Anne Moon, KelleyParato, Manijeh Daneshmand, John C. Bell, David H. Kirn.34 2:15 PMHighly Attenuated Vaccinia Virus with microRNA-Regulated Oncolysisfor Cancer VirotherapyMina Hikichi, Minoru Kidokoro, Hisatoshi Shida, Hideaki Tahara, Takafumi Nakamura.35 2:30 PMA Phase 1 Trial <strong>of</strong> Intravesical (IVE) CG0070 in Patients withSuperficial Bladder Cancer after BCG FailureJames M. Burke, Don Lamm, James Mckiernan, John Nemunaitis, Joe Stephenson, MaxMeng, James Arsenau, Junko Aimi, Daniel Maslyar.36 2:45 PMA Phase 1 Trial <strong>of</strong> the Novel Infectivity-Enhanced BicistronicAdenovirus Ad5.SSTR/TK.RGD in Recurrent Gynecologic CancerKenneth H. Kim, Kellie Schneider, Minghui Wang, Janis O’Malley, Wen Wan, MeredithA. Preuss, Raymond D. Harris, Rosemarie Aurigemma, <strong>Gene</strong> P. Siegal, Kurt Zinn, DavidT. Curiel, Ronald D. Alvarez.Oral Abstract Session 235Immunologic & Host Responses in <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>2:00 PM - 4:00 PMRoom: Virginia Suite41 2:00 PMRepeated Autologous Infusions <strong>of</strong> <strong>Cell</strong>s Modified with VSV-G-Pseudotyped Lentiviral Vector VRX496 Can Induce Anti-VSV-GHumoral Response in Humans without Clinical Consequence or Effecton Modified <strong>Cell</strong>s Persistence: Findings from Lexgenleucel-T Phase 2Clinical TrialFranck Lemiale, Lindsay Shovlin, Kris Andre, Gerard McGarrity, Tessio Rebello, LaurentHumeau.42 2:15 PMIntensive Pharmacological Immunosuppression Allows for RepetitiveLiver <strong>Gene</strong> Transfer with Recombinant Adenovirus in NonhumanPrimatesAntonio Fontanellas, Sandra Hervás-Stubbs, Itsaso Mauleón, Juan Dubrot, UxuaMancheño, María Collantes, Ana Sampedro, Carmen Unzu, Carlos Alfaro, Asis Palazón,Rafael Enríquez de Salamanca, Cristian Smerdou, Alberto Benito, Jesús Prieto, IvánPeñuelas, Ignacio Melero.43 2:30 PMInduction <strong>of</strong> Regulatory T <strong>Cell</strong>s as an Approach To Modulate CTLResponses Against AAV Capsid EpitopesDaniel J. Hui, Gary Pien, Etiena Basner-Tschakarjan, Federico Mingozzi, Jonathan D.Finn, Shangzhen Zhou, William Martin, Anne S. De Groot, Katherine A. High.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 89Abstract DirectoryThursday, May 20, 2010Oral Abstract Session 235 — continuedImmunologic & Host Responses in <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>2:00 PM - 4:00 PMRoom: Virginia Suite44 2:45 PMLong-Term Phenotypic Correction <strong>of</strong> Hemophilia A after NeonatalAdministration <strong>of</strong> AAVChuhong Hu, Fides D. Lay, Joseph Springer, Ronald W. Busuttil, Gerald S. Lipshutz.45 3:00 PMImpact <strong>of</strong> Pre-Existing Neutralizing Antibodies on AAV8-Mediated,Liver-Directed <strong>Gene</strong> Transfer and Vector Biodistribution in NonhumanPrimatesLili Wang, Roberto Calcedo, Peter L. Bell, Martin W. Lock, Rebecca L. Grant, James M.Wilson.46 3:15 PMAutologous Dendritic <strong>Cell</strong> <strong>Therapy</strong> for Tolerance InductionGautam Sule, Masataka Suzuki, Racel Cela, John Rodgers, Brendan Lee.47 3:30 PMDifferential Transgene Expression in Dendritic <strong>Cell</strong>s with UniqueRegulatory Cassettes Impacts Efficacy in Pompe DiseaseBaodong Sun, Ping Zhang, Xiao Yi Yang, Timothy C. Clay, Dwight D. Koeberl.48 3:45 PMImpact <strong>of</strong> the Host Environment on the Control <strong>of</strong> <strong>Gene</strong> Transfer-Induced Immune Responses with miRNA142.3p-Regulated VectorsFlorence Boisgérault, David-Alexandre Gross, Maxime Ferrand, Sylvie Darocha, JérômePoupiot, Marc Bartoli, Isabelle Richard, Anne Galy.Oral Abstract Session 236Oligonucleotide & RNAi Therapeutics I2:00 PM - 4:00 PMRoom: Delaware Suite49 2:00 PMSystemic Delivery <strong>of</strong> Antisense Morpholino Corrects RNA Mis-Splicing and Reduces Myotonia in a Transgenic Mouse Model <strong>of</strong>Myotonic Dystrophy Type 1Thurman M. Wheeler, Yong-Fu Li, Krzyszt<strong>of</strong> Sobczak, Paul A. Morcos, Charles A.Thornton.50 2:15 PMTriplex-Induced Correction <strong>of</strong> IDUA <strong>Gene</strong> Mutations as a Novel HurlerSyndrome <strong>Therapy</strong>Jake del Campo, Karla D’Agostino, Ranjit S. Bindra, Peter M. Glazer.51 2:30 PMUsing rAAV Delivered miRNAs To Knockdown Misfolded HumanAlpha 1 Antitrypsin in a Transgenic Mouse ModelChristian Mueller, Qiushi Tang, Bryan O’Sullivan Murphy, Terence R. Flotte.52 2:45 PMConstitutive Long-Term Expression <strong>of</strong> shRNA in Liver Induces <strong>Cell</strong>ularStressMiwon Ahn, Scott R. Witting, Rafaela Ruiz, Romil Saxena, Nuria Morral.53 3:00 PMIn Vivo AAV Delivered Allele Specific shRNA for the Knockdown <strong>of</strong>Alpha-1 AntitrypsinChristian Mueller, Bryan O’Sullivan-Murphy, Qiushi Tang, Terence R. Flotte.54 3:15 PMExpression Determinants <strong>of</strong> Mammalian Argonaute Proteins inMediating <strong>Gene</strong> SilencingPaul N. Valdmanis, Shuo Gu, Praveen Sethupathy, Mark A. Kay.55 3:30 PMAntisense Oligonucleotide-Mediated <strong>Therapy</strong> Increases SMN Levelsin the CNS and Prolongs Survival in a Severe Mouse Model <strong>of</strong> SpinalMuscular AtrophyMarco A. Passini, Jie Bu, Amy M. Richards, Lisa M. Stanek, S. P. Sardi, Yimin Hua, <strong>Gene</strong>Hung, Lamya S. Shihabuddin, C. F. Bennett, Adrian R. Krainer, Seng H. Cheng.56 3:45 PMMultiple Exon-Skipping Using <strong>Cell</strong>-Penetrating Morpholinos forDystrophic DogsToshifumi Yokota, Takashi Saito, Nobuyuki Urasawa, Tetsuya Nagata, Akinori Nakamura,Ryszard Kole, Peter Sazani, Terence Partridge, Shin’ichi Takeda, Eric H<strong>of</strong>fman.Poster Session I6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-SouthAAV Vectors I57Enveloped AAV Vectors for <strong>Gene</strong> Transfer ApplicationsCasey A. Maguire, Lucia Mincheva-Nilsson, Vladimir Baranov, Bakhos A. Tannous, MiguelSena-Esteves, Xandra O. Breakefield, Johan Skog.58Chimpanzee-Derived Novel Natural Variants <strong>of</strong> AAV9: VectorDevelopment and Interrogation <strong>of</strong> Correlations between CapsidStructure and Vector BiologyLi Zhong, Jun Xie, Qing Xie, Kim Van Vliet, Qiang Wang, Mengxin Li, Qin Su, Ran He,Jason Goetzmann, Mavis Mckenna, Terry Flotte, Guangping Gao.59<strong>Gene</strong> Delivery to the Pig Cone-Enriched Retina Highlights Species-Specific Differences in AAV-Mediated Photoreceptor TransductionClaudio Mussolino, Francesco Viola, Michele Della Corte, Settimio Rossi, Umberto DiVicino, Simona Neglia, Monica Doria, Francesco Testa, Roberto Giovannoni, ManuelaCrasta, Massimo Giunti, Edoardo Villani, Marialuisa Lavitrano, Maria Laura Bacci,Roberto Ratiglia, Francesca Simonelli, Alberto Auricchio, Enrico M. Surace.Thursday, May 20 th


90<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryThursday, May 20 thThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-SouthAAV Vectors I60Enhancing AAV2 Transduction by Anti-Microtubule Drugs throughRhoA GTPase ActivationPing-Jie Xiao, Richard J. Samulski.61Tyrosine Mutations in the Capsid <strong>of</strong> AAV Vectors Alters TransductionProperties in the RetinaHilda Petrs-Silva, Astra Dinculescu, Seok H. Min, Li Zhong, Sergei Zolotukhin, ArunSrivastava, Alfred Lewin, William W. Hauswirth.62Riding Along with AAV: Mechanistic Studies on Vesicular TraffickingPathways <strong>of</strong> AAV SerotypesShen Shen, Xin Ming, Aravind Asokan, Rudy L. Juliano.63Effects <strong>of</strong> DNA Methylation on AAV Integration and TransgeneExpressionDiptiman Chanda, Jonathan A. Hensel, Jerome T. Higgs, Niroop Kaza, Rajat Grover,Selvarangan Ponnazhagan.64Efficient Transduction <strong>of</strong> Mouse Liver and Other Organs FollowingIntravenous Administration <strong>of</strong> a Recombinant Porcine AAVAlexander J. Bello, Allan R. Chand, Monica Doria, Kaylie Tran, Mariacarmela Allocca,Alberto Auricchio, Gary Kobinger.65Rational Design Meets Directed Evolution for Tailoring the AAVCapsidLuk H. Vandenberghe, Ru Xiao, Christie L. Bell, Kim Van Vliet, Jing Lu, Mavis Agbandje-McKenna, James M. Wilson.66AAV5 Transduction and Transgene Expression and Bioactivity inFibroblast-Like Synoviocytes from Rheumatoid Arthritis PatientsCaroline J. Aalbers, Scott A. Loiler, Federico Mingozzi, Paul-Peter Tak, Margriet J.Vervoordeldonk.67Pre-Injection <strong>of</strong> Polymers Can Increase the In Vivo TransductionEfficiency <strong>of</strong> Adeno-Associated Virus Type 2Gilles Moulay, Sylvie Boutin, Carole Masurier, Daniel Scherman, Antoine Kichler.68Therapeutic Expression <strong>of</strong> FIX at Reduced Doses Using AAV-ProteinPhosphotase-5 Helper VirusBrandon K. Sack, Jayandharan R. Giridhara, Zhong Li, Herzog W. Roland, SrivastavaArun.69A Scalable Process for Purification <strong>of</strong> Recombinant Adeno-AssociatedVirus Serotype 9Jingmin Zhou, Xiao Yang, Linda Couto, Katherine A. High, Guang Qu.70Adeno- Associated Virus Type 2 Rep Proteins Mediate PreferentialIntegration <strong>of</strong> a Lentiviral VectorVictor J. McAlister, Tshaka Cunningham, Anthony T. Craig, Roland A. Owens.71High Throughput Assay for Quantifying In Vitro AAV VectorTransduction Efficiency in ARPE19 <strong>Cell</strong>sRenee C. Ryals, Sanford L. Boye, Shannon Boye, Andrew Neely, William W. Hauswirth.72Optimization <strong>of</strong> Parameters for AAV Production in Insect <strong>Cell</strong>s andConditions for Baculovirus StorageHaifeng Chen.Adenovirus and Other DNA Virus Vectors I73Radiation-Induced Liver Fibrosis Is Mitigated by <strong>Gene</strong> <strong>Therapy</strong>Against Transforming Growth Factor-b Signaling in the RatDu Shi-Suo, Zeng Zhao-Chong, Zhou Jian, Tan Yun-shan, Zhang Zheng-Yu, Zeng Hai-Ying,Liu Zhong-shan.74Protection <strong>of</strong> Salivary Glands from Irradiation Damage by hMnSOD<strong>Gene</strong> TransferAna P. Cotrim, Anne M. Rowzee, A. Sowers, James B. Mitchell, Bruce J. Baum.75Intrathecal Injection <strong>of</strong> HDAd Vectors Results in Long-Term TransgeneExpression in Neuroepithelial <strong>Cell</strong>s and NeuronsPasquale Piccolo, Scott Dindot, Nathan Grove, Donna Palmer, Nicola Brunetti-Pierri.76Virion-Associated Co-Factor HMGB1 Facilitates Transposition fromthe HSV/Sleeping Beauty Amplicon Vector PlatformSuresh de Silva, Louis T. Lotta, Clark A. Burris, William J. Bowers.77hKGF <strong>Gene</strong> Transfer for the Prevention <strong>of</strong> Radiation-Induced SalivaryHyp<strong>of</strong>unctionChangyu Zheng, Ana P. Cotrim, Anne M. Rowzee, William D. Swaim, Anastasia Sowers,James B. Mitchell, Bruce J. Baum.78<strong>Gene</strong>ration <strong>of</strong> Replication-Defective Adenovirus Vector ExpressingHCV Structural <strong>Gene</strong> CE1E2 and Comparative Immunogenicity in Micewith Different Delivery RoutesJie Guan, Yao Deng, Xiao Yin, Wenjie Tan, Xiaobing Wu.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 91Abstract DirectoryThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-SouthAdenovirus and Other DNA Virus Vectors I79Interaction between Adenoviral Vector-Transduced Dendritic <strong>Cell</strong>-Based Cancer Vaccine and T Regulatory or T Helper 17 <strong>Cell</strong>sAdele Y. Wang, Sarah Q. Crome, Kristy M. Jenkins, Jeffrey A. Medin, Jonathan L.Bramson, Megan K. Levings.80Replicating Adenoviruses That Harbors the ADP Protein and theSodium Iodide Symporter Protein (NIS) under the Control <strong>of</strong> theMajor Late Adenovirus PromoterMiguel A. Trujillo, Julia Davydova, Michael J. Oneal, Samantha McDonough, MasatoYamamoto, John C. Morris.81FX Domain Fused to Single-Chain Antibodies Improve Oncolytic Effect<strong>of</strong> Ad5 In Vitro and In VivoChristopher Y. Chen, Michael A. Barry.82Sustained Inhibition <strong>of</strong> Hepatitis B Virus Replication In Vivo UsingHelper-Dependent Adenovirus VectorsCarol Crowther, Betty Mowa, Felix Salazar, Patricia Marion, Abdullah Ely, PatrickArbuthnot.83Adenovirus Serotype 5/35 Is Excellent for <strong>Gene</strong>tic Engineering <strong>of</strong>Tumor-Directed T <strong>Cell</strong>sHannah Karlsson, Camilla Lindqvist, Bengt Simonsson, Ulla Olsson-Strömberg, AngelicaS. I. Loskog.84Bipartite Adenoviral Vector Encoding hHGF and hXIAP for ImprovedHuman Islet TransplantationHao Wu, A-Rum Yoon, Chae-Ok Yun, Ram I. Mahato.85Vector Prime-Protein Boost Vaccine for Patients with <strong>Therapy</strong>Induced LymphopeniaTae Hee Han, Yucheng Tang, Yeon Hee Park, Pingchuan Li, Hakan Akbulut, Line Petersen,Jonathan Maynard, Albert Deisseroth.86The Effect <strong>of</strong> Surface Modification <strong>of</strong> Adenovirus with an Arginine-Grafted Bioreducible Polymer on Transduction Efficiency andImmunogenicity in Cancer <strong>Gene</strong> <strong>Therapy</strong>Pyung-Hwan Kim, Tae-il Kim, James W. Yockman, Sung Wan Kim, Chae-Ok Yun.Targeting <strong>Gene</strong> Modification and Integration87Safe and Efficient Delivery <strong>of</strong> Meganucleases for Lentivirus-Mediated<strong>Gene</strong> TargetingAraksya Izmiryan, Alix Bourdel, Arnaud Jollet, Frédéric Pâques, Olivier Danos.88Site-Specific Genome Modification <strong>of</strong> Human Primary T LymphocytesTo Improve the Safety and Efficacy <strong>of</strong> Adoptive ImmunotherapyPietro Genovese, Elena Provasi, Angelo Lombardo, Zulma Magnani, Andreas Reik, Pei-QiLiu, Oscar Muniz Pello, Jurgen Kuball, Philip D. Gregory, Michael C. Holmes, Philip D.Greenberg, Chiara Bonini, Luigi Naldini.89Single-Strand Nicks Induce Homologous Recombination with LessToxicity Than Double-Strand Breaks Using an AAV TemplateMichael J. Metzger, Audrey McConnell-Smith, Barry L. Stoddard, A. Dusty Miller.90Targeted <strong>Gene</strong> Insertion in Human Chromosomes Using a HomingEndonucleaseByoung Y. Ryu, Michael T. Certo, Mikhail Garibov, Andrew M. Scharenberg, David J.Rawlings.91<strong>Cell</strong> Synchronization by Serum Shock Facilitates rAAV-Mediated <strong>Gene</strong>TargetingYonglun Luo, Emil K<strong>of</strong>od-Olsen, Jenny Blechingberg, Rikke Christensen, Nicklas HeineStaunstrup, Lars Bolund, Charlotte Brandt Sørensen.92Critical Amino Acid Residues within the PhiC31 Integrase DNABinding Domain Affect Recombination Activities in Mammalian <strong>Cell</strong>sRaphael Liesner, Wenli Zhang, Nadja Noske, Anja Ehrhardt.93Chromatin Structure <strong>of</strong> Two Genomic Sites for Targeted TransgeneIntegration in Induced Pluripotent Stem <strong>Cell</strong>sRuan van Rensburg, Oleg Denisenko, Karol Bomsztyk, Andre Lieber.94Targeted Plasmid Integration into the PiggyBac Transposon by anEngineered Zinc Finger RecombinaseCharles A. Gersbach, Russell M. Gordley, Thom Gaj, Andrew C. Mercer, Carlos F. Barbas,III.95Development <strong>of</strong> Autonomously Working Zinc-Finger Nuclease Pairsfor Targeted Genome EngineeringCem Söllü, Kaweh Montazami-Astaneh, Morgan L. Maeder, Stacey Thibodeau-Beganny,J. Keith Joung, Toni Cathomen.96Adeno-Associated Virus-2 (AVV2) Integrase Mediates SpecificIntegration to Locus AAVS1 on Chromosome 19q13.3 in aHematopoietic <strong>Cell</strong> LineAshley E. Dunfee, Joshua R. Clevenger, Cynthia E. Dunbar, Andre Larochelle.Thursday, May 20 th


92<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryThursday, May 20 thThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-SouthTargeting <strong>Gene</strong> Modification and Integration97<strong>Gene</strong> Correction in Mouse LiverHiroyuki Kamiya, Masayuki Uchiyama, Jingshu Piao, Yoshimichi Nakatsu, TeruhisaTsuzuki, Hideyoshi Harashima.98A Transient Cold Shock Enhances Zinc-Finger Nuclease-Mediated <strong>Gene</strong>DisruptionYannick Doyon, Vivian M. Choi, Danny Xia, Thuy D. Vo, Michael C. Holmes, Philip D.Gregory.99Single-Polarity rAAV May Enhance Hepatic <strong>Gene</strong> Targeting In VivoNicole K. Paulk, Markus Grompe.Physical Methods <strong>of</strong> Delivery100Over-Expression <strong>of</strong> Adiponectin and Adiponectin Receptor 2 <strong>Gene</strong> bythe Method <strong>of</strong> Hydrodynamic <strong>Gene</strong> Delivery Prevents Weight Gainand Development <strong>of</strong> Insulin Resistance in AKR/J MiceYongjie Ma, Dexi Liu.101Duration <strong>of</strong> Expression after <strong>Gene</strong> Electrotransfer to Skin Depends onthe <strong>Gene</strong> <strong>of</strong> InterestAnita Gothelf, Jens Eriksen, Pernille Hojman, Julie Gehl.102Intravenous Delivery <strong>of</strong> pDNA and siRNA into Muscle by Ultrasound-Mediated Bubble LiposomesYoichi Negishi, Yoko E. Takahashi, Yuko Ishii, Ryo Suzuki, Kazuo Maruyama, YukihikoAramaki.103Prototype Development and Pre-Clinical Immunogenicity Analysis <strong>of</strong>a Novel Minimally Invasive Electroporation DeviceKate E. Broderick, Jay McCoy, Xuefei Shen, Feng Lin, Amir Khan, Matthew Morrow,Stephen Kemmerrer, David B. Weiner, Niranjan Y. Sardesai.104Ultrasound-Assisted Non-Viral <strong>Gene</strong> Transfer to the Salivary GlandsMichael J. Passineau, Lee Zourelias, Laurie Machen, Paul C. Edwards, Raymond L. Benza.105Development <strong>of</strong> Electroporation Formulation for <strong>Cell</strong> <strong>Therapy</strong>Mike Flanagan, Gang Yu, Jeffry Gimble, Shulin Li.106Ca 2+ Electrotransfer; a Quick Stop Procedure Against TransgenicExpressionPernille Hojman, Iben Spanggaard, Julie Gehl, Hanne Gissel.107In Vivo <strong>Gene</strong> Delivery to the Skin by Electroporation Using a MultipleElectrode ArraySiqi Guo, Amy Donate, Gaurav Basu, Kathryn Lundberg, Richard Heller.108Hydrodynamic <strong>Gene</strong> Delivery Efficiency Is Reduced in Fibrotic Liver inRatsTian Zhou, Dexi Liu.109Efficiency <strong>of</strong> Electroporation Delivery <strong>of</strong> Peptide Nucleic Acid (PNA)Depends on PNA Charge and Electrotransfer ProtocolMette Joergensen, Birgit Agerholm-Larsen, Peter E. Nielsen, Julie Gehl.110Biodistribution <strong>of</strong> Plasmid AMEP, a Potent Antitumor <strong>Gene</strong> Productfor the Treatment <strong>of</strong> Metastatic MelanomasCeline Bouquet, Elisabeth Dam, Veronique Trochon-Joseph, Valerie Polard, VincentSainte-Catherine, Emilienne Soma, Sophie Lebel-Binay.111In Vivo Intracellular Injection Needle (IvIn) Technology: A NewNeedle for Improved <strong>Cell</strong>ular Uptake <strong>of</strong> DNA in a Live AnimalLars Frelin, Gustaf Ahlén, Matti Sällberg.112A Study on Techniques for Safety Assessment <strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong>Products Using ElectroporationMin Joung Choi, Il Ung Oh, Sung Yeol Han, Oh Seok Kwon, Seung Hwa Hong.113Toxicology and Safety Studies <strong>of</strong> Plasmid AMEP, a New EfficientAntitumor <strong>Gene</strong> Product for the Treatment <strong>of</strong> Metastatic MelanomasCeline Bouquet, Veronique Trochon-Joseph, Valerie Polard, Vincent Sainte-Catherine,Emilienne Soma, Sophie Lebel-Binay.114Enhancement <strong>of</strong> the Immune Response <strong>of</strong> a Noval DNA VaccineEncoding Conserved NS3 and Core Fusion <strong>Gene</strong> <strong>of</strong> HCV Injected byIntradermal Electrotransfer in MiceXiao Yin, Jian Lu, Wenjie Tan, Jie Guan, Li Ruan.115Electroporation-Enhanced DNA Prime/Protein Boost Strategy ToImprove Cross-Clade Immunogenicity <strong>of</strong> HIV-1 Envelope AntigenKate E. Broderick, K. Muthumani, Xuefei Shen, Jian Yan, Feng Lin, Janess Mendoza,Amir Khan, Niranjan Y. Sardesai, David B. Weiner.116Effectiveness <strong>of</strong> Impedance Spectroscopy To Monitor Impact <strong>of</strong> anElectroporation Protocol on TissueJ. Anthony Llewellyn, Loree Heller, Richard A. Gilbert.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 93Abstract DirectoryThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-SouthChemical and Molecular Conjugates I117Modular Ternary Polyacridine DNA Polyplexes for Targeted <strong>Gene</strong>DeliveryNicholas J. Baumhover, Christian A. Fernandez, Sanjib Khargharia, Kevin G. Rice.118Biodegradable Polyamino Acid-Based Polycations for Safe andEffective <strong>Gene</strong> Transfer Minimizing Cumulative ToxicityKeiji Itaka, Takehiko Ishii, Kazunori Kataoka.119Interplay <strong>of</strong> Clathrin and Caveolin Trafficking Pathways in Polymer-Based <strong>Gene</strong> DeliveryMark E. Hwang, Rahul K. Keswani, Daniel W. Pack.120Folate Linked Targeting Systems Increase Transfection Efficiency andSpecificity in Human Epithelial <strong>Cell</strong>sNathalie Carmoy, Emmanuel Mornet, Céline Lainé, Loic Lemiegre, Tony Le Gall, DanielleGillet, Mattias Lindberg, Rémi Marianowski, Pierre Lehn, Thierry Benvegnu, TristanMontier.121In Vivo <strong>Gene</strong> Delivery from Modified PLG ScaffoldsMisael O. Aviles, Lonnie D. Shea.122The Influence <strong>of</strong> Charge Density and Substituent Composition onImidazolium-Containing Polymers for Mediated DNA DeliveryMichael H. Allen, Matthew D. Green, Timothy E. Long.123Concentrated PEI Formulations Retain Biological Efficacy FollowingLong-Term Storage at 4°CLee A. Davies, Graciela A. Nunez-Alonso, Stephen C. Hyde, Deborah R. Gill.124Charge Density and Molecular Weight <strong>of</strong> Polymeric <strong>Gene</strong> CarrierAre Key Parameters Influencing Its DNA Compaction Ability andTransfection EfficiencyYong Ren, Xuan Jiang, Hai-Quan Mao.125Designing Imidazole-Containing Polymeric Vectors with ControlledCharge Densities for <strong>Gene</strong> DeliveryMatthew D. Green, John M. Layman, Alec S. Good, Sean M. Ramirez, Lulit A. Affin,Michael H. Allen, Timothy E. Long.126Bioreducible Poly(amido ethyleneimine) and Its PolyethyleneGlycol Graft Copolymer Analog Formulations Offer Ease <strong>of</strong> Use inOptimizing Improved Nucleic Acid DeliveryJames W. Yockman, Jonathan H. Brumbach, Young Won Lee, Chao Lin, Johan F. J.Enbersen, Jan Feijen, Sung Wan Kim.127Role <strong>of</strong> Glycosaminoglycans in Peptide-Mediated DNA DeliveryRangeetha J. Naik, Pallavi Chandra, Anindo Chatterjee, Munia Ganguli.128Galactosylated-DPC Nanoparticles for Targeted Hepatic <strong>Gene</strong>Delivery In VivoKeun-Sik Kim, Yeon Kyung Lee, Jung Seok Kim, Jin Ee Back, Sang Il Park, Hwa YeonJeong, Young Eun Shin, Yong Serk Park.129Effect <strong>of</strong> Molecular Weight on Plasmid DNA Delivery Efficiency <strong>of</strong>Palmitic Acid Substituted PolyethylenimineK. C. B. Remant, Breanne Landry, Hasan Uludag.130Electrospun Fibers with the Core-Loading <strong>of</strong> DNA andPoly(ethyleneimine) Complex as Potential Tissue Engineering ScaffoldYe Yang, Long Cheng, Shuhui He, Jie Zou, Fang Chen, Xiaohong Li.131A Novel Cationic Lipophosphoramide with Phytanyl Chains: Gain inTransfection Efficiency and Clinical Approach Concerning the Responseto Lip<strong>of</strong>ectionMattias Lindberg, Nathalie Carmoy, Christophe Lorilleux, Aurore Fraix, Tony Le Gall,Pierre Lehn, Paul-Alain Jaffrès, Tristan Montier.132Cationic Peptide-Based DNA Delivery In Vitro: The Role <strong>of</strong> DNACondensation and ReleaseAnita Mann, Garima Thakur, Vasundhara Shukla, Rangeetha Naik, Anand Kamal Singh,Richa Khanduri, Munia Ganguli.<strong>Gene</strong>tic and Metabolic Diseases <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I133Modeling and Correcting Fibrous Dysplasia with Lentiviral VectorsIsabella Saggio, Cristina Remoli, Stefania Cersosimo, Stefania Piersanti, Paolo Bianco,Mara Riminucci.134Bone Marrow Transplantation Cures Hemophilia AAntonia Follenzi, Sanj Raut, Simone Merlin, Rita Sarkar, Sanjeev Gupta.135<strong>Cell</strong> Autonomy in Methylmalonic Acidemia (MMA): Implications for<strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong>Irini Manoli, Justin R. Sysol, Randy J. Chandler, Jennifer Sloan, Patricia Zerfas, VictoriaH<strong>of</strong>fmann, Mones Abu-Asab, Maria Tsokos, Charles P. Venditti.Thursday, May 20 th


94<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryThursday, May 20 thThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-South<strong>Gene</strong>tic and Metabolic Diseases <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I136Efficacious <strong>Gene</strong> <strong>Therapy</strong> in Pompe Disease Requires Mannose-6-Phosphate Receptor ExpressionBaodong Sun, Deeksha Bali, Y.-T. Chen, Dwight D. Koeberl.137Development <strong>of</strong> a Safe Hematopoietic Stem <strong>Cell</strong> (HSC) MobilizationStrategy for <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> ThalassemiaEvangelia Yannaki, Thalia Papayannopoulou, Erica Jonlin, Ioannis Batsis, Pamela Becker,Fani Zervou, Angeliki Xagorari, Garyfalia Karponi, Varnavas Constantinou, AchillesAnagnostopoulos, Athanasios Fassas, George Stamatoyannopoulos.138Long Term Expression in Liver from scAAV Seems Derived fromIntegrated Vector GenomesPaula S. M. Miranda, Maude Flageul, Christine Kaeppel, Lysbeth ten Bloemendaal,Dominique Aubert, Nowrouzi Ali, Manfred Schmidt, Nicolas Ferry, Piter J. Bosma.139Human Amnion Epithelial (AE) Stem <strong>Cell</strong> Transplant Improves DiseasePhenotype and Survival in Mouse Models <strong>of</strong> Intermediate MapleSyrup Urine Disease (MSUD)K. J. Skvorak, K. Dorko, M. Hansel, F. Marongiu, V. Tahan, K. M. Gibson, Q. Sun, T.Bottiglieri, E. Arning, J. Davila, S. Strom.140<strong>Gene</strong> <strong>Therapy</strong> Approaches Combining the ImmunomodulatoryProperties <strong>of</strong> Liver and IGF-I To Prevent Type I DiabetesSabrina Tafuro, Xavier M. Anguela, Judith Agudo, Alba Casellas, David Callejas, MercèObach, Carles Roca, Albert Ruzo, Fàtima Bosch.141Hepatocyte Engineering and <strong>Gene</strong>tic Modification Approaches <strong>of</strong>Hemophilia Hepatocytes within a Living MouseKazuo Ohashi, Kohei Tatsumi, Chise Tateno, Hiroyuki Nakai, Anja Ehrhardt, Rie Utoh,Katsutoshi Yoshizato, Teruo Okano.142Serotype Optimization <strong>of</strong> an AAV Vector for <strong>Gene</strong> <strong>Therapy</strong> in Canineand Murine Glycogen Storage Disease Type Ia (GSD-Ia)Xiaoyan Luo, Amanda K. Demaster, Songtao Li, Kyha Williams, Talmage T. Brown, DanielM. Kozink, Andrew Bird, Dwight D. Koeberl.143Systemic Delivery <strong>of</strong> Bioactive Glucagon-Like Peptide 1 FollowingAdenoviral Vector-Mediated <strong>Gene</strong> Transfer to Murine SubmandibularGlandsAntonis Voutetakis, Anne M. Rowzee, Ana P. Cotrim, Changyu Zheng, Trushar Rathod,Tulin Yanik, Y. P. Loh, Bruce J. Baum, Niamh X. Cawley.144A Non-Viral, GNE-Lipoplex Treatment to Correct Sialylation Defectsin Gne-Mutant (M712T) MiceTal Yardeni, Carla Ciccone, Shelley Hoogstraten-Miller, Daniel Darvish, Yair Anikster, PhilMaples, Chris Jay, William A. Gahl, John Nemunaitis, Marjan Huizing.145Lentivirally Induced Over-Expression in the Mouse Hypothalamus;a Study <strong>of</strong> the Effects <strong>of</strong> Melanin Stimulating Hormones (MSH) inObesity and DiabetesKim Eerola, Siru Virtanen, Eriika Savontaus, Mikko Savontaus.146Alternative Methods for Ex Vivo <strong>Gene</strong> Transfer to Primary Murine B<strong>Cell</strong>Babak Moghimi, Ou Cao.147A Full Characterization <strong>of</strong> the CAG 140 Huntington’s Disease MouseModelAaron C. Rising, Eileen Denovan-Wright, Ron Mandel.Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I148Assessment <strong>of</strong> Pre-Clinical Safety <strong>of</strong> GMP Grade GNE-Lipoplexesafter Repeat Intravenous Injections in Balb/c MiceAnagha P. Phadke, Chris M. Jay, Phillip B. Maples, Neil Senzer, John Nemunaitis, AlexW. Tong.149Pre-Existing Antibodies to AAV8 Attenuates Micro-DystrophinExpression Following Targeted Vascular DeliveryLouise R. Rodino-Klapac, Chrystal Montgomery, William Bremer, Nancy Davis, KimberlyShontz, Vinod Malik, Katherine Campbell, Thomas J. Preston, Zarife Sahenk, K. ReedClark, Brian D. Coley, Christopher M. Walker, Jerry R. Mendell, Louis G. Chicoine.150AAV Microgene Transfer Reveals the <strong>Cell</strong>ular Motif for Dystrophin-Mediated Sarcolemmal Neuronal Nitric Oxide Synthase (nNOS)LocalizationYi Lai, Yongping Yue, Dongsheng Duan.151<strong>Cell</strong> Therapeutic Approach to Duchenne Muscular Dystrophy UsingMyogenic Differentiation <strong>of</strong> Multipotent Mesenchymal Stromal <strong>Cell</strong>sYuko N. Kasahara, Hiromi H. Kinoh, Hironori Okada, Jin-Hong Shin, Akiyo Nishiyama,Sachiko O. Hosoyama, Michiko W. Maeda, Akinori Nakamura, Takashi Okada, Shin’ichiTakeda.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 95Abstract DirectoryThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-SouthMusculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I152Splice Site Strength and Nonsense-Associated Exon Skipping in theDMD <strong>Gene</strong>Kevin M. Flanigan, Diane M. Dunn, Jerry R. Mendell, Alan Pestronk, Julaine M. Florence,United Dystrophinopathy Project Consortium, Robert B. Weiss.153Long Term Antisense Treatment in Dystrophic Mouse Models forDuchenne Muscular DystrophyAnnemieke Aartsma-Rus, Christa L. de Winter, Hans A. Heemskerk, Judith C. vanDeutekom, Gert-Jan B. van Ommen.154Exon Exchange Approach To Repair Duchenne Dystrophin TranscriptsStéphanie Lorain, Cécile Peccate, Maëva Le Hir, Graziella Griffith, Thomas Voit, LuisGarcia.155Gentamicin Treatment <strong>of</strong> Duchenne Muscular Dystrophy Reinforcesthe Potential for Mutation Suppression <strong>Therapy</strong>Vinod Malik, Louise R. Rodino-Klapac, Laurence Viollet, Cheryl Wall, Wendy King,Roula Al-Dahhak, Sarah Lewis, Christopher J. Shilling, Janaiah Kota, John Hayes, JohnD. Mahan, Katherine J. Campbell, Brenda Banwell, Majed Dasouki, Victoria Watts,Kumaraswamy Sivakumar, Ricardo Bien-Willner, Kevin M. Flanigan, Zarife Sahenk,Richard J. Barohn, Christopher M. Walker, Jerry R. Mendell.156Reverse Perfusion Affords Efficient Whole-Body <strong>Gene</strong> Transfer toMuscle in the Large AnimalFrederick Balzer, Jacqueline Farag, Alock Malik, Mihail Petrov, Andrew Mead, BenjaminKozyak, Kapil Gopal, Marilyn Mitchell, Connie Choi, Jessie Chen, David Low, CharlesYarnall, Charles Bridges, Hansell Stedman.157Manganese Superoxide Dismutase Plasmid/Liposome Complex(MnSOD-PL) and Mitochondrial Targeted Nitroxide JP4-039Ameliorate Irradiation-Induced Delay in Bone Wound HealingJean-Claude Rwigema, Abhay Gokhale, Michael Epperly, Julie Glowacki, Tracy Dixon, KenPatrene, G. David Roodman, Peter Wipf, Joel Greenberger.158<strong>Gene</strong>tic Correction <strong>of</strong> Netherton Syndrome Keratinocyte Stem <strong>Cell</strong>sWaseem Qasim, Katerina Semenova, Gill Talbot, John Harper, Adrian Thrasher, FernandoLarcher, Wei Di.159Adenoviral Human Vascular Endothelial Growth Factor-D <strong>Gene</strong>Transfer Causes Angiogenesis and Growth and Enlargement <strong>of</strong>Lymphatic Vessels in Ischemic Mouse Hind LimbsGalina Dragneva, Ivana Kholová, Svetlana Laidinen, Petra Korpisalo, Pasi Tuunanen,Nina Kaskenpää, Suvi Heinonen, Felicitas Yongabi, Anna Stefanska, Varpu Marjomäki,Olli Gröhn, Seppo Ylä-Herttuala.160Validation <strong>of</strong> Intravascular Administration <strong>of</strong> Adeno-AssociatedVvirus for Muscular Dystrophies in a Non-Human Primate ModelAravind Asokan, Julia Hegge, Joshua C. Grieger, Chengwen Li, Swati Yadav, Jade J.Samulski , Xiao Xiao, Juan Li, Jon Wolff, R. Jude Samulski, Scott W. J. McPhee.Infectious Diseases and Vaccines: Vaccine Related <strong>Gene</strong>Transfer Research161Suppression <strong>of</strong> Cocaine-Induced Hyperactivity in Mice Pre-Treatedwith an Adenovirus-Based Anti-Cocaine VaccineJonathan B. Rosenberg, Martin L. Hicks, Bishnu P. De, Stephen M. Kaminsky, Amira Y.Moreno, Kim D. Janda, Stefan Worgall, Ronald G. Crystal.162Translation <strong>of</strong> Electroporation-Mediated DNA Vaccine Delivery intoa Phase I Clinical Trial Evaluating an HIV DNA Vaccine in HealthyVolunteersClaire F. Evans, Sandhya Vasan, Karen E. Dolter, Arlene Hurley, Sarah J. Schlesinger,Drew Hannaman, David F. Gardiner, Daniel P. Dugin, Yaoxing Huang, Josephine Cox,Tony Tarragona, Len Dally, Carol Smith, Claudia Schmidt, Harriet Park, Jill Gilmour,Patricia Fast, Robert Bernard, David D. Ho.163Longterm Protective Immunity Against Lethal Pulmonary Challengewith the Pseudomonas aeruginosa Following Mucosal Administration<strong>of</strong> AdC7-OprF Expressing the P. aeruginosa Protein OprFAnja Krause, Wen Zhu, Yaqin Xu, Ju Joh, Ronald G. Crystal, Stefen Worgall.164Characterization <strong>of</strong> Adenoviruses Shed from the Macaque Gut andEvaluation <strong>of</strong> Immune Responses to Adenoviruses in the Macaque inthe Context <strong>of</strong> Future Adenoviral Vaccine StudiesSoumitra Roy, Roberto Calcedo, C. Angelica Medina-Jaszek, Qiuyue Qin, JenniferMiliaresis, Surina Boyd, Diane G. Carnathan, Michael R. Betts, James M. Wilson.165A Lentiviral HIV Vaccine Candidate Elicits Long-Term Humoral and<strong>Cell</strong>ular HIV-Specific Immunity and Minimal Neutralizing ActivityBenyam Asefa, Delia Ye, Nikolay Korokhov, Laurent M. Humeau, Franck Lemiale.166Coexpressed RIG-I Agonist Enhances Humoral Immune Response toInfluenza DNA VaccineJeremy M. Luke, Clague P. Hodgson, James A. Williams.Thursday, May 20 th


96<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryThursday, May 20 thThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-SouthInfectious Diseases and Vaccines: Vaccine Related <strong>Gene</strong>Transfer Research167<strong>Gene</strong>tically Engineered Bifidobacterium Displaying Salmonella-AntigenProtects Mice from Lethal Challenge <strong>of</strong> Salmonella Typhimurium in aMurine Typhoid Fever ModelToshiro Shirakawa, Sakura Yamamoto, Jun Wada, Takane Katayama, Takumi Jikimoto,Masakuni Nakamura, Shohiro Kinoshita, Kyung-Mi Lee, Masato Kawabata.168Signaling Lymphocytic Activation Molecule (SLAM, CD150)-BlindMeasles Virus Is Attenuated and Induces Strong Adaptive ImmuneResponses in Rhesus MonkeysVincent Leonard, Gregory Hodge, Jorge Reyes-del Valle, Michael McChesney, RobertoCattaneo.169Evaluation <strong>of</strong> Mismatched Immunity To Expand Cross-ProtectionAgainst Influenza VirusAmi Patel, Isabelle Meunier, Kaylie N. Tran, Anders Leung, Stephane Pillet, DarwynKobasa, Veronika von Messling, Gary P. Kobinger.170Immune Response Against Measles Virus Encoding Helicobacter pyloriNeutrophil-Activating Protein AntigenIanko D. Iankov, Iana Haralambieva, Evanthia Galanis.171<strong>Gene</strong>ration <strong>of</strong> Chimeric GB Virus B Clones Encoding the Hepatitis CVirus E1E2p7 and coreE1E2p7 toward the Infected Marmoset ModelsTingting Li, Lifang Shuai, Zixuan Chen, Anqi Wang, Wenjing Wang, Chengyao Li.172Characterization <strong>of</strong> Immune Response in Mice Induced byRecombinant Vaccinia Virus (Tiantan) Based Multivalent H5N1 AvianInfluenza VaccinesWen Wang, Yao Deng, Wenjie Tan, Hong Chen, Li Ruan, Yuelong Shu.173The Effects <strong>of</strong> the Calreticulin on the Immune Response Induced byDNA Vaccine Containing the Classical Swine Fever Virus E2 <strong>Gene</strong> inMouse ModelMing Kun Hsieh, Chia Chin You, Chienjin Huang.174Evaluation <strong>of</strong> the Multi-Electrode Array for Use in DNA VaccinationAgainst Bacillus anthracisAmy L. Donate, Richard Heller.Cancer – Immunotherapy I175Enhancing the Priming <strong>of</strong> Tumor Antigen Specific T <strong>Cell</strong> ResponsesUsing Oncolytic VirusesPhonphimon Wongthida, Rosa Diaz, Feorillo Galivo, Karen Kaluza, Jill Thompson,Timothy Kottke, Richard Vile.176AdCD40L Immunogene <strong>Therapy</strong> for Bladder Carcinoma – A Phase I/IIa TrialPer-Uno Malmstrom, Angelica S. I. Loskog, Camilla A. Lindqvist, Sara M. Mangsbo, MoaFransson, Alkwin Wanders, Truls Gardmark, Thomas H. Totterman.177Treatment <strong>of</strong> Refractory Cancer Patients with a Serotype 5/3Chimeric Oncolytic Adenovirus Expressing Granulocyte MacrophageColony-Stimulating Factor (GM-CSF): Virological, Immunological andClinical EndpointsAnniina Koski, Sari Pesonen, Vincenzo Cerullo, Sophie Escutenaire, Elina Haavisto, LottaKangasniemi, Pekka Simula, Timo Joensuu, Akseli Hemminki.178IL-15 Reduces Suppressive Effects <strong>of</strong> Regulatory T <strong>Cell</strong>s (Tregs) onAntigen-Specific Cytotoxic T Lymphocytes (CTLs)S. Perna, B. De Angelis, D. Pagliara, L. Zhang, C. Rooney, H. Heslop, M. Brenner, G.Dotti, B. Savoldo.179PiggyBac Transposon Modified EBV Specific Human T Lymphocytesfor HER2-Positive <strong>Cell</strong>ular Immunotherapy In VivoYozo Nakazawa, Nabil Ahmed, Vita Salsman, Ann Leen, Stephen Gottschalk, MatthewH. Wilson, Cliona M. Rooney.180Adenovirus-Mediated Intratumoral Expression <strong>of</strong> ImmunostimulatoryMolecules Together with Systemic Depletion <strong>of</strong> Regulatory T-<strong>Cell</strong>sInduces Tumor-Destructive Immune Responses in Mouse ModelsYing Liu, Jonas Persson, Sebastian Tuve, Roma Yumul, ZongYi Li, Steve R<strong>of</strong>fler, AndreLieber.181Key Role <strong>of</strong> B <strong>Cell</strong> as Antigen Presenting <strong>Cell</strong>s in the Anti-TumorImmunity Induced by a Combined Immune-Stimulatory/ConditionalCytotoxic <strong>Therapy</strong> for GlioblastomaMarianela Candolfi, Kader Yagiz, James F. Curtin, Hikmat Assi, Mia Wibowo, GabrielleAlzadeh, David Foulad, Kurt Kroeger, Anderson Alden, Chunyan Liu, Robert Dunn, PedroR. Lowenstein, Maria G. Castro.182<strong>Gene</strong>tically Engineered Malignant Glioma-Specific T <strong>Cell</strong>s Resistant toGlucocorticoidsSeogkyoung Kong, Richard P. Junghans, Prakash Sampath.183Adoptive Transfer <strong>of</strong> T <strong>Cell</strong>s <strong>Gene</strong>tically Targeted to the MUC16Antigen Eradicates Established Peritoneal Ovarian Tumors in SCID-Beige MiceAlena A. Chekmasova, Thapi D. Rao, David R. Spriggs, Renier J. Brentjens.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 97Abstract DirectoryThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-SouthCancer – Immunotherapy I184Adoptive Transfer <strong>of</strong> Autologous T <strong>Cell</strong>s Targeted to Prostate SpecificMembrane Antigen (PSMA) for the Treatment <strong>of</strong> Castrate MetastaticProstate CancerOriana Borquez-Ojeda, Jolanta Stefanski, Xiuyan Wang, Jason Plotkin, Jinrong Qu,Luis Rodriguez, Elliot Servais, Kei Suzuki, Larrisa Shenker, Vladimir Ponomarev, SusanSlovin, Michel Sadelain, Isabelle Rivière.185A New Human-Derived Reporter <strong>Gene</strong> Suitable for Clinical PETImaging <strong>of</strong> T-<strong>Cell</strong> TraffickingYury Likar, Juan Zurita, Larissa Shenker, Maxim Moroz, Shangde Cai, Anton Neschadim,Jeffrey Medin, Hedvig Hricak, Michel Sadelain, Vladimir Ponomarev.186Inhibition <strong>of</strong> Mice Colon Tumor Growth by Il-15 Immunogene <strong>Therapy</strong>Xianghui He, Weidong Li, Yifeng Wang, Linan Hou, Liwei Zhu.187Development <strong>of</strong> High-Performance HVJ-E for Cancer Immune <strong>Therapy</strong>Kotaro Saga, Katsuto Tamai, Takehiko Yamazaki, Yasufumi Kaneda.188Combined <strong>Gene</strong> <strong>Therapy</strong> Using Adenovirus Expressing Interleukin-12,Granulocyte-Macrophage Colony-Stimulating Factor, and ThymidineKinase with Prodrug GanciclovirJi-seong Kim, Kyoung-ju Choi, Chae-Ok Yun.189Safety Data <strong>of</strong> MGN1601, a Tumor Vaccine, Made <strong>of</strong> Allogeneic,Transfected and Irradiated Tumor <strong>Cell</strong>s in Combination with anImmunomodulator for the Treatment <strong>of</strong> Metastatic Renal <strong>Cell</strong>CarcinomaManuel Schmidt, Baerbel Volz, Matthias Schr<strong>of</strong>f, Kerstin Kapp, Christiane Kleuss, MarinaTschaika, Burghardt Wittig.190Development <strong>of</strong> an HLA-A*0201 Restricted T <strong>Cell</strong> Receptor ThatRecognizes Peptide Epitopes from MAGE-A3 and MAGE-A12 forTargeted Adoptive T <strong>Cell</strong> Immunotherapy <strong>of</strong> Multiple Cancer TypesNachimuthu Chinnasamy, Jennifer A. Wargo, Zhiya Yu, Timothy L. Frankel, John P. Riley,Maria R. Parkhurst, Steven A. Feldman, Nicholas P. Restifo, Paul F. Robbins, Steven A.Rosenberg, Richard A. Morgan.Cancer-Oncolytic Viruses I191 (Moved to Oral Abstract Session 413)A Comparative Study <strong>of</strong> Stem <strong>Cell</strong>-Based <strong>Cell</strong> Carrier Systems for theSystemic Delivery <strong>of</strong> an Oncolytic Adenovirus for Antiglioma <strong>Therapy</strong>Matthew A. Tayler, Atique U. Ahmed, Ilya V. Ulasov, Irina Y. Balyasnikova, Maciej S.Lesniak.192A Novel Coxsackievirus B Shows Remarkable Oncolytic CapacityAgainst Lung CancerShohei Miyamoto, Hiroyuki Inoue, Meiko Yamada, Xin Meng, Takafumi Nakamura,Yasuo Urata, Hiroyuki Shimizu, Kenzaburo Tani.193Preclinical and Clinical Data with a Fully Serotype 3 OncolyticAdenovirus Ad3-hTERT-E1A in the Treatment <strong>of</strong> Advanced SolidTumorsOtto Hemminki, Gerd Bauerschmitz, Silvio Hemmi, Sergio Lavilla-Alonso, Iulia Diaconu,Anna Kanerva, Vincenzo Cerullo, Minna Oksanen, Elina Haavisto, Timo Joensuu, SariPesonen, Akseli Hemminki.194Mechanism <strong>of</strong> Tumour Penetration by Oncolytic Viruses –Investigation in a 3 Dimensional In Vitro ModelC. A. Campion, C. Harty, G. C. O’Sullivan, J. K. Collins.195Mechanisms <strong>of</strong> Cyclophophamide Enhancing Anti-Tumor Effects <strong>of</strong>Oncolytic Vesicular Stomatitis Virus for Breast Cancer TreatmentHongxun Wang, Stephen Russell, Jian Qiao.196Clinical Characterization <strong>of</strong> the Adenovirus Death Protein-PotentiatedOncolytic Adenovirus VRX007Alex W. Tong, Anagha P. Phadke, Staci Horvath, William Wold, John Nemunaitis, NeilSenzer.197Safety and Immunological Effects <strong>of</strong> Corticosteroids in PatientsTreated with Oncolytic AdenovirusesMaria Rajecki, Mari Raki, Andreas Helminen, Sophie Escutenaire, Sari Pesonen, VincenzoCerullo, Timo Joensuu, Elina Haavisto, Laura Ahtiainen, Akseli Hemminki.198Effect <strong>of</strong> Decorin on Overcoming the Extracellular Matrix Barrier forOncolytic VirotherapyIl-Kyu Choi, Young-Sook Lee, Ji Young Yoo, A-Rum Yoon, Hye Jin Choi, Daniela G. Seidler,Chae-Ok Yun.199Chondroitinase ABC1 Mediated Enhancement <strong>of</strong> OV Spread andEfficacyNina Dmitrieva, Mariano Viapiano, Balveen Kaur.200Use <strong>of</strong> PET Imaging for In-Vivo Monitoring <strong>of</strong> Replication CompetentRetrovirus (RCR) Transduction <strong>of</strong> Malignant GliomasWeijun Wang, Hui Wang, Ryan Park, Hancheng Cai, Peter S. Conti, Nori Kasahara,Thomas C. Chen.Thursday, May 20 th


98<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryThursday, May 20 thThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-SouthCancer-Oncolytic Viruses I201Systemic Cancer Screening Using an Oncolytic HSV To Induce Tumor-Selective Expression <strong>of</strong> a Secretable Serum BiomarkerAndrew W. Browne, Jennifer L. Leddon, Mark A. Currier, Jon P. Williams, Nancy Ratner,Timothy P. Cripe.202Therapeutic and Tumor-Specific Immunity Induced by Combination<strong>of</strong> Dendritic <strong>Cell</strong>s and Oncolytic Adenovirus Expressing IL-12 and4-1BBLSong-Nan Zhang, Jing-Hua Huang, Joo-Hang Kim, Kyung-Ju Choi, Il-Kyu Choi, MingulLee, Chae-Ok Yun.203Oncolytic Herpes Simplex Virus Encoding Interferon b <strong>Gene</strong> forSquamous <strong>Cell</strong> Carcinoma <strong>of</strong> the Head and NeckHongtao Li, Stephen J. Russell.204Targeted Suicide <strong>Gene</strong> <strong>Therapy</strong> with Oncolytic AdenovirusCoding for FCU1, a Fusion Protein <strong>of</strong> Cytosine Deaminase andUracilphosphoribosyltransferaseJoão D. Dias, Ilkka Liikanen, Kilian Guse, Johann Foloppe, Marta Sloniecka, Iulia Diaconu,Ville Rantanen, Minna Eriksson, Tanja Hakkarainen, Monika Lusky, Philippe Erbs, SophieEscutenaire, Anna Kanerva, Sari Pesonen, Vincenzo Cerullo, Akseli Hemminki.Cancer-Targeted <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I205Controlled Nuclear Localization <strong>of</strong> p53 in Breast Cancer <strong>Cell</strong>sMohanad Mossalam, Carol S. Lim.206Effect <strong>of</strong> Transcriptionally Targeted Suicide <strong>Gene</strong> <strong>Therapy</strong> for Small<strong>Cell</strong> Lung Cancer In Vitro and In Vivo by DOTAP/Cholesterol- andAdenoviral-Mediated DeliveryCamilla L. Christensen, Torben Gjetting, Frederik Cramer, Skovgaard Hans.207NF-kB Activity Regulates MSC Accumulation at Tumor SitesRyosuke Uchibori, Hiroyuki Mizuguchi, Tomonori Tsukahara, Masashi Urabe, HiroakiMizukami, Akihiro Kume, Keiya Ozawa.208Dynamics <strong>of</strong> <strong>Therapy</strong> <strong>of</strong> Malignant Melanoma with VesicularStomatitis VirusJyoti Dev, Chetan P. Offord, Diana M. Rommelfanger, Richard G. Vile, Zeljko Bajzer,David Dingli.209Therapeutic Efficacy <strong>of</strong> a Lentiviral Mediated Thymidine Kinase/Ganciclovir Treatment in an Immunocompetent Rat Malignant GliomaModelJere T. Pikkarainen, Hanna P. Lesch, Haritha Samaranayake, Minna U. Kaikkonen, Ann-Marie Määttä, Seppo Ylä-Herttuala.210Insertion <strong>of</strong> Transcription Factor Nuclear Factor Kappa B Binding Sitesinto Plasmid DNA Expressing a Suicide <strong>Gene</strong> Facilitates IncreasedSmall <strong>Cell</strong> Lung Cancer <strong>Cell</strong> Death In Vitro Due to Increased NuclearUptakeFrederik Cramer, Camilla L. Christensen, Hans S. Poulsen.211Preirradition <strong>Gene</strong> <strong>Therapy</strong> Against Transforming Growth Factor-bSignal Is Radioprotective for the LiverZhao-Chong Zeng, Shi-Suo Du, Ming Qiang, Zheng-Yu Zhang.212Rabbit VX-2 Brain Tumor Model for Development <strong>of</strong> Improved VectorAdministration TechniquesFarizan Ahmad, Agnieszka Pacholska, Anna Hyvärinen, Venla Tuppurainen, ThomasWirth, Seppo Ylä-Herttuala.213Nano-Flow 2D-HPLC MALDI-TOF MS/MS for Differentially ExpressedProtein Analysis in Breast Cancer Patient SamplesCourtney Haddock, R. B. Holtz, Neil Senzer, Phillip B. Maples, John Nemunaitis.214In Vitro Characterization for In Vivo Translation <strong>of</strong> a Novel Non-Viral<strong>Gene</strong> Delivery VectorLuke J. Higgins, Gloria L. Hwang, Regina H. Katzenberg, Nishita Kothary, Daniel Y. Sze,Lawrence V. H<strong>of</strong>mann.215Altering the Tropism <strong>of</strong> Herpesvirus saimiri to Selectively InfectHepatocellular Carcinoma (HCC) <strong>Cell</strong>sSusan Turrell, Adrian Whitehouse.216Improved <strong>Gene</strong> Transfer into Renal Carcinoma <strong>Cell</strong>s Using anAdenovirus VectorShuji Terao, Bishnu Acharya, Toru Suzuki, Katsuyuki Hamada, Hiroyuki Mizuguchi,Akinobu Gotoh.217Prostate Cancer <strong>Gene</strong> <strong>Therapy</strong> Using Adenovirus Expressing a NovelApoptosis-Inducing <strong>Gene</strong> and Its Conjugation with ChemotherapyXiongwen Zhang, Ben Beheshti, Yi Lu.218Development and Preclinical Testing <strong>of</strong> Large Batches <strong>of</strong> SNS01-A– An eIF5A-Based <strong>Gene</strong> <strong>Therapy</strong> Nanoparticle Designed for theTreatment <strong>of</strong> Multiple MyelomaCatherine A. Taylor, Zhongda Liu, Terence Tang, Bin Ye, Bruce Galton, Kathleen A.Donovan, John A. Lust, John E. Thompson, Richard Dondero.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 99Abstract DirectoryThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-South219Angiotensin II Type 2 Receptor <strong>Gene</strong> Mediated Apoptosis <strong>of</strong> HumanLung Adenocarcinoma A549 <strong>Cell</strong>sYanling Zhang, Ling Zhang, Yongxin Gao, Baihong Chen, Hongwei Li.Hematologic and Immunologic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I220Modeling X-Linked Chronic Granulomatous Disease Using NeutrophilsDifferentiated from Patient-Derived Induced Pluripotent Stem <strong>Cell</strong>sColin L. Sweeney, Jizhong Zou, Uimook Choi, Bin-Kuan Chou, Linzhao Cheng, Harry L.Malech.221Use <strong>of</strong> the Natural Human Artemis Promoter To Regulate HumanArtemis Expression Prevents Toxicity and Corrects Artemis DeficientSCID after Ex Vivo Lentiviral Transduction in a Murine Model <strong>of</strong> theDiseaseMegan M. Multhaup, Kelly Podetz-Pedersen, Sweta Gurram, Andrea Karlen, DebraSwanson, Nikunj V. Somia, Bruce R. Blazar, Morton J. Cowan, R. Scott McIvor, McIvorLab.222Lack <strong>of</strong> Enhancer-Blocking Activity by 400bp cHS4 Insulator in aHuman T- Lymphoid <strong>Cell</strong> lineDisha A. Mody, Taihe Lu, Sheng Zhou, John Gray, Brian P. Sorrentino.223Liver-Directed Intravascular Fetal Delivery <strong>of</strong> scAAV FacilitatesSustained Transgene Expression with Evidence <strong>of</strong> TransplacentalTrafficking and Possible Vector Integration EventsC. N. Mattar, A. C. Nathwani, N. Dighe, S. Waddington, J. McIntosh, A. Biswas, N. Fisk,A. M. David<strong>of</strong>f, A. David, D. Peebles, A. Nowrouzi, C. Kappel, M. Schmidt, M. Choolani,J. Chan.224Helper-Dependent Adenoviral Vector <strong>Gene</strong> <strong>Therapy</strong> Mediates Long-Term Low-Level Correction <strong>of</strong> Hemophilia A in the Murine Model afterNeonatal <strong>Gene</strong> TransferChuhong Hu, Fides D. Lay, Racel G. Cela, Masataka Suzuki, Joseph Springer, Ronald W.Busuttil, Brendan Lee, Gerald S. Lipshutz.225Lentiviral Vector Mediated <strong>Gene</strong> <strong>Therapy</strong> for the Treatment <strong>of</strong>Wiskott-Aldrich Syndrome: In Vitro and In Vivo Preclinical Studieswith Human CD34 + <strong>Cell</strong>sSamantha Scaramuzza, Marita Bosticardo, Anna Ripamonti, Maria C. Castiello, LucaBiasco, Giuliana Vallanti, Marina Radrizzani, Anne Galy, Robbert Bredius, BarbaraCappelli, Maria G. Roncarolo, Luigi Naldini, Anna Villa, Alessandro Aiuti.226Development <strong>of</strong> B-Lineage Specific Lentiviral Vectors for Use in <strong>Gene</strong><strong>Therapy</strong> for B-<strong>Cell</strong> DisordersBlythe D. Sather, Brigid V. Sterling, Byoung Y. Ryu, Stéphanie Baron, David J. Rawlings.227Development <strong>of</strong> a <strong>Gene</strong> Transfer System for Wiskott-AldrichSyndrome Using Foamy Virus VectorsToru Uchiyama, Masaki Shimizu, Fabio Candotti.228Integration Site Distribution in Mice Following Therapeutic <strong>Gene</strong>Transfer To Treat Beta-ThalassemiaKeshet Ronen, Olivier Negre, Nirav Malani, Maria Denaro, Beatrix Gillet-Legrand, PhilippeLeboulch, Julian D. Down, Emmanuel Payen, Frederic D. Bushman.229Validation <strong>of</strong> the Tranduction <strong>of</strong> Patients Hematopoietic Progenitor<strong>Cell</strong>s with a Clinical-Grade Lentiviral Vector Developed for Wiskott-Aldrich Syndrome <strong>Gene</strong> <strong>Therapy</strong>Salima Hacein-Bey Abina, Elena De Falco, Johanna Blondeau, Sabine Charrier, LaureCaccavelli, Mike Blundell, Alain Fischer, Alessandro Aiuti, Adrian J. Thrasher, MarinaCavazzana-Calvo, Anne Galy.230<strong>Gene</strong>ration <strong>of</strong> an Optimized Lentiviral Vector Encoding a High-Expression fVIII Transgene for <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> Hemophilia AJennifer M. Johnston, Robby Moot, Bagirath Gangadharan, Christopher B. Doering, H.Trent B. Spencer.231Biodistribution <strong>of</strong> a High Dose <strong>of</strong> Intraarticularly Administered AAV-5Vectors to Lewis RatsScott A. Loiler, Karin de Cortie, Paul P. Tak, Margriet J. Vervoordeldonk.232Ex Vivo Lentiviral Transduction <strong>of</strong> the Human Purine NucleosidePhosphorylase (PNP) <strong>Gene</strong> in Bone Marrow Derived HematopoieticStem <strong>Cell</strong>s: An In Vivo Model <strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong> for PNP DeficientT-<strong>Cell</strong> ImmunodeficiencyKelly M. Podetz-Pedersen, Megan M. Multhaup, Andrea D. Karlen, Debra L. Swanson, K.Scott Baker, Nikunj V. Somia, R. Scott McIvor.233Signed Outside: A Surface Marker System for Transgenic CytoplasmicProteinsVital Wohlgensinger, Reinhard A. Seger, Martin D. Ryan, Janine Reichenbach, Ulrich Siler.869 (Moved from Poster Session III)Myelospecific Self-Inactivating Gammaretroviral Vectors for p47 phoxCGD <strong>Gene</strong> <strong>Therapy</strong>Vital Wohlgensinger, Christian Brendel, Axel Schambach, Manuel Grez, JanineReichenbach, Reinhard A. Seger, Ulrich Siler.Thursday, May 20 th


100<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryThursday, May 20 thThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-South<strong>Gene</strong> Regulation234Low Levels <strong>of</strong> Expression <strong>of</strong> CD18 Using the pgk Promoter in aFoamy Viral Vector To Treat Animals with Canine Leukocyte AdhesionDeficiencyThomas R. Bauer, Jr., Erik M. Olson, Laura M. Tuschong, James M. Allen, Yi Li, Tanya H.Burkholder, Dennis D. Hickstein, David W. Russell.235A Dual Self-Inactivating Lentiviral Vector System for Mifepristone-Inducible Bicistronic Transgene Expression in Primary NeuralProgenitor <strong>Cell</strong>sMichael A. Mooney, Kenneth H. Fischbeck, Tyler Mark Pierson.236A Novel Human Artificial Chromosome Vector Derived fromChromosome 14 (14HAC): Higher Transgene Expression SystemUsing Sub-Telomere SequencesMinoru Kakeda, Keiko Nagata, Akiko Sano, Kanako Osawa, Akiyo Okazaki, ShingoShitara, Yasuhiro Kazuki, Mitsuo Oshimura, Kazuma Tomizuka.237Effective <strong>Gene</strong> Delivery to Wounds by Transplantation <strong>of</strong> Minced SkinParticles Transfected with Adenoviral-VEGFTaro Koyama, Florian Hackl, Pejman Aflaki, Juri Bergmann, Baraa Zuhaili, EmilyWaisbren, Feng Yao, El<strong>of</strong> Eriksson.238MicroRNA-122 Regulation Prevents Liver Toxicity <strong>of</strong> Suicide <strong>Gene</strong><strong>Therapy</strong>Gang Wang, Wenhong Tian, Yunfan Liu, Wenjie Tan, Xiaobing Wu.239Regulation <strong>of</strong> Endogenous GAD67 Expression Using ArtificialTranscription FactorsErika Elgstrand Wettergren, Fredrik Gussing, Maike Sest, Torbjörn Gräslund, CeciliaLundberg.240Enhanced In Vivo Transgene Expression by Two-Step TranscriptionAmplificationGenki Kanda, Hiroshi Ochiai, Hideyoshi Harashima, Hiroyuki Kamiya.241Targeted Knockdown <strong>of</strong> Serum Response Factor in Hearts <strong>of</strong> AdultSRF Conditional Knock-Out Mice by AAV9-Mediated Transfer <strong>of</strong> Cre-RecombinaseStanislas M. Werfel, Andreas Jungmann, Rolf Sprengel, Jürgen A. Kleinschmidt, AlfredNordheim, Hugo A. Katus, Oliver J. Müller.242Pro-Proliferative Functions <strong>of</strong> Drosophila Small Mitochondrial HeatShock Protein 22 in Human <strong>Cell</strong>sJihoon Ryu, Renu Wadhwa, Ran Gao, Il-Kyu Choi, <strong>Gene</strong>vieve Morrow, Kamal Rajput,Inwook Kim, Sunil C. Kaul, Robert M. Tanguay, Chae-Ok Yun.243Interleukin-(IL)-24 Regulates Epithelial to Mesenchymal Transition(EMT) Transcription FactorsCorbin Goerlich, Manish Shanker, Jiankang Jin, Sendurai Mani, Rajagopal Ramesh.244Transient Epigenetic <strong>Gene</strong> <strong>Therapy</strong> as a Rising Arm <strong>of</strong> a UniversalStem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong> Platform both for Ex Vivo and In VivoApplicationsRoger Bertolotti.Oligonucleotide & RNAi Therapeutics I245The Application <strong>of</strong> “Bi-Functional” Short Hairpin RNA in A FurinshRNAand GM-CSF (FANG) Autologous Vaccine for Cancer <strong>Therapy</strong>Zhaohui Wang, Connor Phalon, Padmasini Kumar, Chris M. Jay, Donald D. Rao, Alex W.Tong, Phillip B. Maples, Neil Senzer, John Nemunaitis.246A pH-Sensitive Fusogenic Peptide Modified siRNA-EncapsulatingNanoparticles Exerts the <strong>Gene</strong> Silencing Synergistically with Tumor-Specific Cleavable PEG-LipidYu Sakurai, Hiroto Hatakeyama, Hidetaka Akita, Motoi Oishi, Yukio Nagasaki, ShirohFutaki, Hideyoshi Harashima.247Systemic Administration <strong>of</strong> Bifunctional shRNA Lipoplex TargetingPDX-1 for Murine Model <strong>of</strong> Insulinoma and Pancreatic CancerShihe Liu, Zhaohui Wang, Chris M. Jay, Donald D. Rao, Nancy S. Templeton, Phillip B.Maples, Neil Senzer, F. C. Brunicardi, John Nemunaitis.248Augmentation <strong>of</strong> Docetaxel Anti-tumor Activity by a Stathmin-Specific Bi-Functional shRNAAlex W. Tong, Yang Yu, Donald D. Rao, Phillip B. Maples, Neil Senzer, John Nemunaitis.249RNAi- and U1i-Mediated <strong>Gene</strong> <strong>Therapy</strong>Annemart Koornneef, Pavlina Konstantinova, Bas Blits, Richard van Logtenstein, TitaRitsema, Puri Fortes, Harald Petry.250Lentiviral Delivery <strong>of</strong> EzH2 shRNA Inhibits Tumor Growth <strong>of</strong>Epithelial Ovarian CancerRamesh B. Batchu, Aamer M. Qazi, Assaad Semaan, Shelly M. Seward, SreedharChamala, Christopher S. Bryant, Robert Morris, Sanjeev Kumar, Christopher Steffes,Masood A. Shammas, Donald W. Weaver.251Prostate Tumor Radiosensitization through PSMA-Aptamer-TargetedsiRNA Knock-Down <strong>of</strong> DNA Repair PathwaysXiahoua Ni, Yonggong Zhang, Theodore L. DeWeese, Shawn E. Lupold.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010101Abstract DirectoryThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-SouthOligonucleotide & RNAi Therapeutics I252Preclinical Safety and Toxicity Assessment <strong>of</strong> Stathmin 1 BifunctionalshRNA Formulation Following a Single Intravenous Injection in RatsAnagha P. Phadke, Chris M. Jay, Donald D. Rao, Neil Senzer, Phillip B. Maples, JohnNemunaitis, Alex W. Tong.253Application <strong>of</strong> siRNA-Containing Nanoparticles for In Vitro and InVivo <strong>Gene</strong> Silencing in Tumor by Controlling Intracellular TraffickingHiroto Hatakeyama, Erika Ito, Hidetaka Akita, Motoi Oishi, Yukio Nagasaki, ShirohFutaki, Hideyoshi Harashima.254The Novel Comb Shaped Amphiphilic Polyallylamine for PotentialOral Delivery <strong>of</strong> siRNA to Gastrointestinal Cancers: PhysicochemicalCharacterizations and In Vitro EvaluationsJianfeng Guo, Colin J. Thompson, Woei Ping Cheng, Caitriona O’Driscoll.255Optimalization <strong>of</strong> Polymerase II sh/miRNA Expression forTherapeutic ApplicationsPiotr Maczuga, Tita Ritsema, Annemart Koorneef, Harald Petry, Sander van Deventer,Pavlina Konstantinova.256The Effect <strong>of</strong> Helper Lipids on Cationic Lipid-Based siRNA DeliverySystemGuoqin Niu, Hartmut Derendorf, Jeffrey A. Hughes.257c-Cbl shRNA-Expressing Adenovirus Functions as a Sensitizer <strong>of</strong>TRAIL-Induced ApoptosisSeeun Oh, So Y. Kim, Bo K. Sun, Dongho Kim, Suhung Lee, Yong J. Lee, Joo-Hang Kim,Jae J. Song.258ncRNAs Originating from the Dystrophin <strong>Gene</strong> as Biomarker forAssessing Antisense <strong>Therapy</strong>Matteo Bovolenta, Annarita Armaroli, Marcella Neri, Simona Brioschi, Marina Fabris,Chiara Scotton, S<strong>of</strong>ia Falzarano, Paola Rimessi, Emanuele Valli, Giovanni Perini, LucianoMerlini, Francesca Gualandi, Alessandra Ferlini.259Efficient In Vivo Delivery <strong>of</strong> Cholesterol-Conjugated siRNA to theLiver Using Endogenous Chylomicron RemnantKazutaka Nishina, Kie Yoshida, Yoshitaka Uno, Hiroya Kuwahara, Wenying Piao,Hidehiro Mizusawa, Takanori Yokota.Stem <strong>Cell</strong> Biology260Controlled Nonviral <strong>Gene</strong> Delivery and Expression Using StableNeural Stem <strong>Cell</strong> Line Transfected with a Hypoxia-Inducible <strong>Gene</strong>Expression SystemYoon Ha, MengLu Liu, KeungNyun Kim, DoHeum Yoon.261Non-Viral Carriers Deliver mRNA Encoding Marker Proteins in HeLacells and mRNA Encoding CXCR4 in Mesenchymal Stem <strong>Cell</strong>sJoanna Rejman, Geertrui Tavernier, Joseph Demeester, Stefaan S. D. Desmedt.262<strong>Gene</strong>tic Modification <strong>of</strong> Neural Stem <strong>Cell</strong>sRavi K. Chandra.263APOA-1 Is a Novel Marker <strong>of</strong> Erythroid <strong>Cell</strong> Maturation fromHematopoietic Stem <strong>Cell</strong>s in Mice and HumansTomoko Inoue, Daisuke Sugiyama, Ryo Kurita, Tatsuo Oikawa, Kasem Kulkeaw, HirotakaKawano, Yoshie Miura, Michiyo Okada, Youko Suehiro, Atsushi Takahashi, TomotoshiMarumoto, Hiroyuki Inoue, Norio Komatsu, Kenzaburo Tani.264A Stem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong> Approach for Enhancing the Efficacy <strong>of</strong>Breast Cancer <strong>Therapy</strong> by Trastuzumab (Herceptin)Ines Beyer, Jonas Persson, ZongYi Li, Ying Liu, Roma Yumul, Andre Lieber.265Ovarian Cancer Stem <strong>Cell</strong>s Are in an Epithelial/Mesenchymal HybridState and Have Multipotential Capacity for DifferentiationRobert Strauss, ZongYi Li, Ying Liu, Nicole Urban, Charles Drescher, Jiri Bartek, AndreLieber.266SB-T-1214 Dramatically Downregulates Stem <strong>Cell</strong>-Related <strong>Gene</strong>Expression Pr<strong>of</strong>iles in Metastatic Colon Cancer <strong>Cell</strong>sYan Leyfman, Yuan Wang, Iwao Ojima, Galina Botchkina.267Identifying <strong>Gene</strong>s and Signaling Pathways Regulating the QuiescentState in Muscle Stem (Satellite) <strong>Cell</strong>sKristy Boyle, Jeffrey S. Chamberlain.268Pathway Analysis <strong>of</strong> Multipotent Mesenchymal Stromal <strong>Cell</strong>s UsingBacMam ToolsLucas Chase, Thomas Machleidt, Mahendra S. Rao, Uma Lakshmipathy.269The Placental Microvasculature Is a Rich Source <strong>of</strong> HighlyProliferative Endothelial Colony Forming <strong>Cell</strong>s Capable <strong>of</strong> DevelopingFunctioning Vessels In VivoBrian M. Rapp, Reza Saadatzadeh, Janak Bhasvar, Richard Ofstein, Michael P. Murphy.270microRNA Function during Human Mesenchymal Stem <strong>Cell</strong> AdipogenicDifferentiationAnnette Enright, Vladimir Benes, Georgina Shaw, Frank Barry, Linda Howard.Thursday, May 20 th


102<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryThursday, May 20 thThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-SouthStem <strong>Cell</strong> Biology271A Novel Fluorescence/Fluorine MRI Dual-Mode Reagent for <strong>Cell</strong>Tracking and Determination <strong>of</strong> <strong>Cell</strong> FateAnthony G. Balducci, Brooke Helfer, Amy K. Wesa, Jelena Janjic, Eric Ahrens.272The Stromal Vascular Fraction <strong>of</strong> Human Term Placenta Is a RichSource <strong>of</strong> Pluripotent Mesenchymal Stem <strong>Cell</strong>sMohammad R. Saadatzadeh, Janak Bhavsar, Brian M. Rapp, Peter J. Moreno, MichaelP. Murphy, Michael P. Murphy.273The HSV-2 Protein, ICP10PK, Potentiates the NeuroprotectiveActivity <strong>of</strong> Stem <strong>Cell</strong>sKyle A. Wilson, Jennifer M. Laing, Aurelian Laure.<strong>Cell</strong> Processing and Vector Production I274Process Improvements for cGMP Manufacture <strong>of</strong> Lexgenleucel-T forHIV TreatmentReuben Cohen, Yelena Skripchenko, Tony Encinas, Gerard J. McGarrity, Shuyuan Zhang.275TAG Vaccine Clinical Trial ManufacturingPhillip B. Maples, Padmasini Kumar, Yang Yu, Beena O. Pappen, Joseph Kuhn, JohnNemunaitis.276Development and GMP Validation <strong>of</strong> Large Scale LentiviralTransduction <strong>of</strong> CD34+ <strong>Cell</strong>s for Metacromatic Leukodystrophy andWiskott-Aldrich Syndrome <strong>Gene</strong> <strong>Therapy</strong> Clinical TrialsGiuliana Vallanti, Chiara Pozza, Francesca Rossetti, Letizia Martelli, Giuseppina Marano,Alessandra Gatti, Lucia Turchetto, Paola Massariello, Monica Salomoni, Claudia Benati,Sergio Fracchia, Alessandro Lorini, Marco Dieci, Raul Ziliotto, Alessandra Biffi, AlessandroAiuti, Claudio Bordignon, Luigi Naldini, Marina Radrizzani.277Large Scale rVSV Viral Vector Production in a cGMP FacilityLara J. Ausubel, Marcia Meseck, Ivy Derecho, Patricia Lopez, Christine Knoblauch, RossMcMahon, Jonathan Anderson, Nina Dunphy, Valerie Quezada, Mathew Luo, David Hsu,Savio Woo, Larry Couture.278Challenges in the Manufacturing <strong>of</strong> Retroviral <strong>Gene</strong> <strong>Therapy</strong> VectorsAna F. Rodrigues, Ana I. Amaral, Manuel J. T. Carrondo, Paula M. Alves, Ana S.Coroadinha.279Production <strong>of</strong> cGMP Grade Lentiviral VectorLara J. Ausubel, Christine Knoblauch, Patricia Lopez, Ross McMahon, Jonathan Anderson,Valerie Quezada, Nina Dunphy, Ivy Derecho, Mathew Luo, David Hsu, Larry Couture.280Design and Operation <strong>of</strong> a New State-<strong>of</strong>-the-Art Multi-Product cGMPCleanroom Facility for Vector Production and <strong>Cell</strong> ManipulationJohannes C. M. van der Loo, William P. Swaney, Kathryn Mangopoulos, MicheleBeuerlein, Diana L. Nordling, Lilith R. Reeves, Punam Malik.281Highly Efficient Manufacturing <strong>of</strong> Clinical Grade Lentiviral Vector in aNew, State <strong>of</strong> the Art Academic Good Manufacturing Practice (GMP)FacilityGerhard Bauer, Joseph Anderson, Matt Lindsey, Jan Nolta.282The Role <strong>of</strong> JNK in Lentivirus <strong>Gene</strong> TransferMeng Horng Lee, Roshan Padmashali, Stelios T. Andreadis.Late Breaking Science I922HSV-mediated Transfer <strong>of</strong> the Sox11 <strong>Gene</strong> and Peripheral NerveRegenerationXiaotang Jing, Shaohua Huang, Joseph C. Glorioso, Kathryn M. Albers923Real-Time In Vivo Imaging <strong>of</strong> Neural Stem <strong>Cell</strong>s for Targeted <strong>Therapy</strong><strong>of</strong> MedulloblastomaMargarita Gutova, Vazgen Kandalyyan, Ira Harutyunyan, Joseph Najbauer, Rex A.Moats, Gregory M. Shackleford, Michael E. Barish and Karen S. Aboody924Clinical Trial Entry <strong>of</strong> a Novel Prodrug Activator <strong>Gene</strong> TransferTechnology for the Treatment <strong>of</strong> CancerH. Gruber, C. Ibanez, A. daSilva, W. Gunzburg, D. Pertschuk, D. Gessner, N. Kasahara,C.R. Logg, O. Perez, D. Ostertag, J. Robbins, D.J. Jolly925Integration-Defective Lentiviral Vectors for Hemophilia: A SaferPlatform for Immune Tolerance Induction and Reduced GenotoxicityJanka Mátrai, Alessio Cantore, Cynthia C. Bartholomae, A Annoni, Abel Acosta-Sanchez, Ermira Samara-Kuko, Liesbeth De Waele, Ling Ma, Tim C. Nichols, Christ<strong>of</strong>von Kalle, Roncarolo, Marie-Gracia, Marinee K.L. Chuah, Manfred Schmidt, ThierryVandenDriessche, Luigi Naldini926Abstract Withdrawn927Targeting CD70 Positive Malignancies with T cells Expressinga CD27-Zeta Chimeric Antigen Receptor Reveals ImportantCostimulatory Role <strong>of</strong> CD27D. Shaffer, B Savoldo, G. Dotti, D Spencer, S. Kenney, C.M. Rooney, S. GottschalkEXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010103Abstract DirectoryThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-SouthLate Breaking Science I928Engineering Aptamers Against HIV-1 RT for Improved AntiviralBioactivityMargaret J. Lange, Angela S. Whately, Donald H. Burke929Directed Integration <strong>of</strong> New Insulated Lentiviral Vectors to theHeterochromatin towards Safer <strong>Gene</strong> Transfer to Stem <strong>Cell</strong>sAlexandre Artus, Caroline Duros, Yaïr Botbol, Simone Schultz, Manfred Schmidt, Christ<strong>of</strong>von Kalle, Marc Lavigne, Odile Y Cohen-Haguenauer930Genotoxic Potential <strong>of</strong> scAAV Vectors in Mouse LiverLucia E. Rosas, Kimberly Zaraspe, Douglas M. McCarty931In vivo Persistence <strong>of</strong> ex vivo Expanded CCR7+CD45RA+ T <strong>Cell</strong>sUsing a RetroNectin Stimulation MethodTatsuji Enoki, Nobuko Muraki, Akiko Kato, Mitsuko Ideno, Junichi Mineno932Self-inactivating ASLV Based Vectors for the <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> X-CGDKerstin B. Kaufmann, Julia Suerth, Christian Brendel, Axel Schambach, ChristopherBaum, Manuel GrezThursday, May 20 th


104<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryFriday, May 21 stFriday, May 21, 2010Plenary Abstract Session 300Plenary Abstract Session8:00 AM - 10:00 AMRoom: Marriott Ballroom Salons 1, 2 & 3283 8:05 AMTherapeutic Transgene Expression in Patient-Specific iPS <strong>Cell</strong>s afterSelection <strong>of</strong> “Safe Harbors” for Lentiviral Vector IntegrationEirini P. Papapetrou, Gabsang Lee, Nirav Malani, Isabelle Riviere, Frederic D. Bushman,Lorenz Studer, Michel Sadelain.284 8:25 AMA Novel Lentiviral Expression System Reveals Patchwork ColonyFormation during Reprogramming to Induced Pluripotent Stem <strong>Cell</strong>sEva Warlich, Johannes Kuehle, Tobias Cantz, Martijn Brugman, Tobias Maetzig, MelanieGalla, Christine Völkel, Adam Filipczyk, Stephan Halle, Hannes Klump, Hans Schöler,Timm Schroeder, Christopher Baum, Axel Schambach.285 8:45 AMTargeted Genomic Modification and Inactivation <strong>of</strong> the CCR5 <strong>Gene</strong>Using PNA-Induced Homologous RecombinationErica B. Schleifman, Ranjit S. Bindra, Jean Leif, Nicole Ali McNeer, Jacob Del Campo,Faye Rogers, Pradeep Uchil, Leonard D. Shultz, W. Mark Saltzman, Priti Kumar, DaleGriener, Peter M. Glazer.286 9:05 AMDetermining the Specificity <strong>of</strong> Zinc Finger Nucleases by AnalyzingInsertion Site Distribution <strong>of</strong> Integrase Defective Lentiviral Vectors(IDLV) Trapped into DNA Double Strand BreaksRichard Gabriel, Angelo Lombardo, Anne Arens, Pietro Genovese, Michael C. Holmes,Philip D. Gregory, Manfred Schmidt, Luigi Naldini, Christ<strong>of</strong> von Kalle.287 9:25 AMSystemic Administration <strong>of</strong> Dendrimer- and Aptamer-siRNAConjugates Efficiently Suppresses HIV-1 InfectionJiehua Zhou, Preston C. Neff, Xiaoxuan Liu, Jane Zhang, Haitang Li, Piotr Swiderski, LingPeng, Ramesh Akkina, John J. Rossi.288 9:45 AMCASPALLO: Phase I Clinical Trial <strong>of</strong> Allodepleted T <strong>Cell</strong>s Transducedwith Inducible Caspase 9 Suicide <strong>Gene</strong> after Haploidentical Stem <strong>Cell</strong>TransplantationAntonio Di Stasi, Siok K. Tey, Yuriko Fujita, Russell Cruz, Caridad Martinez, AlanaKennedy-Nasser, George Carrum, Carlos A. Ramos, Stephen M. Gottschalk, KarinStraath<strong>of</strong>, Lin Yu-Feng, Enli Liu, April Durett, Bambi Grilley, Hao Liu, Barbara Savoldo,Eric Yvon, Teresita Lopez, Oumar Diouf, Deborah Lyon, Adrian Gee, Ellen Vitetta, JohnSchindler, Catherine M. Bollard, Robert A. Krance, David M. Spencer, Gianpietro Dotti,Cliona M. Rooney, Helen E. Heslop, Malcolm K. Brenner.Oral Abstract Session 320Clinical <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> Oral Abstract Session2:00 PM - 4:00 PMRoom: Marriott Ballroom Salons 2 & 3289 2:00 PMTherapeutic DNA Vaccination Followed by Standard-<strong>of</strong>-Care <strong>Therapy</strong>in Patients with Chronic Hepatitis C: A Rapid Clearance <strong>of</strong> ViremiaMatti Sallberg, Lars Frelin, Helmut M. Diepolder, Maria C. Jung, Iacob Mathiesen,Michael Fons, Gustaf Ahlen, Margaret Chen, Ola Weiland.290 2:15 PMSustained Alpha-Sarcoglycan <strong>Gene</strong> Expression in LGMD2D Following<strong>Gene</strong> TransferJerry R. Mendell, Louise R. Rodino-Klapac, Xiomara Rosales-Quintero, Brian D. Coley,Gloria Galloway, Sarah Lewis, Vinod Malik, Christopher J. Shilling, Barry J. Byrne,Thomas Conlon, Katherine J. Campbell, William G. Bremer, Christopher M. Walker, ZarifeSahenk, K. Reed Clark.291 2:30 PMAdvanced <strong>Gene</strong>ration RGD-Modified Oncolytic Adenovirus ICOVIR-7in the Treatment <strong>of</strong> Cancer Refractory to Other ModalitiesPetri Nokisalmi, Sari Pesonen, Sophie Escutenaire, Mari Raki, Vincenzo Cerullo, RamonAlemany, Juanjo Rojas, Maria Rajecki, Lotta Kangasniemi, Anna Kanerva, Timo Joensuu,Laura Ahtiainen, Akseli Hemminki.292 2:45 PMFinal Results <strong>of</strong> Phase I Trial <strong>of</strong> GMCSF <strong>Gene</strong>-TGFb2 Antisense <strong>Gene</strong>Autologous Tumor <strong>Cell</strong> (TAG) Vaccine in Advanced CancerJohn Nemunaitis, Padmasini Kumar, Neil Senzer, Joseph Kuhn, Minal Barve, JairoOlivares, Gerald Edelman, Cynthia Bedell, Yang Yu, Mitchell Magee, Beena O. Pappen,Gladice Wallraven, Alex W. Tong, Phillip B. Maples.293 3:00 PMAdministration <strong>of</strong> Tumor-Specific Cytotoxic T Lymphocytes Engineeredto Resist TGF-ß to Subjects with EBV-Associated LymphomasCatherine M. Bollard, Gianpietro Dotti, Stephen Gottschalk, Enli Liu, Andrea M. Sheehan,George Carrum, Hao Liu, Chung-Che Chang, Adrian P. Gee, Malcolm K. Brenner, Helen E.Heslop, Cliona M. Rooney.294 3:15 PMEfficient Engraftment <strong>of</strong> MGMTP140K <strong>Gene</strong>-Modified CD34+ <strong>Cell</strong>sFollowing Nonmyeloablative BCNU Conditioning in Patients withGlioblastomaJennifer E. Adair, Brian C. Beard, Alexander Spence, Jason Rockhill, Daniel L. Silbergeld,Robert Rostomily, Pamela Becker, Marc Chamberlain, Maciej Mrugala, Hans-Peter Kiem.295 3:30 PMThymic Renewal and Anti-Leukemic Effect in Adults afterHaplodentical Transplantation and Suicide <strong>Gene</strong> <strong>Therapy</strong>Luca Vago, Giacomo Oliveira, Maddalena Noviello, Corrado Soldati, Domenico Ghio,Roberto Nicoletti, Immacolata Brigida, Alessandro Aiuti, Maria Teresa Lupo Stanghellini,Jacopo Peccatori, Attilio Bondanza, Katharina Fleischhauer, Claudio Bordignon, FabioCiceri, Chiara Bonini.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010105Abstract DirectoryFriday, May 21, 2010Oral Abstract Session 320 I — continuedClinical <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> Oral Abstract Session2:00 PM - 4:00 PMRoom: Marriott Ballroom Salons 2 & 3296 3:45 PMUpdate on a Clinical <strong>Gene</strong> <strong>Therapy</strong> Trial for Adenosine DeaminaseDeficient Severe Combined Immune Deficiency (ADA-SCID)Kit L. Shaw, Yeong “Christopher” Choi, Linda Muul, Robert Sokolic, Denise A. Carbonaro,Alan Ikeda, Monika Smogorzewska, Jayashree Jagadeesh, Elizabeth Garabedian, AmiShah, Neena Kapoor, Michael S. Hershfield, Fabio Candotti, Donald B. Kohn.Oral Abstract Session 321RNA Virus Vectors2:00 PM - 4:00 PMRoom: Virginia Suite297 2:00 PMSensitive In Vivo Models To Assess the Risk <strong>of</strong> InsertionalMutagenesis in the Liver upon Vector Systemic DeliveryMarco Ranzani, Daniela Cesana, Manfred Schmidt, Francesca Sanvito, FabrizioBenedicenti, Cynthia Bartholomä, Maurilio Ponzoni, Alessio Cantore, Lucia Sergi Sergi,Claudio Doglioni, Christ<strong>of</strong> VonKalle, Luigi Naldini, Eugenio Montini.298 2:15 PMSystemic Injection in a Tumor Prone Mouse Unravels the ResidualGenotoxicity <strong>of</strong> Self-Inactivating Lentiviral VectorsDaniela Cesana, Marco Ranzani, Cynthia Bartholomä, Manfred Schmidt, FabrizioBenedicenti, Monica Volpin, Christ<strong>of</strong> VonKalle, Luigi Naldini, Eugenio Montini.299 2:30 PMSpecific Insertions <strong>of</strong> Zinc Finger Domains into Gag-Pol YieldsEngineered Retroviral Vectors with Selective Integration PropertiesKwang-il Lim, Ryan Klimczak, Julie Yu, David Schaffer.300 2:45 PMThe D4Z4 Insulator Protects Retrovirus Vectors from Silencing inStem <strong>Cell</strong>sSylvie Rival-Gervier, Susan Anderson, Mandy Lo, Peter Pasceri, James Ellis.301 3:00 PMExpression <strong>of</strong> the LEDGF Integrase Binding Domain (IBD) SeverelyRestricts HIV-1 Replication and Drives Evolution <strong>of</strong> PhenotypicallyDistinctive Integrase MutantsDyana T. Saenz, Anne Meehan, Mary E. Peretz, Eric M. Poeschla.302 3:15 PMTransient and Highly Efficient Delivery <strong>of</strong> Sleeping BeautyTransposase and Flp Recombinase mRNA in Target <strong>Cell</strong>s Via RT-Deficient Retroviral ParticlesMelanie Galla, Axel Schambach, Tobias Maetzig, Niels Heinz, Zsuzsanna Izsvak, ZoltanIvics, Christopher Baum.303 3:30 PMErasable Non-Integrating RNA Replicon Vectors for <strong>Gene</strong>ration <strong>of</strong>Immaculate iPS <strong>Cell</strong>s Free from Transgenes and Sequence-InterferenceHiroshi Ban, Noemi Fusaki, Toshiaki Tabata, Koichi Saeki, Akihiro Iida, Makoto Inoue,Mamoru Hasegawa.304 3:45 PMSelf-Inactivating Alpharetroviral Vectors with a Split-PackagingDesignJulia D. Sürth, Tobias Mätzig, Melanie Galla, Ute Modlich, Christopher Baum, AxelSchambach.Oral Abstract Session 322AAV Vectors: Vector Biology2:00 PM - 4:00 PMRoom: Marriott Ballroom Salon 1305 2:00 PMDissecting rAAV Integration Frequency in Non-Human Primates byDeep SequencingAli Nowrouzi, Magalie Penaud-Budloo, Christine Kaeppel, Anne Arens, Caroline Le Guiner,Adrien Leger, Karim Zaoui, Christian Weber, Richard O. Snyder, Christ<strong>of</strong> von Kalle,Philippe Moullier, Manfred Schmidt.306 2:15 PMNon-Canonical Integrating AAV Vectors as a Safe Tool for the Future<strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong>Leszek Lisowski, Zhongya Wang, Ashley Lau, Feijie Zhang, Markus Grompe, Mark A.Kay.307 2:30 PMInteraction with Homologous Recombination Pathways Leads To Loss<strong>of</strong> Functional Recombinant AAV (rAAV) GenomesMarcela P. Cataldi, Douglas M. McCarty.308 2:45 PMHigh-Throughput Sequencing <strong>of</strong> AAV Vector Integrants To AssessPotential for GenotoxicityHojun Li, Nirav Malani, Shari R. Hamilton, Alexander Schlachterman, Giulio Bussadori,Shyrie E. Edmonson, Rachel Shah, Federico M. Mingozzi, J. F. Wright, Valder R. Arruda,Frederic D. Bushman, Katherine A. High.309 3:00 PMA Self-Complimentary rAAV Vector Genome but Not Variations in theCapsid Sequence Magnifies the Innate Immune Response FollowingLiver Directed DeliveryAshley T. Martino, Masataka Suzuki, Brendan Lee, Roland W. Herzog.310 3:15 PMA Proteomic Analysis <strong>of</strong> the Protein Composition in Recombinant AAVVector PreparationsBiao Dong, Xunbao Duan, Fayaz Khazi, Kirk Chu, Jihong Dai, Jenni A. Snyder, Hui Lu,Weidong Xiao.311 3:30 PMDownregulation <strong>of</strong> DNA-Damage Response Proteins in PostmitoticTissues Correlates with Increased AAV TransductionJasmina Lovric, Miguel Mano, Lorena Zentilin, Mauro Giacca.Friday, May 21 st


106<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryFriday, May 21 stFriday, May 21, 2010Oral Abstract Session 322 — continuedAAV Vectors: Vector Biology2:00 PM - 4:00 PMRoom: Marriott Ballroom Salon 1312 3:45 PMInnate Immune Response towards Adeno-Associated Viral (AAV)Vectors in Human Liver Non-Parenchymal <strong>Cell</strong>sMarianna Hoesel, Mathias Broxtermann, Hanna Janicki, Knud Esser, Silke Arzberger,Miriam John von Freyend, Dirk Stabenow, Percy Knolle, Michael Hallek, Ulrike Protzer,Hildegard Buening.Oral Abstract Session 323<strong>Gene</strong> <strong>Therapy</strong> for Lysosomal Storage Diseases andOther <strong>Gene</strong>tic Disorders2:00 PM - 4:00 PMRoom: Delaware Suite313 2:00 PMTowards a Safe and Effective <strong>Gene</strong> <strong>Therapy</strong> Approach for Globoid <strong>Cell</strong>LeukodystrophyIlaria Visigalli, Silvia Ungari, Bernhard Gentner, Sabata Martino, Daniela Ungaro, LetterioS. Politi, Lucia Sergi Sergi, Ubaldo Del Carro, Angelo Quattrini, Luigi Naldini, AlessandraBiffi.314 2:15 PMMesenchymal Stem <strong>Cell</strong> <strong>Therapy</strong> for the Twitcher Mouse Model <strong>of</strong>Krabbe’s DiseaseCynthia Ripoll, Mette Flaat, Jessica Eiermann, Jeanne Perkins, Brittni Scruggs, MarjorieMcCants, Michelle Eagle, William Wimley, Cynthia Trygg, Jeffrey Gimble, Bruce Bunnell.315 2:30 PMTherapeutic Efficacy <strong>of</strong> Hematopoietic Stem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong> forHurler Type 1 MucopolysaccharidosisStefania Delai, Ilaria Visigalli, Letterio S. Politi, Carmela Di Domenico, Emanuela Mrak,Riccardo Brambilla, Federica Cerri, Daniela Ungaro, Merel Stok, Francesca Sanvito,Ubaldo Del Carro, Alessandro Rubinacci, Katherine P. Ponder, Angelo Quattrini, Paola DiNatale, Luigi Naldini, Alessandra Biffi.316 2:45 PMTherapeutic Potential <strong>of</strong> <strong>Gene</strong>tically Modified Neural Stem <strong>Cell</strong>s(NSCs) in a Mouse Model <strong>of</strong> Globoid <strong>Cell</strong>s Leukodystrophy (GLD)Margherita Neri, Alessandra Ricca, Silvia Ungari, Ilaria Visigalli, Alessandra Biffi, Ilaria DiGirolamo, Sabata Martino, Lucia Sergi Sergi, Luigi Naldini, Angela Gritti.317 3:00 PMAdeno-Associated Virus and Tetanus Toxin Fragment C Based Vectorsfor <strong>Gene</strong> <strong>Therapy</strong> in the CNSCarlos Gay-Antaki, John H. Wolfe.318 3:15 PMLineage Specific Lentiviral Mpl Expression Rescues HematopoieticStem <strong>Cell</strong> Defects in Mpl-/- MiceDirk Heckl, Daniel C. Wicke, Johann Meyer, Axel Schambach, Johanna Krause, MatthiasBallmaier, Christopher H. Baum, Ute Modlich.319 3:30 PMSafety <strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong> for ThalassemiaRiccardo Mezzadra, Emanuela Roselli, Claudia Rossi, Francesca Tiboni, GiacomoMandelli, Sarah Marktel, Fulvio Mavilio, Giulietta Maruggi, Marco Andreani, GuidoLucarelli, Giuliana Ferrari.320 3:45 PMLong-Term Phenotypic Correction <strong>of</strong> Skin by Fetal <strong>Gene</strong> Transfer in aLethal Murine Model <strong>of</strong> Epidermolysis BullosaMasayuki Endo, Philip W. Zoltick, Antenata Radu, Jiang Qiujie, Chihiro Matsui, Peter M.Marinkovich, Jouni Uitto, Alan W. Flake.Oral Abstract Session 324Physical Methods <strong>of</strong> Delivery2:00 PM - 4:00 PMRoom: Wilson ABC321 2:00 PMBile Duct Infusion <strong>of</strong> Plasmid DNA Mediated High Level <strong>of</strong> TransgeneExpression in Rat LiverXuan Jiang, Derek Leong, Deng Pan, Hai-Quan Mao.322 2:15 PMImage-Guided, Computer-Controlled Hydrodynamic <strong>Gene</strong> Delivery toLiver Results in Therapeutic Level <strong>of</strong> Human Alpha-1 Antitrypsin inPigs and DogsKenya Kamimura, Takeshi Suda, Guisheng Zhang, Dexi Liu.323 2:30 PMTargeting Magnetic Nanoparticles Impregnated with Adenovirus toStented Arteries Using Uniform-Field Induced Magnetization in theRat Carotid Model <strong>of</strong> RestenosisMichael Chorny, Ilia Fishbein, Richard Adamo, Robert J. Levy.324 2:45 PMUltrasound-Activated Microbubbles for <strong>Gene</strong> Transfer: In VivoEfficiency and Confocal Microscopy Real Time IntracellularInvestigationsAnthony Delalande, Patrick Midoux, Michiel Postema, Chantal Pichon.325 3:00 PMLong-Term <strong>Gene</strong> Expression after Hydrodynamic Delivery <strong>of</strong> SleepingBeauty Transposons to Canine Liver Using Balloon CathetersPerry B. Hackett, Myra Urness, Jason B. Bell, Erik R. Olson, Elena L. Aronovich, R. ScottMcIvor.326 3:15 PMEfficient Microbubble- and Ultrasound-Mediated Plasmid DNATransfer into a Specific Rat Liver Lobe Via a Targeted Injection andAcoustic Exposure Using a Novel Ultrasound SystemShuxian Song, Misty L. Noble, Liping Chen, Samuel S. Sun, Andrew A. Brayman, CarolH. Miao.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010107Abstract DirectoryFriday, May 21, 2010Oral Abstract Session 324 — continuedPhysical Methods <strong>of</strong> Delivery2:00 PM - 4:00 PMRoom: Wilson ABC327 3:30 PMA Novel Prototype Device for Electroporation-Enhanced DNA VaccineDelivery to Both Skin and MuscleFeng Lin, Jay McCoy, Xuefei Shen, Janess Mendoza, Kate E. Broderick, Jian Yan, SteveKemmerrer, David B. Weiner, Niranjan Y. Sardesai.328 3:45 PMSafety and Feasibility <strong>of</strong> Transvenous Limb Perfusion with Saline inHuman Muscular DystrophyWilliam Powers, Zheng Fan, Keith Kocis, Robert Valley, Manisha Chopra, James Howard,Jr, Joseph Muenzer.Oral Abstract Session 325Cancer-Targeted <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>2:00 PM - 4:00 PMRoom: Thurgood Marshall W334 3:15 PMTumor-Selectivity <strong>of</strong> Replication-Competent Retrovirus (RCR) VectorsRegulated by Prostate-Specific Promoters and Cytotoxic Effect <strong>of</strong>Suicide <strong>Gene</strong> <strong>Therapy</strong> Mediated by RCR Vectors In VitroShuichi Kamijima, Takahiro Kimura, Kazunori Haga, Kei Hiraoka, Akihito Inagaki,Christopher R. Logg, Chinghai Kao, Bernard H. Bochner, Noriyuki Kasahara.335 3:30 PMTranslational Phase I Study in Advanced Cancer Patients <strong>of</strong> Dual-Function Autologous (FANG) Vaccine Incorporating BifunctionalshRNA furin and GMCSF Transgene ExpressionNeil Senzer, Padmasini Kumar, Joseph Kuhn, Minal Barve, Cynthia Bedell, Yang Yu, AlexW. Tong, Phillip B. Maples, John Nemunaitis.336 3:45 PMEfficient <strong>Gene</strong> Transfer in Quiescent Healthy and Cancer B <strong>Cell</strong>s by aMeasles Virus gp Pseudotyped LVCecilia Frecha, Caroline Costa, Camille Levy, Didier Negre, Andrea Maisner, Stephen J.Russell, Nicholas Rachinel, Gilles Salles, Francois-Loic Cosset, Els Verhoeyen.Oral Abstract Session 326Hematologic/Immunologic: Basic2:00 PM - 4:00 PMRoom: Maryland Suite329 2:00 PMA <strong>Gene</strong>tically Engineered Multifunctional Vector for HER2 TargetedCancer Suicide <strong>Gene</strong> <strong>Therapy</strong>Yuhua Wang, Arash Hatefi.330 2:15 PMComparative Analysis <strong>of</strong> Enzyme and Pathway Engineering Strategiesfor Suicide <strong>Gene</strong> <strong>Therapy</strong> ApplicationsAdam J. Johnson, Andressa Ardiani, Kinta Serve, Marilyn Sanchez-Bonilla, Margaret E.Black.331 2:30 PMConditional and Effective <strong>Gene</strong> Knock-Down in ProangiogenicMacrophages To Disrupt Tumor AngiogenesisFerdinando Pucci, Roberta Mazzieri, Bernhard Gentner, Luigi Naldini, Michele De Palma.332 2:45 PM<strong>Cell</strong> Fusion between Mesenchymal Stem <strong>Cell</strong>s and Cancer <strong>Cell</strong>s: AMechanism <strong>of</strong> Cancer ProgressionYan Xiao, Dirk H. Hondmann, Carla J. Aldrich, Chenzhong Kuang.333 3:00 PMNext-<strong>Gene</strong>ration Lentiviral <strong>Cell</strong> Fate Control <strong>Gene</strong> <strong>Therapy</strong> Based onEngineered Variants <strong>of</strong> Human Deoxycytidine KinaseAnton Neschadim, Arnon Lavie, Takeya Sato, Daniel H. Fowler, Vladimir Ponomarev,Jeffrey A. Medin.337 2:00 PMSafely Exploiting Hematopoeitic Stem and Progenitor <strong>Cell</strong> SpecificmicroRNAs To Target Transgene Expression to the DifferentiatedProgenyBernhard Gentner, Alice Giustacchini, Francesco Boccalatte, Massimo Saini, Giulia Schira,Luigi Naldini.338 2:15 PMTherapeutic Fetal Hemoglobin Production in Erythroid Progeny <strong>of</strong>Normal and b-Thalassemic Human CD34 + <strong>Cell</strong>s Using LentiviralVectors Encoding g-Globin or shRNA To Down Modulate theTranscriptional Repressor BCL11AAndrew Wilber, Vijay G. Sankaran, Phillip W. Hargrove, Yoon-Sang Kim, EleniPapanikolaou, Nicholas P. Anagnou, Stuart H. Orkin, Arthur W. Nienhuis, Derek A.Persons.339 2:30 PMPotentially Therapeutic Levels <strong>of</strong> Fetal Hemoglobin in ErythroidProgeny <strong>of</strong> Normal and b-Thalassemic CD34 + <strong>Cell</strong>s FollowingLentivirus-Mediated Delivery <strong>of</strong> a Zinc-Finger TranscriptionalActivator Targeting the g-Globin <strong>Gene</strong> PromotersAndrew Wilber, Ulrich Tschulena, Phillip W. Hargrove, Yoon-Sang Kim, Carlos F. Barbas,III, Derek A. Persons, Arthur W. Nienhuis.340 2:45 PMDevelopment <strong>of</strong> an S/MAR Based Episomal Vector <strong>of</strong> a Specific Zinc-Finger Activator That Mediates Gamma-Globin <strong>Gene</strong> Activation, forthe <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> HemoglobinopathiesEleana Stavrou, Eleni Lagadinou, Nikolas Zoumbos, Alexandros Spyridonidis, CarlosBarbas, III, Kenneth Peterson, Aglaia Athanassiadou.Friday, May 21 st


108<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryFriday, May 21 stFriday, May 21, 2010Oral Abstract Session 326— continuedHematologic/Immunologic: Basic2:00 PM - 4:00 PMRoom: Maryland Suite341 3:00 PMEngineered Human Regulatory T <strong>Cell</strong>s Expressing Lentiviral PDL1under <strong>Cell</strong> Fate Control Prevent Lethal Xenogeneic GVHDShoba Amarnath, James Wang, Courtney Mangus, James Riley, Bruce Levine, Carl June,Jeffrey Medin, Daniel Fowler.342 3:15 PMCorrection <strong>of</strong> Murine Hemophilia A Following NonmyeloablativeTransplantation <strong>of</strong> Hematopoietic Stem <strong>Cell</strong>s Engineered To Expressa Bioimproved Human FVIII Using a Safety-Augmented, B-LymphoidLineage-Restricted Lentiviral VectorAli Ramezani, Sara Karandish, Lynnsey A. Zweier-Renn, Teresa S. Hawley, Robert G.Hawley.343 3:30 PMLentiviral Vectors Containing an Endogenous Btk Promoter RestoreB and Myeloid Lineage Expression and Function in a Murine Model <strong>of</strong>X-Linked AgammaglobulinemiaHannah M. Kerns, Byoung Y. Ryu, Brigid V. Stirling, Blythe D. Sather, AlexanderAstrakhan, Stephanie Humblet-Baron, Denny Liggitt, David J. Rawlings.344 3:45 PMEarly Gestational <strong>Gene</strong> Transfer <strong>of</strong> IL-10 by Lentiviral Vector CanPrevent Arthritis in a Murine ModelJessica L. Roybal, Masayuki Endo, Antoneta Radu, Carlyn Todorow, Philip W. Zoltick,Alan W. Flake.Poster Session II6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-SouthRNA Virus Vectors I345Expression Pr<strong>of</strong>ile and Epigenetic Landscape Influence in a <strong>Cell</strong>-Specific Fashion Integration Site Selection and In Vivo Distribution <strong>of</strong>MLV-Vector in PatientsLuca Biasco, Alessandro Ambrosi, Danilo Pellin, Cynthia Bartholomae, ImmacolataBrigida, Maria Grazia Roncarolo, Clelia Di Serio, Christ<strong>of</strong> Von Kalle, Manfred Schmidt,Alessandro Aiuti.346High-Definition Mapping <strong>of</strong> Retroviral Integration Sites Defines theFate <strong>of</strong> Allogeneic T <strong>Cell</strong>s after Donor Lymphocyte InfusionAlessandra Recchia, Claudia Cattoglio, Danilo Pellin, Giulietta Maruggi, Serena KimiPerna, Alessandro Ambrosi, Clelia Di Serio, Nirav Malani, Frederic D. Bushman, CynthiaBartholomae, Manfred Schmidt, Chiara Bonini, Fulvio Mavilio.347Genome Wide Characterization <strong>of</strong> Insertion Pr<strong>of</strong>iles <strong>of</strong> IntegrationDeficient Lentiviral Vectors Using Two-Sided Non-Restrictive andStandard LAM-PCRWei Wang, Cynthia C. Bartholomae, Anne Arens, Richard Gabriel, Hanno Glimm, Christ<strong>of</strong>von Kalle, Manfred Schmidt.348Multiple Pathways Facilitate Transduction <strong>of</strong> Quiescent HumanCD34+ <strong>Cell</strong>s by HIV-1 VectorsCathy X. Wang, Charles DeRossi, Bruce E. Torbett.349Targeted Lentiviral <strong>Gene</strong> Delivery to <strong>Cell</strong>s Expressing c-fms or IL-2ReceptorAnil Chandrashekran, Giedre Milinkeviciute, Rosana Cardoso, Fouzia Abdulrasul, Shao-AnXue, Nick Dibb, Carol Readhead, Colin Casimir.350Regulation <strong>of</strong> Transgene Expression In Vivo by ProteasomalDegradationKhalid Tai, Fredrik Gussing, Christina Isaksson, Luis Quintino, Mari Iwamoto, Tom J.Wandless, Cecilia Lundberg.351Clonal Analysis Allows Characterization and Risk Assessment<strong>of</strong> Viral-<strong>Cell</strong>ular Fusion Transcripts <strong>Gene</strong>rated by TranscriptionalTermination Read-Through in Keratinocytes Transduced with Self-Inactivating Retroviral VectorsDavid Almarza, Fernando Larcher, Rodolfo Murillas, Fulvio Mavilio.352Evaluation <strong>of</strong> Cytokine Effects on Lentiviral Transduction <strong>of</strong> HumanCD34 + <strong>Cell</strong>sNaoya Uchida, Kareem N. Washington, Matthew M. Hsieh, John F. Tisdale.353Towards Quantitative High Throughput Clonality Screening:Redefining the Frontier <strong>of</strong> Retroviral Integration AnalysisTroy B. Hawkins, Aparna Jasti, Jessica Dantzer, Nate Van Dusen, Brandon Downing,Sean Mooney, Mary C. Dinauer, Keithanne Mockaitis, Kenneth G. Cornetta.354Development <strong>of</strong> a pCL20c-Backbone Based HIV Lentivirus Vector ThatAchieves Efficient Functional Correction <strong>of</strong> Neutrophils Arising fromTransduced CD34+ Hematopoietic Progenitor <strong>Cell</strong>s <strong>of</strong> Patients withX-Linked Chronic Granulomatous DiseaseUimook Choi, Robert E. Throm, Elizabeth M. Kang, Suk See DeRavin, Narda Theobald,Gilda M. Linton, Brian P. Sorrentino, Mary C. Dinauer, John T. Gray, Harry L. Malech.355Packaging Heterologous Retroviral Vectors into HIV-1 Viral ParticlesAdam S. Cockrell, Henriette van Praag, Nicholas Santistevan, Thomas J. McCown, BonitaBlake, Hong Ma, John Olsen, Tal Kafri.356High Throughput Vector Integration Analysis with SeqMap 2.0Troy B. Hawkins, Jessica Dantzer, Brandon Peters, Mary Dinauer, Keithanne Mockaitis,Sean Mooney, Kenneth G. Cornetta.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010109Abstract DirectoryFriday, May 21, 2010Poster Session II— continued6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-SouthRNA Virus Vectors I357‘Self’ Marker CD47 on Viral EnvelopesNisha Sosale, Richard K. Tsai, Irena Ivanovska, Philip W. Zoltick, Dennis E. Discher.358Lentiviral Microarrays for High-Throughput and Real-Time Monitoring<strong>of</strong> <strong>Gene</strong> Expression DynamicsJun Tian, Stella Allimperti, Pedro Lei, Stelios T. Andreadis.359Fibrin-Conjugated Pseudotyped Lentivirus for <strong>Cell</strong>-Controlled andSpatially Localized <strong>Gene</strong> Delivery on Microarrayed SurfacesRoshan Padmashali, Pedro Lei, Stelios Andreadis.360Improved Transduction Efficiency <strong>of</strong> Human Amniotic MesenchymalStem <strong>Cell</strong>s Using Optimized Lentiviral VectorsF. Pisano, P. Danieli, E. Cervio, C. Ciuffreda, M. Roccio, G. Viarengo, M. Gnecchi.361Purification <strong>of</strong> Lentivirus Vector by Iodixanol Self-Forming DensityGradientJulia Transfiguracion, Sven Ansorge, Rosa Tran, Amine Kamen.AAV Vectors II362rAAV-Mediated Delivery <strong>of</strong> Micro RNA Scavengers Leads to Efficientand Stable Knock-Down <strong>of</strong> Cognate Micro RNAs, Upregulation <strong>of</strong>Their Natural Target <strong>Gene</strong>s and Phenotypic Changes in MiceJun Xie, Jennifer Broderick, Qing Xie, Hongwei Zhang, Ran He, Qin Su, Phillip D.Zamore, Guangping Gao.363Activation <strong>of</strong> the Non-Canonical NF-kB Pathway by AAV2 Vectors:Implications in Immune Response and <strong>Gene</strong> <strong>Therapy</strong>Giridhara R. Jayandharan, George Aslanidi, George Q. Perrin, Li Zhong, Angela E. Rivers,Ashley T. Martino, Roland W. Herzog, Arun Srivastava.364Multifunctional Peptide Ligands for AAV Capsid RecognitionNagesh Pulicherla, Jana L. Phillips, Aravind Asokan.365The Rous Sarcoma Virus Promoter Carried by a rAAV Vector IsResistant to DNA Methylation in Primate Muscle and LiverAdrien Léger, Magalie Penaud-Budloo, Michael L. Nickerson, Caroline Le Guiner, PhilippeMoullier, Richard O. Snyder.366The Hybrid AAV2.5 Vector Transduces Neurons Bordering the Thirdand Fourth Ventricles after Lateral Ventricular Infusion in RatsDawn E. Bowles, Chengwen Li, Joseph E. Rabinowitz, R. Jude Samulski, Thomas J.McCown.367Differential Lymphatic Transport <strong>of</strong> Chimeric and Naturally OccurringAAV StrainsJana L. Phillips, Roland Cheung, Chengwen Li, Aravind Asokan.368Optimizing Liver Transduction <strong>of</strong> the Low Seroprevalent AAV rh32.33Luk H. Vandenberghe, Ru Xiao, James M. Wilson.369Role <strong>of</strong> a <strong>Cell</strong>ular Chaperone Protein, FKBP52, in Uncoating <strong>of</strong> AAV2VectorsMengxin Li, Giridhara R. Jayandharan, Baozheng Li, Chen Ling, Wenqin Ma, ArunSrivastava, Li Zhong.370Strategies To Achieve Enhanced Transduction <strong>of</strong> Primary HumanDendritic <strong>Cell</strong>s by AAV2 Vectors and Reduced Cytotoxic T <strong>Cell</strong>Response to Viral Capsids and Transgene ProductsGeorge Aslanidi, George Q. Perrin, Giridhara R. Jayandharan, Angela E. Rivers, Li Zhong,Arun Srivastava.371Optimizing Capsid Composition for the Production <strong>of</strong> RecombinantAdeno-Associated Virus Using the Baculovirus Expression SystemKirsten Erger, David Markusic, Ramaz Geguchadze, Barry J. Byrne, Sergei Zolotukhin.372<strong>Gene</strong>tically Re-Engineered AAV Rep78 Identifies Sequence-RestrictedInhibitory Regions Affecting Adenoviral ReplicationVarsha Sitaraman, Patrick Hearing, Steffen Mueller, Charles Ward, Dmitri Gnatenko,Steven Skiena, Eckard Wimmer, Wadie F. Bahou.373Improved and Persistent Expression with Novel AAV Serotypes forNeonatal <strong>Gene</strong> <strong>Therapy</strong>Chuhong Hu, Fides D. Lay, Joseph Springer, Ronald W. Busuttil, Gerald S. Lipshutz.374Optimization <strong>of</strong> AAV Vectors for Improved Therapeutic ProteinExpression in the Canine Models for Duchenne Muscular DystrophyAlock Malik, Marilyn Mitchell, Andrew Mead, Mike Petrov, Hansell Stedman.375rAAV1 Infect Mouse Bone Marrow-Derived Dendritic <strong>Cell</strong>s throughReceptors with a2,3 and a2,6 Sialic AcidsYuanqing Lu, Sihong Song.376Using AAV Vector as Carrier for Biosensor: Detecting microRNA’sActivities by Reverse Infection <strong>of</strong> AAV VectorsWenhong Tian, Xiaoyan Dong, Gang Wang, Shuping Tan, Xiaobing Wu.Friday, May 21 st


110<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryFriday, May 21 stFriday, May 21, 2010Poster Session II— continued6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-South377Clearance <strong>of</strong> Herpes Simplex Virus by Orthogonal ManufacturingProcess <strong>of</strong> Recombinant Adeno-Associated Virus (rAAV) VectorsGuo-jie Ye, David R. Knop, Darby L. Thomas, Lijun Wang, Jeffrey D. Chulay.Adenovirus and Other DNA Virus Vectors II378Production <strong>of</strong> a Novel Class <strong>of</strong> Human Polyomavirus JC Vectors UsingBaculovirus Expression SystemKirsten E. Erger, Oleg Gorbatyuk, George Aslanidi, Ramaz Geguchadze, Barry J. Byrne,Sergei Zolotukhin.379Induction <strong>of</strong> <strong>Cell</strong> Surface Integrins in Lymphomas and PotentialApplications to <strong>Gene</strong> <strong>Therapy</strong>Ann Marie O’Neill, Farruk M. L. Kabir, David T. Curiel, Laura Timares, Bruce F. Smith.380FX-Binding Ablated Ad5 Do Not Transduce Hepatocytes but ExhibitSplenic Uptake at High DosesRaul Alba, Angela C. Bradshaw, Robert A. McDonald, Lynda Coughlan, Alan L. Parker,Ashley M. Miller, Nico van Rooijen, Stuart A. Nicklin, Andrew H. Baker.381Targeting Adenovirus <strong>Cell</strong> Entry Via Tumor-Associated EphA2 byInsertion <strong>of</strong> a Ligand Peptide into Different Loops <strong>of</strong> the Short Fiber<strong>of</strong> Ad41Michael Behr, Dirk M. Nettelbeck.382Induction <strong>of</strong> a Prostate Specific Promoter and Replication Kinetics byp21/Waf-1 shRNA Armed CRAdNaser Uddin Hoti, Sadaf Mustafa, Wasim Chowdhury, Ronald Rodriguez.383Capacitance-Based Real Time Monitoring <strong>of</strong> Virus TransductionJoung-Woo Choi.384Inhibition <strong>of</strong> PD-1 Greatly Augments Therapeutic Effect <strong>of</strong> Suicide<strong>Gene</strong> <strong>Therapy</strong> for Gastrointestinal CancersHae-hyun Seo, Mi-Hyang Lee, In-Hoo Kim, Seong-Phil Shin, Kyung-Hee Chun, Sang-JinLee.385Method for Fast and Easy Adoption <strong>of</strong> First-<strong>Gene</strong>ration AdenoviralVectors to High-Capacity Adenoviral VectorsMartin A. Hausl, Christina Rauschhuber, Richard Voigtlander, Zsolt Ruzsics, Anja Ehrhardt.386PAUF Targeting RNA-Based <strong>Gene</strong> <strong>Therapy</strong>Sue Kyung Kim, Eun Ok Kim, Hyun Mi Dae, Ju Young Moon, Kyun Heo, In-Hoo Kim,Yun-Hee Kim.387Combining Oncolytic Adenoviruses and Antibody <strong>Therapy</strong>Dominik E. Dorer, Roland Kontermann, Dirk M. Nettelbeck.388Direct Injection <strong>of</strong> HDAd into the Liver Is as Effective but Less ToxicThan Intravenous Injection for Crigler-Najjar Syndrome <strong>Gene</strong> <strong>Therapy</strong>Franesco Vetrini, Nathan Grove, Donna Palmer, Philip Ng, Nicola Brunetti-Pierri.389Dual-Expression Adenoviral Vectors as Vaccine CarriersXiangyang Zhou, Heng Chen, Dongming Zhou, Ang Bian, Hildegund C. Ertl.390Optimized Adenovirus Targeting through Virus-Displayed PeptideLibrary Screening in a Prostate Cancer ModelPing Wu, Tarana A. Kudrolli, Wasim H. Chowdhury, Minzhi M. Liu, Ronald Rodriguez,Shawn E. Lupold.391Human Adenovirus Type 40 Vector Engineering for Intestinal MucosaTargetingSatoshi Yamasaki, Eric Brown, Julia Davydova, Selwyn M. Vickers, Masato Yamamoto.Chemical and Molecular Conjugates II392Macropinocytosis Is the Major Pathway Leading to Transfection for aCharge-Reversal AmphiphileXiao-Xiang Zhang, Phil Allen, Mark W. Grinstaff.393Ornithine-Based Cationic Lipids: Highly Charged DendriticHeadgroups, New Lipoplex Phases and Degradable LipidsKai K. Ewert, Alexandra Zidovska, Rahau S. Shirazi, Joel Quispe, Bridget Carragher,Clinton S. Potter, Cyrus R. Safinya.394Nanoparticle-Mediated Delivery <strong>of</strong> <strong>Gene</strong>s and Imaging Agents to theBrainStephen L. Hart, Alison Bienemann, Steven Gill, Panos Kyrtatos, Andrew S. Lowe, MarkLythgoe, Edward A. White, Michele J. Writer.395In Vitro and In Vivo Transfection Efficiency <strong>of</strong> DNA NanoparticlesMade with Histidinylated lPEI and Plasmid DNA Containing NFkBDNA Binding SequencePatrick Midoux, Gilles Breuzard, Ludivine Billiet, Emilie Bertrand, Tony Le Gall, TristanMontier, Pierre Lehn, Hervé Chéradame, Chantal Pichon, Philippe Guégan.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010111Abstract DirectoryFriday, May 21, 2010Poster Session II— continued6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-SouthChemical and Molecular Conjugates II396Intracellular Trafficking and Decondensation Kinetics <strong>of</strong> ChitosanpDNAPolyplexesMarc Thibault, Surendra Nimesh, Lavertu Marc, Buschmann Michael.397Dual Function Effect on PEI-Mediated <strong>Gene</strong> Delivery by DiphtheriaToxin-Derived Ligand and Fusogenic DomainsTakeshi Nagasaki, Shinji Kakimoto, Hideki Azuma.398Tumour <strong>Therapy</strong> with Listeria Monocytogenes Vector-Mediated DNADeliveryJan P. Van Pijkeren, David Morrissey, Michelle Cronin, Simon Rajendran, Gerald C.O’Sullivan, Cormac G. M. Gahan, Mark Tangney.399Efficient <strong>Gene</strong> Transfer to Umbilical Cord Blood-Derived MesenchymalStem <strong>Cell</strong>s Mediated by MR Imageable Inorganic-Organic HybridConjugateYong Sook Kim, In-Kyu Park, Jin Sook Kwon, Myung Ho Jeong, Youngkeun Ahn.400Histidine-Rich Amphipathic Peptides Promote Efficient Delivery <strong>of</strong>Nucleic Acids into Mammalian <strong>Cell</strong>sAntoine Kichler, A. James Mason, Christian Leborgne, Daniel Scherman, BurkhardBechinger.401MHC Class-I Mediated Antigen Presentation by a Non-Viral T-MENDVector Expressing a High Luciferase Activity in Murine DerivedDendritic <strong>Cell</strong>s (JAWS-II)Sharif M. Shaheen, Hidetaka Akita, Takashi Nakamura, Shiroh Futaki, Atsushi Yamashita,Ryo Katoono, Nobuhiko Yui, Hideyoshi Harashima.402Evaluation <strong>of</strong> Polymer-DNA Nanoparticles for <strong>Gene</strong> Transfer toCardiac MusclesXiaohong Qiu, Lepeng Zeng, Jon Urban, Alena Nikolskaya, Sarah Audet.403Chemical Manipulation <strong>of</strong> AAV Tissue TropismEric D. Horowitz, Jana L. Phillips, Aravind Asokan.404Versatile Self-Assembling Nanoparticles for Enhanced siRNA Deliveryinto Mammalian <strong>Cell</strong>sGulam H. Dar, Madhusudhana R. Nalam, Vijaya Gopal.405Novel L-Lysine Reducible Copolymers for Efficient Nonviral <strong>Gene</strong>DeliveryMohamed M. Ismail Nounou, Samuel Chung, Kalyopy Emmanouil, Thu Pham, ZhuoyuanLu, Malavosklish Bikram.406JL1-Directed Small Interfering RNA Delivery Using Anti-JL1 Immuno-Sendai Viroplexes to T-Leukemic <strong>Cell</strong>sJung Seok Kim, Yeon Jyung Lee, Jin Ee Baek, Sang Il Park, Hwa Yeon Jeong, HongSung Kim, Keun Sik Kim, Sang Soon Yoon, Kyeong Cheon Jung, Hyung Geun Song,Yong Serk Park.407Antibody-Directed Drug Delivery Using Protein A-Based CarriersNahyun Kim, Hong-Seok Ban, Priti Kumar, Sang-Kyung Lee.<strong>Gene</strong>tic and Metabolic Diseases <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II408Lentiviral Vector-Transduced Hematopoietic Stem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong>for CystinosisBrian A. Yeagy, Frank Harrison, Matthew V. Tadlock, Daniel R. Salomon, StephanieCherqui.409Creation and Rescue <strong>of</strong> a Severe Hyperammonaemic Phenotype inOrnithine Transcarbamylase Deficient spf ash Mice Using rAAV andshRNASharon C. Cunningham, Cindy Y. Kok, Allison P. Dane, Kevin H. Carpenter, Ian E.Alexander.410Long Term Correction <strong>of</strong> Biochemical and Neurological Abnormalities<strong>of</strong> Metachromatic Leukodystrophy Model Mice by Systemic NeonatalInjection <strong>of</strong> Serotype 9 AAV VectorNoriko Miyake, Koichi Miyake, Nagisa Asakawa, Motoko Yamamoto, Takashi Shimada.411Neural Stem <strong>Cell</strong>s as a Therapeutic for Krabbe’s DiseaseMette Flaat, Cynthia Ripoll, Jimin Yu, Jessica Eiermann, Jeanne Fisher-Perkins, BrittniScruggs, Helen Paige Leonard, Amy Lin, Marjorie McCants, Michelle Eagle, WilliamWimley, Bruce A. Bunnell.412AAV8 Mediated Expression <strong>of</strong> LDLR AmelioratesHypercholesterolemia and Regresses Pre-Existing Atherosclerosis ina Humanized Mouse Model <strong>of</strong> Familial HypercholesterolemiaSadik H. Kassim, Luk H. Vandenberghe, Christian Hinderer, Dawn Marchadier, PeterL. Bell, Hui Li, Debra Cromley, Aisha Wilson, Hongwei Yu, James M. Wilson, Daniel J.Rader.413Widespread Enzymatic Correction <strong>of</strong> CNS Tissues by a SingleIntracerebral Injection <strong>of</strong> Therapeutic Lentiviral Vector inLeukodystrophy Mouse ModelsAnnalisa Lattanzi, Claudio Maderna, Margherita Neri, Ilaria Di Girolamo, Sabata Martino,Aldo Orlacchio, Mario Amendola, Luigi Naldini, Angela Gritti.Friday, May 21 st


112<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryFriday, May 21 stFriday, May 21, 2010Poster Session II— continued6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-South<strong>Gene</strong>tic and Metabolic Diseases <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II414<strong>Gene</strong> <strong>Therapy</strong> Ex Vivo in the ADA-Deficient Mouse: IncreasedSurvival and Efficacy with Short-Term Enzyme Replacement <strong>Therapy</strong>Post <strong>Gene</strong> <strong>Therapy</strong>Denise A. Carbonaro, Xiangyang Jin, Donald B. Kohn.415Towards Targeted Transgene Integration in Human Hematopoietic andInduced Pluripotent Stem <strong>Cell</strong>sHongjie Wang, Ying Liu, Michael C. Holmes, Philip D. Gregory, Andre Lieber.416Optimization <strong>of</strong> the Expression Cassette Using an AAV2/8 BasedVector for the Treatment <strong>of</strong> Ornithine Transcarbamylase DeficiencyLili Wang, Huan Wang, Hiroki Morizono, Peter L. Bell, David Jones, Mark L. Batshaw,James M. Wilson.417<strong>Gene</strong>ration <strong>of</strong> Early AMD Mouse Model by Induction <strong>of</strong> OxidativeStressSoojung Gibson, Jian W. Liu, Yun Le, Wiliam W. Hauswirth, Alfred S. Lewin.418Increased Expression <strong>of</strong> Human Glucocerebrosidase Activity in BoneMarrow <strong>of</strong> Gaucher Mice Following Macrophage Mediated <strong>Gene</strong><strong>Therapy</strong>Arlene T. Lim, Edward I Ginns.419<strong>Cell</strong> Sheet Engineering toward Bioengineering <strong>of</strong> Functional Neo-IsletsTakahiro Saito, Kazuo Ohashi, Hir<strong>of</strong>umi Shimizu, Rie Utoh, Kazuya Ise, MasayukiYamato, Teruo Okano, Mitsukazu Gotoh.420Liver-Specific Over-Expression <strong>of</strong> PGC-1a Does Not Induce a DiabeticPhenotypeLuke Roode, Chunping Qiao, Ruhang Tang, Jiangjang Jiang, Juan Li, Xiao Xiao.421Promoter Selection for AAV Vector-Mediated Factor IX Expression inSkeletal MuscleAkihiro Kume, Hiroya Yagi, Hiroaki Mizukami, Masashi Urabe, Tomonori Tsukahara, AkiraIshiwata, Jun Mimuro, Seiji Madoiwa, Tsukasa Ohmori, Yoichi Sakata, Keiya Ozawa.422Development <strong>of</strong> HIV-1 Based Lentiviral Vectors Expressing Pre-Trans-Splicing Molecules (PTMs) for Liver-Directed TherapiesMadaiah Puttaraju, S. Gary Mansfield, Jun Wang, Nikolay Korokhov, Jenice D’Costa,Ziping C. Chen, Katherine Radd, Gerard J. McGarrity, Laurent M. Humeau.764 (Moved from Poster Session III)Microporous Polymer Scaffolds for Islet TransplantationRomie F. Gibly, Lonnie D. Shea.Cardiovascular <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>423AAV-Mediated Intramyocardial VEGF-B167 Expression Exerts aPotent Cardioprotective Effect in Small and Large Animal Models <strong>of</strong>Myocardial Infarction and Heart FailureLorena Zentilin, Vincenzo Lionetti, Uday Puligadda, Silvia Moimas, Serena Zacchigna,Martino Pepe, Mohammed Mamdani, Khaled Qanud, Xiabin Xu, Thomas H. Hintze,Fabio A. Recchia, Mauro Giacca.424Osteopontin Enhances the Angiogenic Potential <strong>of</strong> EndothelialProgenitor <strong>Cell</strong>sErin E. Vaughan, Angela Duffy, Timothy O’Brien.425Arginine and Tetrahydrobiopterin Synergistically Potentiate theAntirestenotic Effect <strong>of</strong> Vascular <strong>Gene</strong> <strong>Therapy</strong> with Inducible NitricOxide SynthaseIlia Fishbein, Peter Sobolewki, Richard F. Adamo, Ivan S. Alferiev, Ariel Fishbein, MarinaBakay, Michael Chorny, Robert J. Levy.426Intra-Myocardial Administration <strong>of</strong> ACRX-100 Promotes CardiacBenefit and Improves Angiogenesis in a Porcine Model <strong>of</strong> HeartFailureJoseph M. Pastore, Timothy J. Miller, Rahul A. Aras, Marc S. Penn.427Strategies for Promoting Survival <strong>of</strong> Transplanted Mesenchymal Stem<strong>Cell</strong>s in a Myocardial Infarction ModelLisa McGinley, Daniel O’Toole, Aoife Duffy, Xizhe Chen, Alessia Stocca, Jill McMahon,Timothy O’Brien.428Systemic Delivery <strong>of</strong> Interleukin-10 by an AAV Vector AttenuatesObesity-Related Glomerulopathy in Zucker Fatty RatsManabu Ogura, Masashi Urabe, Akira Onishi, Takayuki Ito, Tomonori Tsukahara, HiroakiMizukami, Akihiro Kume, Eiji Kusano, Keiya Ozawa.429AAV/B7H1-<strong>Gene</strong> Delivery Lowers Th1 Cytotoxic T LymphocyteResponse In Vitro and Atherogenesis In VivoMaohua Cao, Junaid A. Khan, Yong Liu, Paul L. Hermonat.430Dose-Dependent Angiogenesis by Human and Murine VEGF IsSpecies-SpecificEdin Mujagic, Roberto Gianni-Barrera, Marianna Trani, Lorenz Gürke, Michael Heberer,Thomas Wolff, Andrea Banfi.431AAV-Sema3A-Mediated Recruitment <strong>of</strong> Neuropilin-1-ExpressingMononuclear <strong>Cell</strong>s (NEMs) Contributes to Vessel Stabilization andNormalizationAlessandro Carrer, Serena Zacchigna, Silvia Moimas, Lucia Pattarini, Lorena Zentilin,Mauro Giacca.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010113Abstract DirectoryFriday, May 21, 2010Poster Session II— continued6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-SouthCardiovascular <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>432Identification <strong>of</strong> AAV6 as Most Efficient Vector for Transvascular<strong>Gene</strong> Transfer into Porcine Myocardium Based on an In Vitro Modelfor Prediction <strong>of</strong> the Cardiac <strong>Gene</strong> Transfer PerformanceJan Ksienzyk, Philip W. Raake, Philipp Schlegel, Changguang Shan, Christophe Weber,Jürgen A. Kleinschmidt, Hugo A. Katus, Oliver J. Müller.433Percutaneous Transendocardial Adminstration <strong>of</strong> AAV6 and AAV8Vectors to Rhesus Macaques Results in Highly Efficient and GlobalCardiac <strong>Gene</strong> TransferGuangping Gao, Meg M. Sleeper, Lawrence Bish, Lan Sun, You Lu, Jiachuan Duan,Chunyan Hu, Li Wang, H. Lee Sweeney.434<strong>Gene</strong> Transfer <strong>of</strong> VEGF165, HGF, Urokinase and Their CombinationsUsing Novel PC4W Plasmid for Induction <strong>of</strong> Vasculogenesis in MiceHind Limb Ischemia ModelPavel Makarevich, Zoya Tsokolaeva, Alexander Shevelev, Igor Rybalkin, EugeniyShevchenko, Irina Beloglazova, Tatyana Vlasik, Elena Parfyonova, Vsevolod A. Tkachuk.435PEGylation <strong>of</strong> Helper-Dependent Adenoviral Vector-MediatedLong-Term Expression <strong>of</strong> Human Apolipoprotein A-I ReducesAtherosclerosis in LDLR Deficient MiceEleonora Leggiero, Dalila Astone, Piyanuch Wonganan, Cristina Mazzaccara, GiuseppeLabruna, Luciana Sacchetti, Francesco Salvatore, Maria Croyle, Lucio Pastore.436AAV/IL13-<strong>Gene</strong> Delivery Lowers Atherogenesis in LDLR KO MiceJunaid A. Khan, Maohua Cao, Yong Liu, Paul L. Hermonat.437AAV/hSTAT3-<strong>Gene</strong> Delivery Lowers Atherosclerosis in LDLR KO Miceon High Cholesterol DietJunaid A. Khan, Maohua Cao, Bum-Yong Kang, Jawahar L. Mehta, Paul L. Hermonat.438Intramuscular Delivery <strong>of</strong> ACRX-100 Enhances Angiogenesis inRabbits with Hind Limb IschemiaTimothy J. Miller, Joseph M. Pastore, Rahul A. Aras, Marc S. Penn.439A Comparison <strong>of</strong> Three Liposomal Formulations for <strong>Gene</strong> Deliveryfrom Coronary Artery StentsSandra Ganly, Sean O. Hynes, Faisal Sharif, Karl McCullagh, Valerie Barron, Jim Crowley,Peter McHugh, Timothy O’Brien, Udo Greiser.440Efficient and Localized <strong>Gene</strong> Transference in Rat Heart byElectroporationLeonardo P. Carvalho, Eduardo G. Yasumura, Vivian Somoto, Priscila M. A. Denapoli,Flavia H. Silva, Valderez B. Valero, Hans F. Dohmann, Christina M. Takiya, Sang W. Han.Ophthalmic Disorders and Technological Advances441<strong>Gene</strong> Delivery <strong>of</strong> Erythropoietin Protects Retinal Ganglion <strong>Cell</strong>s inDBA/2J Glaucomatous MiceTimothy A. Sullivan, Eldon E. Geisert, Tonia S. Rex.442Pan-Retinal Secretion <strong>of</strong> GDNF Following Intravitreal Transduction <strong>of</strong>Müller Glia Slows Photoreceptor Loss in Retinal DegenerationDeniz Dalkara, Kathleen D. Kolstad, Meike Visel, Ryan R. Klimczak, Karen Guerin, DavidV. Schaffer, John G. Flannery.443AAV-Mediated <strong>Gene</strong> Supply <strong>of</strong> Non-Erythropoietic ErythropoietinDerivatives Improves Photoreceptor Morphology and Function inModels <strong>of</strong> Retinal Degenerative DiseasesPasqualina Colella, Carolina Iodice, Umberto Di Vicino, Alberto Auricchio.444Early Indicators <strong>of</strong> Biological Efficacy in a Phase 1/2 <strong>Gene</strong> <strong>Therapy</strong>Trial for Leber Congenital Amaurosis (LCA)Shalesh Kaushal, Margaret Humphries, Elena Filippova, George Asdourian, RadouilTzekov, Michael Stalvey, Terrence Flotte.445Positive Responses in a Mouse Model <strong>of</strong> Leber Congenital Amaurosisafter Treatment with Compacted DNA NanoparticlesKiichiro Okano, Tadao Maeda, Linas Padegimas, Tomasz H. Kowalczyk, Sharon M.Oette, Christopher R. Gedeon, Ozge Sesenoglu-Laird, Susannah L. Hyatt, Karla A. Dines,Elena Tyr, Nikia L. Beal, Robert C. Moen, Krzyszt<strong>of</strong> Palczewski, Mark J. Cooper.446Adeno-Associated Virus Mediated <strong>Gene</strong> <strong>Therapy</strong> with a RhodopsinRibozyme for Treatment <strong>of</strong> Autosomal Dominant Retinitis Pigmentosa(Adrp)Haoyu Mao, Marina S. Gorbatyuk, William W. Hauswirth, Alfred S. Lewin.447POD DNA Nanoparticles Rescue an Adult Mouse Model <strong>of</strong> RetinalDegenerationSarah P. Read, Siobhan M. Cashman, Rajendra Kumar-Singh.448Protection Against Laser-Induced Choroidal NeovascularizationFollowing Delivery <strong>of</strong> an Adenovirus Expressing a Soluble Form <strong>of</strong>Human CD59 to Murine RetinaSiobhan M. Cashman, Kasmir Ramo, Rajendra Kumar-Singh.449Dose Finding Study in Galactosialidosis Mice Using scAAV2/8-LP1-ppca VirusHuimin Hu, Elida Gomero, Eric Bonten, John Gray, Christopher Calabrese, Arthur Nienhuis,Alessandra d’Azzo.Friday, May 21 st


114<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryFriday, May 21 stFriday, May 21, 2010Poster Session II— continued6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-SouthOphthalmic Disorders and Technological Advances450Characterization <strong>of</strong> 12 AAV Vectors for Intravascular Delivery ToTarget CNS and Detarget Non-CNS Tissues by miRNA Regulation:Implications in Treatment <strong>of</strong> Canavan DiseaseHongwei Zhang, Bin Yang, Jun Xie, Qing Xie, Qin Su, Ran He, Miguel Sena-Esteves,Zuoshang Xu, Guangping Gao.451Efficient Expression <strong>of</strong> Smn from Integration-Deficient LentiviralVectorsSherif O. Ahmed, Hugo Peluffo, Edmund Foster, Natalia Lago, Lawrence Moon, ThomasHutson, Ping Yip, Klaus Wanisch, Stephen B. McMahon, Rafael J. Yáñez-Muñoz.452Efficacy <strong>of</strong> an Adenovirus Vector/Cocaine Conjugate-Based Anti-Cocaine Vaccine in Shielding the Brain from Systemically AdministeredCocaineMartin J. Hicks, Bishnu P. De, Jonathan B. Rosenberg, Jesse T. Davidson, Amira Y.Moreno, Kim D. Janda, Neil R. Hackett, Stephen M. Kaminsky, Stefan Worgall, RonaldG. Crystal.453<strong>Gene</strong>tically Engineered Suppressor <strong>Cell</strong>s Target CNS and InhibitActivated Effector T-<strong>Cell</strong>sMoa Fransson, Elena Piras, Hao Wang, Ida Rasmusson, Katarina LeBlanc, Eva Brittebo,Angelica S. I. Loskog.Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II454First <strong>Gene</strong>ration AAVmicroutrophin Vector Infused into the IsolatedPelvic Limb <strong>of</strong> a Canine Model for Duchenne Muscular DystrophyMihail Petrov, Marilyn Mitchell, Alock Malik, Andy Mead, Frederick Balzer, Leonard Su,Jacqueline Farag, Benjamin Kozyak, Kapil Gopal, Charles Bridges, Janet Bogan, MartinChilders, Joe Kornegay, Hansell Stedman.455Characterization <strong>of</strong> A20 as an Inhibitor <strong>of</strong> NF-kB Activation inDystrophic MiceRakshita Charan, Paula R. Clemens.456Plasticity <strong>of</strong> Skeletal Muscle <strong>Cell</strong>s during Muscle Injury and Repair –A Dedifferentiation StudyXiaodong Mu, Hairong Peng, Johnny Huard, Yong Li.457Increased Muscle Regeneration in Hindlimb and Diaphragm <strong>of</strong> mdxMice Treated with AAV9 Mini-Dystrophin and Octalysine-NEMOBinding Domain PeptideDaniel P. Reay, Geno Raggi, Bing Wang, Xiao Xiao, Paul D. Robbins, Paula R. Clemens.458External, Non-Invasive Monitoring <strong>of</strong> Progressive CardiorespiratoryDysfunction in a Canine Model <strong>of</strong> DMDAndrew Mead, Alock Malik, Mihail Petrov, Martin Childers, Janet Bogan, JosephKornegay, Hansell Stedman.459The Polyproline Site in Hinge 2 Influences the Functional Capacity <strong>of</strong>Truncated DystrophinsGlen B. Banks, Luke M. Judge, James M. Allen, Jeffrey S. Chamberlain.460Combining <strong>Gene</strong> and Stem <strong>Cell</strong> <strong>Therapy</strong> in the Treatment <strong>of</strong>DysferlinophatiesDaniele Parolini, Claire Navarro, Andrea Farini, Mirella Meregalli, Marzia Belicchi, PaolaRazini, M. Krahn, Louis Garcia, Nicolas Lévis, Yvan Torrente.461Deletion <strong>of</strong> Functional Domains <strong>of</strong> Dp116 Leads to AlteredSarcolemmal Expression and Subcellular Localization When Expressedin Skeletal MuscleAndrea L. Arnett, Guy L. Odom, Kristy Boyle, Eric Finn, Jeffrey S. Chamberlain.462A New Therapeutic Approach for Duchenne Muscular Dystrophy:Restoration <strong>of</strong> the Dystrophin Reading Frame with a MeganucleaseJacques P. Tremblay, Pierre Chapdelaine, Christophe Pichavant, Joel Rousseau, FrédéricPâques.463Regulatory Cassettes Derived from the Slow Troponin I <strong>Gene</strong>Confer High-Level Expression in Skeletal Muscle after <strong>Gene</strong> TransferMediated by Helper-Dependent Adenovirus and Recombinant AAVRénald Gilbert, Mehdi Bendjelloul, Yué Zeng, Claude Guérin, Nancy Larochelle, GeorgeKarpati, Bernard Massie, Josephine Nalbantoglu.464<strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> Muscular Dystrophy with Engineered Cd133+ <strong>Cell</strong>sAndrea Farini, Mirella Meregalli, Daniele Parolini, Marzia Belicchi, Simona Maciotta,Paola Razini, Joao da Silva Bizario, Luis Garcia, Nereo Bresolin, Yvan Torrente.465Improved Regenerative Capacity <strong>of</strong> Adult Stem <strong>Cell</strong>s Isolated fromInjured Skeletal Muscle <strong>of</strong> MouseXiaodong Mu, Ian Bellayr, Haiying Pan, Yong Li.466Sarcolemmal nNOS Anchoring Reveals a Functional Differencebetween Dystrophin and UtrophinYongping Yue, Dejia Li, Luke Judge, Akshay Bareja, Yi Lai, Kay E. Davies, Jeffrey S.Chamberlain, Dongsheng Duan.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010115Abstract DirectoryFriday, May 21, 2010Poster Session II— continued6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-SouthMusculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II467Detailed Physical Mapping <strong>of</strong> Deletion Breakpoints in the GSHP DogModel for DMDAlock Malik, Acong Xu, Jesse Chen, Andrew Mead, Martin Childers, Janet Bogan, ScottSchatzberg, Joe Kornegay, Hansell Stedman.468Lentiviral Vector Mediated Delivery <strong>of</strong> Full-Length Dystrophin for<strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> Muscular DystrophyEn Kimura, Katsuhisa Uchio, Tomohiro Suga, tatsuya Koide, Yuji Uchida, Yasushi Maeda,Satoshi Yamoshita, Jeffrey S. Chamberlain, Makoto Uchino.Cancer – Immunotherapy II469Elimination <strong>of</strong> Cancer Stem <strong>Cell</strong>s with <strong>Gene</strong>tically Engineered T <strong>Cell</strong>sExpressing a Stage Specific Embryonic Antigen-1 (SSEA1) ChimericAntigen ReceptorAntonina V. Kurtova, Juan Vera, An Lu, Adham Bear, Aaron E. Foster.470Targeting Cancer Stem <strong>Cell</strong>s in OsteosarcomaAlexia Ghazi, Nino Rainusso, Vita Salsman, Helen Heslop, Steven Gottschalk, NabilAhmed.471CMV-Specific HER2-Redirected T <strong>Cell</strong>s for the AdoptiveImmunotherapy <strong>of</strong> GlioblastomaAlexia Ghazi, Vita Salsman, Yvonne Kew, Donald Shaffer, Suzanne Powell, RobertGrossman, Michael Scheurer, Ann Leen, Catherine Bollard, Helen Heslop, StephenGottschalk, Nabil Ahmed.472Combining mTor Inhibitors with Rapamycin-Resistant T <strong>Cell</strong>s: A Two-Pronged Approach to Tumor EliminationLeslie E. Huye, Barbara Savoldo, Gianpietro Dotti, Matthew H. Wilson, Cliona M. Rooney.473Evaluating the Combination <strong>of</strong> 5’Azacytidine and MAGE-A4-SpecificT <strong>Cell</strong> <strong>Therapy</strong> as a Potential Immunotherapeutic Strategy forRefractory Hodgkin’s LymphomaConrad R. Cruz, Ulrike Gerdemann, Jessica A. Shafer, Ann M. Leen, Tamara Tripic, TerzahM. Horton, Anas Younes, Cliona M. Rooney, Helen E. Heslop, Catherine M. Bollard.474The iCD40.MyD88 Dendritic <strong>Cell</strong> (DC) <strong>Gene</strong> Switch Leads to PotentAnti-Tumor <strong>Therapy</strong>Natalia Lapteva, Priyadharshini Narayanan, Mamatha Seethammagari, Kevin M. Slawin,David M. Spencer.475Emozolomide Inhibits the Migration <strong>of</strong> Immune <strong>Cell</strong>s into the BrainTumor Microenvironment and Their Systemic Expansion, but Exertsa Synergistic Anti-Tumor Effect When Combined with CombinedConditional Cytotoxic/Immune-<strong>Gene</strong> <strong>Therapy</strong>Maria G. Castro, Marianela Candolfi, Kader Yagiz, Kurt M. Kroeger, David Foulad,Garbielle Ahlzadeh, Matthew Tesarfreund, Pedro R. Lowenstein.476TCR Engineered and In Vitro Expanded T Lymphocytes Recapitulatethe Properties <strong>of</strong> Central Memory T <strong>Cell</strong>sShicheng Yang, Zhili Zheng, Ling Zhang, Steven A. Rosenberg, Richard A. Morgan.477Phase I Safety and Immunogenicity <strong>of</strong> HPV 16 and 18 DNA VaccinesDelivered Via ElectroporationJian Yan, Panyupa Pankhong, Xuefei Shen, Mary Giffear, Jessica Lee, Drea Harris,Deanna Diaz, Amir Khan, Mark Bagarazzi, Jean Boyer, David B. Weiner, Niranjan Y.Sardesai.478Improving Adoptive T <strong>Cell</strong> <strong>Therapy</strong> by Transduction <strong>of</strong> TIL with aVector That Mediates the Inducible Expression <strong>of</strong> IL-12 in the TumorMicroenvironmentLing Zhang, Sid P. Kerkard, Shicheng Yang, Zhili Zheng, Zhiya Yu, Nichaolas P. Restifo,Steven A. Rosenberg, Richard A. Morgan.479Robust and Sustained Ex Vivo Expansion <strong>of</strong> NK <strong>Cell</strong>s with ArtificialAPCs Bearing Membrane-Bound IL-21 Is Associated with a DistinctPhenotype and Transcriptional Pr<strong>of</strong>ileCecele J. Denman, Vladimir Senyukov, Lisa M. Kopp, Maria Lima de Silva, Harjeet Singh,Lenka Hurton, Simon Olivares, Sourindra Maiti, Mary H. Huls, Richard E. Champlin,Laurence J. N. Cooper, Dean A. Lee.480STAT3-Inactivated Dendritic <strong>Cell</strong> Vaccination Could Induce StrongerAnti-Tumor Immune ResponsesHidetoshi Sumimoto, Tomoko Iwata-Kajihara, Hiroyuki Mizuguchi, Kiyoshi Takeda,Yutaka Kawakami.481Combination <strong>Therapy</strong> for Melanoma Using Virotherapy and AlternateTLR Stimulating AgonistsRosa Diaz, Marta Compte-Grau, Elizabeth Ilett, Phonphimon Wongthida, Feorillo Galivo,Timothy Kottke, Jill Thompson, Alan Melcher, Richard Vile.482Bid Is a Key Factor for Eliciting the Antiproliferative Activity <strong>of</strong> IFN-aMediated by TRAILTakaya Tsuno, Josef Mejido, Tongmao Zhao, Terry Phillips, Kathryn Zoon.483Oral Adeno-Associated Virus-neu (AAV-neu) Immunotherapy Inhibitsthe Growth <strong>of</strong> neu+ Murine Breast CancerJason C. Steel, Giovanni Di Pasquale, Charmaine A. Ramlogan, Caroline Morris, John A.Chiorini, John C. Morris.Friday, May 21 st


116<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryFriday, May 21 stFriday, May 21, 2010Poster Session II— continued6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-SouthCancer – Immunotherapy II484Tumor-Specific Antitumor Activity <strong>of</strong> mIL-10 Revealed byHydrodynamic Tail Vein Injection <strong>of</strong> pCMV-mIL Plasmids in Tumor-Bearing MiceJianfeng Li, Li Hai, Shulin Li, Dexi Liu.485Liver Specific, Inducible, AAV-Mediated IL12 Delivered Resulted ina Strong Anti Tumoral Inmune Response in a Murine Model <strong>of</strong> LiverMetastasisLucia Vanrell, Laura Blanco, Pedro Berraondo, Marianna Di Scala, Astrid Paneda, VictorBaldim, Jesus Prieto, Gloria González-Aseguinolaza.486AdCD40L <strong>Gene</strong> <strong>Therapy</strong> Enhances the Efficacy <strong>of</strong> Chemotherapy inExperimental Bladder CancerLina S. E. Liljenfeldt, Lisa H. Christiansson, Katerina Gkirtzimanaki, Angelica S. I. Loskog,Aristides G. Eliopoulos.487Xenograft Expanded FANG Autologous Tumor <strong>Cell</strong> VaccineDevelopment and Manufacturing for Clinical UsePhillip B. Maples, Padmasini Kumar, Yang Yu, Beena O. Pappen, David Monsma,Stephanie Scott, Dawna Dylewski, Craig Webb, Neil Senzer, John Nemunaitis.Cancer-Oncolytic Viruses II488Optimization <strong>of</strong> Measles Virus Infected Mesenchymal Stem <strong>Cell</strong>Carriers for Clinical Testing in Patients with Recurrent Ovarian CancerKah-Whye Peng, Emily K. Mader, Greg Butler, Marla Sommerfield, Sean C. Dowdy, AllanB. Dietz.489Expression <strong>of</strong> Interferon Alpha from an Oncolytic Adenovirus VectorDecreases Toxicity but Retains the Anti-Tumor Efficacy <strong>of</strong> the VectorJacqueline F. Spencer, Debanjan Dhar, Elena V. Shashkova, Karoly Toth, William S. M.Wold.490Measles Virus Encoding H. pylori Neutrophil-Activating Protein forTreatment <strong>of</strong> Metastatic Breast CancerIanko D. Iankov, Cory Allen, Stephen J. Russell, Mark J. Federspiel, Evanthia Galanis.491Carrier <strong>Cell</strong>s Delivery <strong>of</strong> Inducible Oncolytic Adenovurs for BrainTumorHiroshi Nakashima, E. A. Chiocca.492Optimizing the Genomic Stability, Transgene Expression, andTherapeutic Efficacy <strong>of</strong> Replication-Competent Retrovirus Vectors forProdrug Activator <strong>Gene</strong> TransferKei Hiraoka, Logg R. Christopher, Akihito Inagaki, Hiroshi Matsumoto, Oscar Diago,Omar Perez, Joan Robbins, Harry Gruber, Douglas Jolly, Noriyuki Kasahara.493<strong>Gene</strong> <strong>Therapy</strong> for Head and Neck Squamous Carcinoma <strong>Cell</strong>s byIAI.3B Promoter-Driven Oncolytic Adenovirus-Infected Carrier <strong>Cell</strong>sKatsuyuki Hamada, Ting Zhang, Koh-ichi Nakashiro, Hiroyuki Hamakawa.494Evaluation <strong>of</strong> Monocytes and Cancer <strong>Cell</strong>s as Carriers for OncolyticAdenoviruses in Immune-Competent Models <strong>of</strong> Pancreatic Cancer inSyrian HamstersEva Garcia-Aragoncillo, Sergia Bortolanza, Maria Buñuales, Jesus Prieto, RubenHernandez-Alcoceba.495Combination <strong>Therapy</strong> for Breast Cancer Using Oncolytic Viruses andHistone Deacetylase InhibitorsHongxun Wang, Stephen J. Russell, Jian Qiao.496Combination Oncolytic VirotherapyTimothy Kottke, Jill Thompson, Konstantin Doronin, Sonia Guedan Carrio, William Wold,Ramon Alemany, Richard Vile.497Conditionally Replicative Adenovirus with an Adenoviral Major LatePromoter-Driven Imaging Cassette Predicts In Vivo Anti-Tumor EffectJulia Davydova, Tatyana Gavrikova, Eric J. Brown, Xianghua Luo, David T. Curiel, SelwynM. Vickers, Masato Yamamoto.498Susceptibility <strong>of</strong> Neuroblastoma Primary Tumor-Initiating <strong>Cell</strong>s toOncolytic Herpes Simplex Virus Is Determined by Nectin-1 ExpressionPin-Yi Wang, Mark A. Currier, Margaret H. Collins, Betsy A. DiPasquale, Loen Hansford,David Kaplan, Sean Lawler, E. Antonio Chiocca, Timothy P. Cripe.499Activity <strong>of</strong> Oncolytic Measles Virus Strains Against Brain Tumor Stem<strong>Cell</strong>sCory Allen, Mark A. Schroeder, Jann N. Sarkaria, Mark J. Federspiel, Stephen J. Russell,Evanthia Galanis.500Replication-Competent Retrovirus Vector-Mediated Suicide <strong>Gene</strong><strong>Therapy</strong> Achieves Significant Therapeutic Efficacy Against HumanMalignant Mesothelioma XenograftsYoshiko Kawasaki, Norie Yamaoka, Nobuyuki Terada, Haruki Okamura, NoriyukiKasahara, Shuji Kubo.501Oncolytic Adenovirus with Somatostatin Motif for Selective Infection<strong>of</strong> Neuroendocrine TumorsDi Yu, Justyna Leja, Berith Nilsson, Magnus Essand.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010117Abstract DirectoryFriday, May 21, 2010Poster Session II — continued6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-SouthCancer-Oncolytic Viruses II502Silencing a p53 Inhibitor in Cancer <strong>Cell</strong>s Increases Killing Potency <strong>of</strong>Oncolytic AdenovirusChristie Vermeulen, Nikki Tol, Winald R. Gerritsen, Victor W. van Beusechem.503Safety <strong>of</strong> Intravenous Ad5-Mda-7, a Potential Anti-Metastatic Agentin Gynecologic CancerKenneth H. Kim, Meredith A. Preuss, Minghui Wang, Anand C. Annan, Justin A. Barnes,Devanand Sarkar, Steven Grant, Paul Dent, Paul B. Fisher, Ronald D. Alvarez, David T.Curiel.504Characterization <strong>of</strong> a Novel Dual-Lock Oncolytic Cadvex-VectorThat Combines Efficient Prostate-Specific Replication with CytokineExpressionBrian D. Hoel, Min Luo, David A. Einfeld, Danher Wang, John Y. Dong.505A Probasin Promoter, Conditionally Replicating Adenovirus thatExpresses the Sodium Iodide Symporter (NIS) for Radiovirotherapy<strong>of</strong> Prostate CancerMiguel A. Trujillo, Michael J. Oneal, Samantha McDonough, Rui Qin, John C. Morris.506Adenovirus Vectors Transductionally Retargeted to Her2/neu forProstate Tumour TreatmentMaria K. Magnusson, Robert Kraaij, Corrina M. A. De Ridder, Leif Lindholm.Cancer-Targeted <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II507The Potential <strong>of</strong> Conditionally Replicating Adenovirus ExpressingTIMP2 for Ovarian Cancer <strong>Therapy</strong>Sherry W. Yang, James J. Cody, Joanne T. Douglas, Ronald D. Alvarez, SelvaranganPonnazhagan.508Mechanisms <strong>of</strong> the Tmpk/AZT-System Bystander Effect; <strong>Cell</strong> FateControl <strong>Gene</strong> <strong>Therapy</strong> for Anti-Cancer TreatmentTakeya Sato, Anton Neschadim, Jun Sukegawa, Teruyuki Yanagisawa, Jeffrey A. Medin.509Truncated CD19 Fused with Mutant Human Tmpk: A Versatile <strong>Cell</strong>Fate Control Safety CassetteMatthew D. Scaife, Sean P. Devine, Anton Neschadim, Natalia A. Pacienza, Daniel H.Fowler, Jeffrey A. Medin.510Muscle Directed Systemic Cancer <strong>Gene</strong> <strong>Therapy</strong> for Lymphoma UsingType 8 AAV Vector Expressing mda-7/IL24Nanya Wang, Bai Fan, Koichi Miyake, Noriko Miyake, Takashi Shimada.511A Liposome Formulation with High Accumulation in Tumor Tissue forTargeted Delivery <strong>of</strong> Nucleic AcidTorben Gjetting, Camilla L. Christensen, Frederik Cramer, Hans S. Poulsen, Thomas L.Andresen.51215-LO-1 Induced Apoptosis Inhibits Tumour Growth and ExtendsSurvival in Rat Glioma ModelAgnieszka Pacholska, Helena Viita, Farizan Ahmad, Anna Hyvärinen, Thomas Wirth,Seppo Ylä-Herttuala.513An Efficient Targeted <strong>Gene</strong> <strong>Therapy</strong> Using Brain Tumor-SpecificPromoterToshio Yawata, Eri Ishida, Yu Kawanishi, Masakazu Tamura, Keiji Shimizu.514Nitric Oxide Synthase One Homologous Recombinant Negative(NOS1 -/-) Mice Are Protected from Thoracic Irradiation InducedSeizures by Esophageal Administration <strong>of</strong> Manganese SuperoxideDismutase Plasmid Liposome Complex (MnSOD-PL)Malolan Rajagopalan, Jean-Claude Rwigema, Brandon Stone, Michael Epperly, JulieG<strong>of</strong>f, Tracy Dixon, Darcy Franicola, Joel Greenberger.515Maternal Manganese Superoxide Dismutase Plasmid/LiposomeComplex (MnSOD-PL) <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> E13 Pregnant Mice ReducesTotal Body Irradiation Induced Growth Retardation in Newborn MiceMichael Epperly, Tracy Dixon, Xichen Zhang, Benjamin Greenberger, Paavani Komanduri,Hong Wang, Joel Greenberger.516Adenovirus-Relaxin <strong>Gene</strong> <strong>Therapy</strong> on Keloids: AttenuatedProliferative Response and Collagen DegradationIl-Kyu Choi, Il-Kyu Lee, Yong Oock Kim, Dong Kyun Rah, Chae-Ok Yun.517Inducing Systemic Anti-Tumor Immune Responses through DirectTumor TargetingG. Campbell Kaynor, Liaomin Peng, Jorge Sanchez-Salazar, Yang-Xin Fu, XinzhongWang.518Angiotensin Type 2 Receptor Mediated Apoptosis <strong>of</strong> Human ProstateCancer <strong>Cell</strong>sHongwei Li, Yanfei Qi, Chengyao Li, Leah N. Braseth, Yongxin Gao, Arseniy E. Shabashvili,Michael J. Katovich, Colin Sumners.519 Abstract Withdrawn520Regulatory Submission Process for <strong>Gene</strong> and <strong>Cell</strong>ular TherapiesGladice Wallraven, Phillip B. Maples, John Nemunaitis.Friday, May 21 st


118<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryFriday, May 21 stFriday, May 21, 2010Poster Session II — continued6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-SouthHematologic and Immunologic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II521Lentiviruses Containing a Modified Ankyrin-1 Promoter DirectUniform, Copy-Number-Dependent Expression <strong>of</strong> a g-Globin <strong>Gene</strong> atTherapeutic LevelsFaith Harrow, Karina Laflamme, Amanda Cline, David M. Bodine.522Non-Myeloablative Conditioning Regimen To Increase Engraftment <strong>of</strong><strong>Gene</strong>-Modified Hematopoietic Stem <strong>Cell</strong>sTeiko Sumiyoshi, Chang J. Lee, Roger Hollis, Francesca Giannoni, Stan F. Louie, Alice F.Tarantal, Donald B. Kohn.523In Vivo Selection <strong>of</strong> Hematopoietic Stem <strong>Cell</strong>s by Expressing aTruncated Epo Receptor (tEpoR)Maria Rosa Lidonnici, Francesca Tiboni, Giacomo Mandelli, Claudia Cattoglio, MargheritaGalletti, Alexis Grande, Fulvio Mavilio, Giuliana Ferrari.524Successful Treatment <strong>of</strong> Canine Leukocyte Adhesion Deficiency Usinga SIN Lentiviral Vector with the PGK Promoter Expressing CanineCD18Michael J. Hunter, Huifen Zhao, Laura M. Tuschong, Tanya H. Burkholder, Derek A.Persons, Dennis D. Hickstein.525Diminished End Organ Damage and Correction <strong>of</strong> Anemia in Sickle<strong>Cell</strong> Mice Treated with Non-Myeloablative Busulfan Conditioning andg-Globin Lentiviral Vector Transduced HSCsTamara I Pestina, Phillip W. Hargrove, Huifen Zhao, Peter Vogel, Derek A. Persons.526Small but Powerful, Predicting the Outcome <strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong> TrialUsing NSG MiceTomoyasu Higashimoto, Elke Grassman, Jose A. Cancelas, Punam Malik.527Development <strong>of</strong> a Bioengineered Recombinant Factor VIII TherapeuticUsing a Lentiviral Production PlatformChristopher B. Doering, Gabriela Denning, Richard Gautney, Boro Dropulic, Lajos Baranyi,Andre J. Roy, Bagirath Gangadharan, Ernest T. Parker, Pete Lollar, H. Trent Spencer.528Transgenic Expression <strong>of</strong> Cytidine Deaminase (CDD) forMyeloprotective PurposesNico Lachmann, Nils Pfaff, Sebastian Brennig, Doreen Lüttge, Tobias Cantz, ChristopherBaum, Axel Schambach, Thomas Moritz.529Development <strong>of</strong> a Pre-Clinical Model for the <strong>Gene</strong> <strong>Therapy</strong> Treatment<strong>of</strong> Wiskott-Aldrich Syndrome (WAS)Alexander Astrakhan, Byoung Ryu, Brigid Stirling, Blythe Sather, Jit Khim, MikhailGaribov, Stephanie Humblet-Baron, Hans Ochs, David J. Rawlings.530Lentiviral <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> Murine Hematopoietic Stem <strong>Cell</strong>s UsingCodon Optimized IL2RG cDNA: A Comparison <strong>of</strong> Multiple PromoterElements and Transplant ConditionsMarshall W. Huston, Niek P. van Til, Trudi P. Visser, Roya Sawari, Shazia Arshad, GerardWagemaker.531Human Fetal Liver HSCs Demonstrate High Transduction Rates andSustained Transgene Expression Following ImplantationNiraja Dighe, Maroun Khoury, Mark Chong, Citra Mattar, Michael N. Antoniou, JianzhuChen, Mahesh Choolani, Jerry Chan.532FANG Autologous Tumor <strong>Cell</strong> Vaccine Development and ManufacturingPhillip B. Maples, Padmasini Kumar, Yang Yu, Beena O. Pappen, Chris M. Jay, ZhaohuiWang, Donald D. Rao, Joseph Kuhn, John Nemunaitis, Neil Senzer.533Atomic Force Microscopy (AFM) Shows Lower Membrane Resistance<strong>of</strong> Human T <strong>Cell</strong>s Rendered Deficient in WASP by RNAiVéronique Pariétti, Ruthger Van Zwieten, Malgorzata Lekka, Nicolas Mermod, Anne Galy.534A Combination <strong>of</strong> Codon Optimization and Amino Acid Substitution inthe Human Factor IX <strong>Gene</strong> Yields a High Level <strong>of</strong> Superactive FactorIXThipparat Suwanmanee, William Hannah, Tong Gui, Hong Ma, Adam S. Cockrell, PaulE. Monahan, Tal Kafri.Immunologic & Host Responses in <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>535In Vivo Expansion <strong>of</strong> Regulatory T <strong>Cell</strong>s with IL2-mAb ComplexesPrevents and Modulates Anti-Factor VIII Immune Responses inHemophilia A Mice after Factor VIII Plasmid-Mediated <strong>Gene</strong> <strong>Therapy</strong>Chao-Lien Liu, Peiqing Ye, Benjamin Yen, Baowei Peng, Carol H. Miao.536Phase I Safety and Pharmacokinetic Study <strong>of</strong> Systemic p53 <strong>Gene</strong><strong>Therapy</strong> (SGT-53) Using Immunolipoplexes Targeted by Anti-Transferrin Receptor (TfR) Single Chain Antibody Fragment (scFv):Updated ResultsNeil Senzer, John Nemunaitis, Cynthia Bedell, Robert Nunan, Henry Allen, Leanne Sleer,Esther Chang.537Induction <strong>of</strong> Type I Interferon (IFN) by Adenovirus-Encoded SmallRNAsTomoko Yamaguchi, Kenji Kawabata, Emi Kouyama, Takayuki Suzuki, Fuminori Sakurai,Shinnosuke Kurachi, Kazufumi Katayama, Hiroyuki Mizuguchi.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010119Abstract DirectoryFriday, May 21, 2010Poster Session II — continued6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-SouthImmunologic & Host Responses in <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>538Immunological Effects <strong>of</strong> Oral and Intravenous Cyclophosphamide inCancer Patients Treated with Oncolytic AdenovirusesVincenzo Cerullo, Sophie Escutenaire, Sari Pesonen, Iulia Diaconu, Liisa Sirrka, AnnaKanerva, Lotta Kangasniemi, Elina Haavisto, Minna Oksanen, Tiimo Joensuu, A. Karioja-Kallio, Petteri Arstila, Akseli Hemminki.539The Role <strong>of</strong> TGF-b and IL-10 in the Development <strong>of</strong> ImmuneTolerance Following Hepatic <strong>Gene</strong> <strong>Therapy</strong>Brad E. H<strong>of</strong>fman, Ashley T. Martino, Brandon K. Sack, Roland W. Herzog.540Murine Hepatocytes Transduced with Y-F Mutant Capsid Are MoreResistant to Killing by Capsid Specific CD8+ T <strong>Cell</strong>sAshley Martino, David M. Markusic, Arun Srivastava, Roland W. Herzog.541 Abstract Withdrawn542The Innate Immune Response to Intravenously Injected AdenoviralVectors: Role <strong>of</strong> Endosomal Escape in MAP Kinase Activation and theCytokine/Chemokine ResponseJeffrey S. Smith, Zhili Xu, Jie Tian, Donna J. Palmer, Philip Ng, Andrew P. Byrnes.543AAV8 Vectors Evade CTL Mediated Destruction by Failing To Activatea Potent Inflammatory ResponseSuryanarayan Somanathan, Ekaterina Breous, Peter L. Bell, James M. Wilson.544Robust and Sustained Factor IX Expression by IV Administration <strong>of</strong>AAV8-Based Vectors in MacaquesHiroaki Mizukami, Jun Mimuro, Akira Ishiwata, Hiroya Yagi, Tsukasa Ohmori, SeijiMadoiwa, Tomonori Tsukahara, Masashi Urabe, Akihiro Kume, Yoichi Sakata, KeiyaOzawa.545Induction <strong>of</strong> Transgene Immunological Tolerance by a Single Injection<strong>of</strong> miR-142-Regulated Integrase Defective Lentiviral VectorAndrea Annoni, Alessio Cantore, Lucia Sergi Sergi, Luigi Naldini, Maria Grazia Roncarolo.546T <strong>Cell</strong> Hyperactivity in Cmah Null Mice: A Pre-Clinical rAAVImmunotoxicity Model?George Buchlis, Federico Mingozzi, Daniel J. Hui, Harre Downey, Virginia Haurigot,Shangzhen Zhou, Ajit Varki, Katherine A. High.547Complement-Mediated Clearance <strong>of</strong> AAV by Tissue ResidentMacrophages Limits Transduction <strong>of</strong> Target <strong>Cell</strong>sChelsea M. Bolyard, Jeffrey S. Bartlett.548Overcoming Anti-AAV Neutralizing Antibodies with AlternateSerotype and Pharmacological StrategiesFederico Mingozzi, Margriet J. Vervoordeldonk, Yifeng Chen, Shyrie Edmonson, RogierThurlings, Katherine High, Paul P. Tak.549Switching the Fiber Knob <strong>of</strong> Oncolytic Adenoviruses To AvoidNeutralizing Antibodies in Human Cancer PatientsMari Raki, Iulia Diaconu, Merja Sarkioja, Sophie Escutenaire, Lotta Kangasniemi, AnnaKanerva, Vincenzo Cerullo, Timo Joensuu, Sari Pesonen, Akseli Hemminki.550Depletion <strong>of</strong> B <strong>Cell</strong>s by Anti-CD20 Partially Regulates Anti-FVIIIAntibody Production in the Nonviral <strong>Gene</strong> <strong>Therapy</strong> ModelPeiqing Ye, Baowei Peng, Marilyn Kehry, David J. Rawlings, Carol H. Miao.551Sheep Harbor Naturally-Occurring Antibodies to Human AAV: A NewLarge Animal Model for AAV ImmunologyJoseph Tellez, Jonathan D. Finn, Nick Tschernia, Graça Almeida-Porada, Valder R. Arruda,Christopher D. Porada.552Effects <strong>of</strong> Various TLR/Inflammation Pathways on Humoral and<strong>Cell</strong>ular Immune Response to rAAV1 in MiceMuriel Sudres, Virginie Vasseur, Séverine Ciré, Bernhard Ryffel, Anne Galy.553Monitoring and Suppression <strong>of</strong> Immune Responses to AAV-Mediated<strong>Gene</strong> <strong>Therapy</strong> in Canine MuscleZejing Wang, Rainer Storb, Donghoon Lee, Martin Kushmeric, Baocheng Chu, JefferyChamberlain, Stainly Riddell, Stephen Tapscott.554Prevalence <strong>of</strong> Neutralizing Antibodies Against Adeno-AssociatedVirus 2, 8 and 9 in Non-Human Primate ColoniesHiroya Yagi, Hiroaki Mizukami, Tomonori Tsukahara, Masashi Urabe, Hiromi Hamada,Akihiro Kume, Hiroyuki Yoshikawa, Keiya Ozawa.Oligonucleotide & RNAi Therapeutics II555Dual Function BAFF Receptor Aptamers Block BAFF1 Ligand InducedProliferation and Deliver siRNAS to NHL <strong>Cell</strong>sKatrin Tiemann, Stephen J. Forman, John J. Rossi, Jiehua Zhou.556Efficient Delivery <strong>of</strong> siRNAs into Primary <strong>Cell</strong>s In Vitro andSystemically In Vivo Using Chemically Modified PepFect PeptidesSamir El Andaloussi, Edvard C. I. Smith, Ülo Langel.Friday, May 21 st


120<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryFriday, May 21 stFriday, May 21, 2010Poster Session II — continued6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-SouthOligonucleotide & RNAi Therapeutics II557Reduction <strong>of</strong> Mutant Huntingtin by Antisense OligonucleotidesAmeliorates Phenotypic Deficits in YAC128 Mouse Model <strong>of</strong>Huntington’s DiseaseJonathan A. Fidler, Lisa M. Stanek, Wendy Yang, Amy M. Richards, Edward Wancewicz,Curt Mazur, <strong>Gene</strong> Hung, C. Frank Bennett, Seng H. Cheng, Lamya S. Shihabuddin.558Antibody-Mediated Targeting <strong>of</strong> pH-Responsive NanoparticlesFacilitates siRNA Delivery to Cancer <strong>Cell</strong>sMaria C. Palanca-Wessels, Richelle Cutler-Strom, Anthony C. Convertine, Fan Lee,Michael F. Press, Scott E. James, Patrick S. Stayton, Oliver W. Press.559Adenoviral shRNA-Mediated Knockdown <strong>of</strong> TNFR1(P55) EitherLocally in the Knee Joint or Systemically Ameliorates Collagen-Induced ArthritisOnno J. Arntz, Jeroen Geurts, Sharon Veenbergen, Miranda B. Bennink, Wim B. van denBerg, Fons A. J. van de Loo.560Specific and Efficacious RNA Aptamer Against Hepatic Metastasis <strong>of</strong>Colon CancerSeong-Wook Lee, Young Ju Lee, Seung Ryul Han, Min jung Kim, Hanna Cho, Won IlLee, Jin-Sook Jeong.561Development <strong>of</strong> Aptamer Therapeutics Against <strong>Cell</strong> Surface Receptorsfor Lymphoma and Auto-Immune DiseasesElizabeth D. Pratico, David Boczkowski, Smita Nair, Bruce A. Sullenger.562A Microscopic Study <strong>of</strong> the Intracellular Fate <strong>of</strong> Cationic MediatedsiRNA DeliveryMeysam Abbasi, Rakesh Bhatnagar, Hasan Uludag.563Deriving Effective Anti-HIV Guide Sequences from Enhanced PrimarymicroRNA Secondary StructuresFiona van den Berg, Patrick Arbuthnot, Marc S. Weinberg.564“Bifunctional” shRNA Strategy for Cancer <strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong>Donald D. Rao, Zhaohui Wang, Padmasini Kumar, Yang Yu, Shihe Liu, Chris M. Jay,Nancy S. Templeton, Courtney Haddock, John Vorhies, Alex W. Tong, Phillip B. Maples,Neil Senzer, F. C. Brunicardi, John Nemunaitis.565Feasibility and Effectiveness <strong>of</strong> Exon 51 skipping in Human-Like mdxMutationYoshitsugu Aoki, Toshifumi Yokota, Takashi Saito, Akinori Nakamura, Tetsuya Nagata,Hitoshi Okazawa, Shin’ichi Takeda.566The Effect <strong>of</strong> RNA Interfering a1,3GT and NF-kB in CardiacXenotransplantationFeng Qi, Chengyu Lv, Yachuan Gu, Xianghui He, Liwei Zhu.567Inhibition <strong>of</strong> Breast Cancer <strong>Cell</strong> Growth Via Silencing IKKeBin Qin, Ravi S. Shukla, Kun Cheng.568Cationic Polymer Mediated siRNA Delivery for P-glycoprotein (P-gp)Down-Regulation in Tumor <strong>Cell</strong>sMeysam Abbasi, Afsaneh Lavasanifar, Michael Weinfeld, Hasan Uludag.569<strong>Gene</strong> Editing Induces Double-Stranded Breakage in Mammalian <strong>Cell</strong>sMelissa A. Bonner, Eric B. Kmiec.<strong>Cell</strong> Processing and Vector Production II570Development <strong>of</strong> a MDCK <strong>Cell</strong> Line for the Manufacturing <strong>of</strong> CanineAdenovirus Type 2 (CAV-2) VectorsVirgínia Santiago, Eric Kremer, Paula M. Alves, Ana S. Coroadinha.571Production <strong>of</strong> a rAAV1-CB-hAAT Human Clinical Trial Lot by rHSVCo-Infection <strong>of</strong> Suspension BHK <strong>Cell</strong>sDavid R. Knop, Brad Grimm, Lijun Wang, Darby L. Thomas, Guo-jie Ye.572RNA Based Plasmid Selection System for Antibiotic-Free PlasmidDNA Vector ProductionAaron E. Carnes, Jeremy M. Luke, Justin M. Vincent, Clague P. Hodgson, James A.Williams.573Robust Recombinase-Free System Production <strong>of</strong> Helper-DependentAdenoviral Vectors with Negligible Contamination by DelayedPackaging ProcessDan Cots, Raul Alba, Patrick Hearing, Eric J. Kremer, Maria Mercedes Segura, AssumpcióBosch, Miguel Chillón.574Removal <strong>of</strong> Clinically Relevant Contaminants from cGMP GradePreparations <strong>of</strong> Plasmid DNA: Implications for Non-Viral <strong>Gene</strong><strong>Therapy</strong> and DNA-Based VaccinesNancy S. Templeton, Pervez Firozi, Wei Zhang, Li Chen, Florante A. Quiocho, Kim C.Worley, Phillip B. Maples, John Nemunaitis.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010121Abstract DirectoryFriday, May 21, 2010Poster Session II — continued6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-South<strong>Cell</strong> Processing and Vector Production II575Escherichia coli Plasmid DNA Fermentation Strain and Process-SpecificEffects on Vector Yield, Quality and Transgene ExpressionAaron E. Carnes, Jeremy M. Luke, Justin M. Vincent, Clague P. Hodgson, James A.Williams.576An Assay To Monitor the Residual Contamination in the PlasmidDNA Manufacturing Area Based on the Correlation <strong>of</strong> Plasmid DNAConcentration to TOCRameswari Rameswaran, Lee Allen, Shane Johnson, Henry Hebel.577Counting and Sizing <strong>of</strong> Viral Vector Particles and Aggregates byNanoparticle Tracking Analysis (NTA)Andrew Malloy, Duncan Griffiths, Patrick Hole, Bob Carr.578Manufacturing and Stability Testing <strong>of</strong> a GNE-Lipoplex for a ClinicalTrial for the Treatment <strong>of</strong> Hereditary Inclusion Body Myopathy Type(HIBM)Chris M. Jay, Courtney Haddock, Phillip B. Maples, John Nemunaitis.579Dissolved Oxygen and pH Pr<strong>of</strong>ile Evolution in Shake Flasks during293 <strong>Cell</strong> Growth and Infection with AdenovirusAna Carina Silva, Daniel Simão, Marcos F. Q. Sousa, Pedro E. Cruz, Manuel J. T.Carrondo, Paula M. Alves.Late Breaking Science II933Targeted Lentiviral Vectors for the <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> X-Linked ChronicGranulomatous DiseaseChristian Brendel, Stefan Schultze-Strasser, Uta Müller-Kuller,Serap Tokmak, HanaKunkel, Linping Chen-Wichmann, Stefan Stein, Eva Rudolf, Michael Antoniou, ManuelGrez934Meganucleases: A Novel Anti-Viral ApproachJulianne Smith, Roman Galetto, Stephanie Grosse, Cécile Schiffer Mannioui, SylvainArnould, Marine Gailledrat, Philippe Duchateau, Frédéric Pâques & Carole Desseaux935Monomeric Designer T <strong>Cell</strong>s are More Potent against IL13Ra2Expressing GBMSeogkyoung Kong, Richard P Junghans, , Prakash Sampath936pSuperShuttle – An Easy Method for Modifications at any Site in theAdenoviral Genome by Conventional Bacterial RecombinationDaniel Oberg, Nick R Lemoine, Gunnel Hallden937<strong>Gene</strong>ration and Characterization <strong>of</strong> Lipid-Encapsulated AAV vectors:A Strategy to Evade Neutralization by AAV AntibodiesJ. Fraser Wright, Sissel Lund-KatzFriday, May 21 st


122<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectorySaturday, May 22 ndSaturday, May 22, 2010Oral Abstract Session 410Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>11:00 AM - 1:00 PMRoom: Delaware Suite580 11:00 AMIrradiation <strong>of</strong> Adult mdx Mice Prior to Full-Length Murine DystrophincDNA Transfer Results in a Delayed and Diminished Anti-DystrophinHost Immune ResponseSaman Eghtesad, Heng Zheng, Hiroyuki Nakai, Michael W. Epperly, Paula R. Clemens.581 11:15 AMTransient Expression <strong>of</strong> a Therapeutic Dystrophin Transgene inDuchenne Muscular Dystrophy Revealed by T <strong>Cell</strong> Mediated ImmunityJerry R. Mendell, Katherine Campbell, Louise R. Rodino-Klapac, Zarife Sahenk, ChristopherShilling, Sarah Lewis, Dawn Bowles, Steven Gray, Chengwin Li, Gloria Galloway, VinodMalik, Brian Coley, K. Reed Clark, Juan Li, Xiao Xiao, Jade Samulski, Scott McPhee, R.Jude Samulski, Christopher M. Walker.582 11:30 AMInduction <strong>of</strong> Oral Immunotolerance to rAAV9-Microdystrophin inCanine X-Linked Muscular DystrophyHiromi H. Kinoh, Naoko Yugeta, Hironori Okada, Yuko N. Kasahara, Takashi Okada,Shin’ichi Takeda.583 11:45 AMBaculovirus-Engineered Mesenchymal Stem <strong>Cell</strong>s Heal the Critical-Sized Femoral Segmental Bone Defects in RabbitsChin-Yu Lin, Yu-Han Chang, Kun-Ju Lin, Tzu-Chen Yen, Wen-Yi Lo, Chia-Hsin Luo, Ching-Lung Tai, Yu-Chen Hu.584 12:00 PM<strong>Gene</strong>tic Evidence for a Structural Role <strong>of</strong> a–dystrobrevin-3 within theDystrophin-Glycoprotein ComplexGuy L. Odom, Glen B. Banks, Dewayne Townsend, Marvin E. Adams, Joseph M. Metzger,Stanely C. Froehner, Jeffrey S. Chamberlain.585 12:15 PMrAAV8-Mediated Protein-Anchoring <strong>Therapy</strong> for Targeting CollagenQ-Tailed Acetylcholinesterase to the Neuromuscular JunctionMikako Ito, Yumi Suzuki, Takashi Okada, Takayasu Fukudome, Toshiro Yoshimura, AkioMasuda, Shin’ichi Takeda, Eric Krejci, Kinji Ohno.586 12:30 PMA Single Patient, Multiple Intravenous (IV) Infusion, Clinical Trial <strong>of</strong>GNE-Lipoplex for <strong>Therapy</strong> <strong>of</strong> Hereditary Inclusion Body Myopathy(HIBM)Gregory Nemunaitis, Chris M. Jay, Neil Senzer, Henry Allen, Anagha P. Phadke, Nancy S.Templeton, Phillip B. Maples, John Nemunaitis.587 12:45 PMSuccessful Expression <strong>of</strong> a R16/17 Micro-Dystrophin <strong>Gene</strong> inDystrophic Dogs with a Tyrosine-Mutant AAV Vector and a BriefCourse <strong>of</strong> Immune SuppressionJin-Hong Shin, Yi Lai, Yongping Yue, Arun Srivastava, Bruce F. Smith, Dongsheng Duan.Oral Abstract Session 411Chemical and Molecular Conjugates11:00 AM - 1:00 PMRoom: Virginia Suite588 11:00 AMTranscription Factor Binding by Plasmid DNA Is Needed forInteraction with Microtubules and Trafficking in <strong>Cell</strong>sMelissa A. Badding, Erin E. Vaughan, David A. Dean.589 11:15 AMQuantitative Analysis <strong>of</strong> the Effect <strong>of</strong> Functional Peptides onIntracellular Trafficking <strong>of</strong> Polymer <strong>Gene</strong> Delivery SystemsNicole M. Moore, Denis Pristinski, Shelly E. Sakiyama-Elbert.590 11:30 AMThe Effects <strong>of</strong> PNA-Based prePEGylation on the In Vitro and In VivoProperties <strong>of</strong> DNA PolyplexesPeter G. Millili, Ulhas P. Naik, Millicent O. Sullivan.591 11:45 AMParticle Tracking for Intracellular Trafficking <strong>of</strong> Octaarginine-ModifiedLiposome: Comparative Study with AdenovirusHidetaka Akita, Kaoru Enoto, Tomoya Masuda, Hiroyuki Mizuguchi, Tomomi Tani,Hideyoshi Harashima.592 12:00 PMAdvanced In Vitro Models Combined with MathematicalRepresentations Yield New Quantitative Understanding <strong>of</strong> Synthetic<strong>Gene</strong> Delivery System PerformanceMichelle C. Lowe, Todd D. Giorgio.593 12:15 PM<strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> the Blood Brain Barrier: Systemic Delivery <strong>of</strong>shRNA-Producing Plasmid DNA for Transient Suppression <strong>of</strong> MRP4Drug Efflux Transporter ProteinHongwei Zhang, Michelle L. Varney, Rakesh K. Singh, Serguei V. Vinogradov.594 12:30 PMPEGylated Polyacridine-Peptide DNA Open-Polyplexes with LongPharmacokinetic Half-Life Have Improved Hydrodynamic Stimulated<strong>Gene</strong> ExpressionChristian A. Fernandez, Nicholas J. Baumhover, Jason Duskey, Sanjib Khargharia, KevinG. Rice.595 12:45 PMSuccessful <strong>Gene</strong> Delivery with Receptor-Targeted PAMAMDendrimers Occurs Via Caveolae-Mediated PathwaysMark E. Hwang, William R. Gunther, Daniel W. Pack.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010123Abstract DirectorySaturday, May 22, 2010Oral Abstract Session 412Cardiovascular <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>11:00 AM - 1:00 PMRoom: Wilson ABC596 11:00 AMSystemic Delivery <strong>of</strong> AAV2/9 Vectors Improve Cardiac Function inPompe DiseaseDarin J. Falk, Meghan S. Soustek, Cathryn S. Mah, Denise A. Cloutier, Sean A. Germain,Barry J. Byrne.597 11:15 AMCoordinated Co-Expression <strong>of</strong> VEGF 164and PDGF-BB PreventsAberrant Angiogenesis and Accelerates Vascular StabilizationRoberto Gianni-Barrera, Silvia Reginato, Thomas Wolff, Lorenz Gürke, Michael Heberer,Andrea Banfi.598 11:30 AMMitral Valve Disease Is Due to Abnormal Collagen Structure in CanineMucopolysaccharidosis I and VII and Can Be Reduced with NeonatalIntravenous Injection <strong>of</strong> a Gamma Retroviral VectorKatherine P. Ponder, Meg M. Sleeper, Susan Wu, Patricia O’Donnell, Mark E. Haskins.599 11:45 AMAAV9-Mediated proBNP Delivery Improves Diastolic Performance ina Model <strong>of</strong> Spontaneous Hypertensive RatsJason M. Tonne, Jarryd M. Campbell, Diego Bellavia, Elise A. Oehler, Fernando L. Martin,Alessandro Cataliotti, John C. Burnett, Yasuhiro Ikeda.600 12:00 PMIn Vivo Over-Expression <strong>of</strong> a b-Adrenergic Receptor Kinase InhibitorUsing scAAV6-Mediated Molecular Cardiac Surgery with RecirculatingDelivery (MCARD) Enhances Cardiac FunctionJaBaris D. Swain, Michael G. Katz, Anthony Fargnoli, Marina Sumaroka, CatherineTomasulo, Mihail Petrov, James J. Pilla, Hansell H. Stedman, Walter J. Koch, JosephRabinowitz, Charles R. Bridges.601 12:15 PMAAV-Mediated SERCA2a Expression Improves theElectrocardiographic Pr<strong>of</strong>ile <strong>of</strong> Aged mdx MiceJin-Hong Shin, Brian P. Bostick, Yongping Yue, Roger J. Hajjar, Dongsheng Duan.602 12:30 PMRNAi-Mediated Knockdown <strong>of</strong> HMG CoA Reductase In Vitro and InVivo Increases Transgene Expression from Genomic LDLR Mini-<strong>Gene</strong>VectorsOlivia C. Hibbitt, Sarina Agkatsev, Carina Owen, Len Seymour, Keith M. Channon,Richard Wade-Martins.603 12:45 PMSDF-1a-Engineered <strong>Cell</strong>-Based <strong>Therapy</strong> for Diabetic Wound HealingZhao-Jun Liu, Runxia Tian, Weijun An, Ying Zhuge, Yan Li, Hongwei Shao, Bianca Habib,Omaida C. Velazquez.Oral Abstract Session 413Cancer-Oncolytic Viruses II11:00 AM - 1:00 PMRoom: Marriott Ballroom Salons 2 & 3604 11:00 AMHigh Resolution Analysis <strong>of</strong> the Intratumoral Spread <strong>of</strong> anIntravenously Administered Oncolytic VirusShruthi Naik, Rebecca Nace, Elizabeth Hadac, Glen Barber, Stephen J. Russell.605 Abstract Withdrawn191 11:15 AMA Comparative Study <strong>of</strong> Stem <strong>Cell</strong>-Based <strong>Cell</strong> Carrier Systems for theSystemic Delivery <strong>of</strong> an Oncolytic Adenovirus for Antiglioma <strong>Therapy</strong>Matthew A. Tayler, Atique U. Ahmed, Ilya V. Ulasov, Irina Y. Balyasnikova, Maciej S.Lesniak.606 11:30 AMCombination <strong>of</strong> Anti Angiogenic Cancer <strong>Therapy</strong> with SystemicOncolytic Virotherapy To Treat Established TumorsTimothy Kottke, Ge<strong>of</strong>f Hall, Jose Pulido, Rosa Diaz, Jill Thompson, DebabrataMukhopadhyay, Heung Chong, Peter Selby, Matt C<strong>of</strong>fey, Hardev Pandha, John Chester,Alan Melcher, Kevin Harrington, Richard Vile.607 11:45 AMThe Type III Interferon IL-28 Mediates Anti Tumor Efficacy <strong>of</strong>Oncolytic VSV in a Fully Immune Competent ModelPhonphimon Wongthida, Rosa Diaz, Feorillo Galivo, Timothy Kottke, Jill Thompson, JosePulido, Kevin Pavelko, Larry Pease, Alan Melcher, Richard Vile.608 12:00 PMA New Oncolytic Herpes Simplex Virus That Synergizes with SmallMolecule Inhibitors <strong>of</strong> PI3K/Akt To Treat Malignant GliomaRyuichi Kanai, Matthew Gardner, Hiroaki Wakimoto, Robert L. Martuza, Samuel D.Rabkin.609 12:15 PMImproving the Lytic Capacity <strong>of</strong> a Conditionally ReplicativeAdenoviruses by Incorporating Promoter Elements Responsive toTumor Environmental ConditonsDiego L. Viale, Veronica M. Lopez, Eduardo G. Cafferata, David Gould, Yuti Chenajovsky,Osvaldo L. Podhajcer.610 12:30 PMDefining the Role <strong>of</strong> Innate Immune Mechanisms in the Efficacy <strong>of</strong>Oncolytic VSV Virotherapy in a Fully Immune Competent ModelPhonphimon Wongthida, Rosa Diaz, Feorillo Galivo, Timothy Kottke, Jill Thompson, JosePulido, Alan Melcher, Richard Vile.611 12:45 PMDifferential Effects <strong>of</strong> VSV and Cyclophosphamide on NK <strong>Cell</strong>sLeads to Antagonistic Effects on Anti-Tumor <strong>Therapy</strong> <strong>of</strong> a MurineMesothelioma ModelCandice L. Willmon, Jill Thompson, Timothy Kottke, Stephen Albelda, Richard Vile.Saturday, May 22 nd


124<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectorySaturday, May 22 ndSaturday, May 22, 2010Oral Abstract Session 414Hematologic/Immunologic: Clinical, Preclinical and Safety11:00 AM - 1:00 PMRoom: Maryland Suite612 11:00 AMPolyclonal Reconstitution <strong>of</strong> the Hematopoietic System in TwoPatients <strong>of</strong> a WAS Clinical <strong>Gene</strong> <strong>Therapy</strong> TrialAnna Paruzynski, Kaan Boztug, Claudia Ball, Ali Nowrouzi, Christina Lulay, Marie Böhm,Sonja Naundorf, Klaus Kühlke, Rainer Blasczyk, Irina Kondratenko, Laszló Maródi,Hanno Glimm, Christoph Klein, Christ<strong>of</strong> von Kalle, Manfred Schmidt.613 11:15 AMSustained Partial Phenotypic Correction <strong>of</strong> Hemophilia B in the Dogby a microRNA-Regulated Lentiviral VectorAlessio Cantore, Marco Ranzani, Lucia Sergi Sergi, Giuliana Vallanti, Marina Radrizzani,Brian Brown, Eugenio Montini, Timothy Nichols, Luigi Naldini.614 11:30 AMTiming <strong>of</strong> Anti-T <strong>Cell</strong> Immunosuppressive <strong>Therapy</strong> Is Critical for theSafety <strong>of</strong> Liver Mediated AAV2 Transduction in Rhesus MacaquesJonathan D. Finn, Patricia Favaro, J. Fraser Wright, Federico Mingozzi, Katherine A.High, Valder R. Arruda.615 11:45 AMFactor IX-R338L (FIX Padua) as a Novel Alternative for theTreatment <strong>of</strong> Canine Severe Hemophilia BJonathan D. Finn, Paolo Simioni, Nicholas Iacobelli, Shangzhen Zhou, Timothy C.Nichols, Katherine A. High, Valder R. Arruda.616 12:00 PMErythropoeisis Sustained 1 Year by the EPODURE BioPump inPatients with Chronic Kidney Disease: Further Results <strong>of</strong> Phase I/IIPro<strong>of</strong> <strong>of</strong> Concept TrialBaruch S. Stern, Ehud Shoshani, Andrew L. Pearlman, Philip Ng, Allen R. Nissenson,Eithan Galun, Anatole Besarab, Avi Rimler, Haim Goltzmann, Reem Miari, Alex Okun,Haddas Bookay, Tamar Jibly, Javier A. Abrameto, Michal Elhalel.617 12:15 PMLentiviral <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> RAG1 Severe CombinedImmunodeficiency: Risks and Implications for TreatmentNiek P. van Til, Roya Sarwari, Trudi P. Visser, Helen de Boer, Yuedan Li, Julia Hauer,Chantal Lagresle-Peyrou, Olivier Danos, Arnaud Jollet, Fang Zhang, Adrian Thrasher,Barbara Cassani, Monique M. Verstegen, Anna Villa, Marina Cavazzana-Calvo, GerardWagemaker.618 12:30 PMDisruption <strong>of</strong> Glucocorticoid Regulation in Therapeutic Human T <strong>Cell</strong>swith Zinc Finger NucleasesRebecca A. Gardner, Andreas Reik, Carolina Berger, Seitaro Terakura, Philip D. Gregory,Michael C. Jensen, Michael C. Holmes, Stanley R. Riddell.619 12:45 PMLentiviral <strong>Gene</strong> Transfer under Optimized Cytokine ConditionsFacilitates Polyclonal Hematopoiesis <strong>of</strong> Recipient MiceTobias Maetzig, Martijn H. Brugman, Olga S. Kustikova, Zhixiong Li, Melanie Galla, NielsHeinz, Ute Modlich, Bernhard Schiedlmeier, Axel Schambach, Christopher Baum.Oral Abstract Session 415Lung and Respiratory Disease <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>11:00 AM - 12:00 PMRoom: Thurgood Marshall W620 11:00 AM<strong>Gene</strong>ration <strong>of</strong> a Transgenic Gut-Corrected CFTR Knockout FerretModel with Intestinal-Specific Expression <strong>of</strong> Ferret CFTRXiaoming Liu, Xingshen Sun, John T. Fisher, Meihui Luo, Hongshu Shui, Ziying Yan,Yaling Yi, Michael J. Goodheart, Gergely Lukacs, David Meyerholz, John F. Engelhardt.621 11:15 AMPersistent Long Term Lentiviral Correction <strong>of</strong> the CFTR <strong>Gene</strong> Defect inCystic Fibrosis MicePatricia Cmielewski, Donald Anson, David W. Parsons.622 11:30 AMDevelopment <strong>of</strong> Optimized hCFTR <strong>Gene</strong> Sequence and ProlongedExpression Vector for Cystic Fibrosis <strong>Therapy</strong>Linas Padegimas, Karla A. Dines, Samuel J. Adams, Sharon M. Oette, Susannah L. Hyatt,Olena Tyr, Christopher R. Gedeon, Ozge Sesenoglu-Laird, Robert C. Moen, Tomasz H.Kowalczyk, Mark J. Cooper.623 11:45 AMExpression <strong>of</strong> Human Alpha-1 Antitrypsin in Mice and Dogs FollowingAAV6 Vector-Mediated <strong>Gene</strong> Transfer to the LungsChristine L. Halbert, David K. Madtes, Andrew E. Vaughan, Zejing Wang, Rainer Storb,Stephen J. Tapscott, A. Dusty Miller.Oral Abstract Session 416<strong>Gene</strong> Regulation12:15 PM - 1:15 PMRoom: Thurgood Marshall W624 12:15 PMEngineered Zinc Finger Transcription Factors for the Treatment <strong>of</strong>Gain <strong>of</strong> Function MutationsClaudio Mussolino, Daniela Sanges, Ciro Bonetti, Elena Marrocco, Umberto Di Vicino,Valeria Marigo, Alberto Auricchio, Germana Meroni, Enrico M. Surace.625 12:30 PMAAV Vectors for Tissue-Specific, Regulated Expression in Heart, Liverand LungShu-Jen Chen, Maria P. Limberis, Lawrence Bish, Ruben Hovhannisyan, Sheena Saighal,Odella Jno-Charles, H. Lee Sweeney, Arbans Sandhu, Julie Johnston, James M. Wilson.EXHIBITOR PROSPECTUSfinal program


126<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectorySaturday, May 22 ndSaturday, May 22, 2010Oral Abstract Session 430 — continuedAdenovirus and Other DNA Virus Vectors3:00 PM - 5:00 PMRoom: Maryland Suite642 4:30 PMThe Cytoplasmic Sensor, NOD2, Induces the Innate Immune Responseand Transgene Silencing <strong>of</strong> Vector after Systemic Administration <strong>of</strong>Helper-Dependent AdenovirusMasataka Suzuki, Vincenzo Cerullo, Racel G. Cela, Terry K. Bertin, Christian Clarke, LeeH. Brendan.643 4:45 PMCAdVax-Vectored Multivalent Filovirus Vaccine Protects NHP AgainstChallenge at 1000 Times the Lethal Dose <strong>of</strong> Ebola and MarburgVirusesJohn Y. Dong, Danher Wang, William D. Pratt.Oral Abstract Session 431Infectious Diseases and Vaccines3:00 PM - 5:00 PMRoom: Delaware Suite644 3:00 PMChim3 as a New Transgene for Anti-HIV-1/AIDS <strong>Gene</strong> <strong>Therapy</strong>:Antiviral Effect and Survival Advantage Features in a Preclinical InVitro ModelSimona Porcellini, Luca Alberici, Francesco Gubinelli, Gian-Paolo Rizzardi, ChiaraBovolenta.645 3:15 PMIn Vivo Selection <strong>of</strong> CD4 + T <strong>Cell</strong>s Transduced with a Gamma-Retroviral Vector Expressing a Single-Chain Intrabody DirectedAgainst HIV-1 TatStephen E. Braun, Ran Taube, Quan Zhu, Fay E. Wong, Akikazu Murakami, Erick Kamau,Markryan Dwyer, Angela Carville, R. Paul Johnson, Wayne A. Marasco.646 3:30 PMSafety and Efficacy <strong>of</strong> Autologous CD4+ T cells Transduced with aLentiviral Vector Delivering Anti-HIV RNA Antisense env in HIV+Subjects Failing One or More HAART RegimensTessio Rebello, David Stein, Gary Blick, Richard Greenberg, Clifford Kinder, AndrewZolopa, Gerard McGarrity.647 3:45 PMLentiviral Delivery <strong>of</strong> TRIM-Cyclophilin Restriction Factors AgainstHIV-1Emma Chan, Torsten Schaller, Lu Zhao, Adrian Thrasher, Greg Towers, Waseem Qasim.648 4:00 PMDNA Vaccination with a Consensus H1HA Influenza ImmunogenInduces Immune Responses Capable <strong>of</strong> Protecting from a Lethal NovelA/H1N1-2009 and A/H1N1-1918 ChallengeM. P. Morrow, J. Yan, A. Khan, M. Pope, A. Patel, G. Kobinger, D. B. Weiner, N. Y.Sardesai.649 4:15 PMInduction <strong>of</strong> Immune Responses Against Multiple Species <strong>of</strong>Ebolavirus Using DNA and Retroviral-Based Virus-Like ParticlesBearing the Envelope Glycoprotein <strong>of</strong> a Single Species ZaireEbolavirusWu Ou, Josie Delisle, Carolyn Wilson.650 4:30 PMLow Levels <strong>of</strong> CXCR4 Are Sufficient for HIV-1 Entry and InfectionJinyun Yuan, Charles DeRossi, Michael C. Holmes, Philip D. Gregory, Bruce E. Torbett.651 4:45 PMOverexpression <strong>of</strong> the SLAM Receptor Adaptor Molecule EAT-2 as aNovel Strategy for Enhancing <strong>Cell</strong>ular Immunity to HIVantigensYasser A. Aldhamen, Daniel M. Appledorn, Sergey S. Seregin, Joyce C. Liu, Nathan J.Schuldt, Sarah S. Godbehere, Andrea Amalfitano.Oral Abstract Session 432Cancer – Immunotherapy II3:00 PM - 5:00 PMRoom: Marriott Ballroom Salons 2 & 3652 3:00 PMA Powerful Retroviral Vector for TCR <strong>Gene</strong> <strong>Therapy</strong>: Silencing <strong>of</strong>Endogenous TCR Improved Expression and Anti-Tumor Reactivity <strong>of</strong>Transduced Tumor-Specific TCRs in Human LymphocytesSachiko Okamoto, Hiroaki Ikeda, Hiroshi Fujiwara, Masaki Yasukawa, Hiroshi Shiku,Junichi Mineno.653 3:15 PMOrgan Specific Oncolytic Virus Incorporated Antigen Libraries(OSOVIAL) for the Immunotherapy <strong>of</strong> CancerJose Pulido, Timothy Kottke, Jill Thompson, Peter Selby, Alan Melcher, Richard Vile.654 3:30 PMEditing Human Lymphocyte Specificity for Safe and Effective AdoptiveImmunotherapy <strong>of</strong> LeukemiaElena Provasi, Pietro Genovese, Zulma Magnani, Angelo Lombardo, Andreas Reik, LiuPei-Qi, Oscar Muniz Pello, Jurgen Kuball, Attilio Bondanza, Giulia Casorati, Philip D.Gregory, Claudio Bordignon, Michael C. Holmes, Philip D. Greenberg, Luigi Naldini,Chiara Bonini.655 3:45 PMConstitutive 4-1BBL Expression Dramatically Increases Proliferation,Survival and In Vivo Persistence <strong>of</strong> CD19-Targeted T <strong>Cell</strong>s, PromotingComplete Tumor EliminationMaud Condomines, Jason Plotkin, John C. Markley, Gertrude Gunset, Michel Sadelain.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010127Abstract DirectorySaturday, May 22, 2010Oral Abstract Session 432 — continuedCancer – Immunotherapy II3:00 PM - 5:00 PMRoom: Marriott Ballroom Salons 2 & 3656 4:00 PMAn A20-Silenced, Stroma-Targeted DC Vaccine Reverses theImmunosuppressive Tumor Microenviroment Resulting in PotentAntitumor ActivityXiao-Tong Song, Xiaoou Zhou, Lisa Rollins, Cliona M. Rooney, Stephen Gottschalk.657 4:15 PMA CD46-Targeting Recombinant Protein that Increases theTherapeutic Efficacy <strong>of</strong> Monoclonal Antibodies for CancerHongjie Wang, Ying Liu, Zong Li, Akseli Hemminki, Andre Lieber.658 4:30 PMTreatment <strong>of</strong> Cancer Patients with Integrin-Targeted OncolyticAdenoviruses Ad5-D24-RGD and Ad5-RGD-D24-GMCSFSari Pesonen, Vincenzo Cerullo, Sophie Escutenaire, Mari Raki, Lotta Kangasniemi, EerikaKarli, Elina Haavisto, Minna Oksanen, Timo Joensuu, Anna Kanerva, Akseli Hemminki.659 4:45 PMEffective Dendritic <strong>Cell</strong> Immuno-<strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> Established ErbB2-Expressing Tumors Using Pentostatin-Based Host Immune Depletionand Subsequent Host Th1/Tc1 <strong>Cell</strong> TransferMiriam Mossoba, Jason Foley, Anu Gangopadhyay, Kris Ylaya, Matthew Winterton,Justin Taylor, Paul Massey, Stephen M. Hewitt, Jeffrey A. Medin, Daniel H. Fowler.Oral Abstract Session 433Oligonucleotide & RNAi Therapeutics II3:00 PM - 5:00 PMRoom: Thurgood Marshall W660 3:00 PMAlleviation <strong>of</strong> Potentially Adverse shRNA Off-Targeting Via AAVVector-Encoded Passenger Strand DecoysStefan Mockenhaupt, Nina Schürmann, Dirk Grimm.661 3:15 PMMultiplexing <strong>of</strong> snoRNAs for HIV-1 InhibitionJane Zhang, John J. Rossi.662 3:30 PMDelivery <strong>of</strong> Chemo-Sensitizing siRNAs to HER2-Positive BreastCancer <strong>Cell</strong>s Using RNA AptamersKristina W. Thiel, William H. Thiel, Xiuying Liu, Justin P. Dassie, Katie R. Stockdale,William M. Rockey, James O. McNamara, II, Paloma H. Giangrande.663 3:45 PMMethods for Isolating <strong>Cell</strong>-Internalizing RNA Aptamers for TargetedsiRNA DeliveryWilliam H. Thiel, Kristina W. Thiel, Justin P. Dassie, Liu Xiuying, Katie R. Stockdale,William M. Rockey, James O. McNamara, II, Paloma H. Giangrande.664 4:00 PMTreatment with the TLR9 Agonist MGN1703 in Patients withMetastatic Solid Tumors – EFFICACY Results <strong>of</strong> a Phase 1 ClinicalStudyMartin R. Weihrauch, Manuel Schmidt, Heike Richly, Bahar Bahar, Michael S. vonBergwelt-Baildon, Ulrich Hacker, Alexander Shimabukuro-Vornhagen, Marina Tschaika,Burghardt Wittig, Max E. Scheulen.665 4:15 PMmiR-7 Inhibits Schwannoma Tumor Growth by Directly Targetingp21-Activated Kinase 1 and Epidermal Growth Factor ReceptorOkay Saydam, Ozlem Senol, Arda Mizrak, Thomas Wurdinger, Anat O. Stemmer-Rachamimov, Anna M. Krichevsky, Xandra O. Breakefield.666 4:30 PMNanoparticles are Effective Vehicles for Systemic Delivery <strong>of</strong>2’OMePS Antisense Oligonucleotides in Exon Skipping-MediatedDystrophin RestorationAlessandra Ferlini, Marina Fabris, Elena Bassi, S<strong>of</strong>ia Falzarano, Daniela Perrone,Francesca Gualandi, Patrizia Sabatelli, Luciano Merlini, Paola Rimessi.667 4:45 PM<strong>Therapy</strong> for Vaso-Occlusion in Sickle <strong>Cell</strong> Disease Includes RNAAptamerAngela D. Burnette, Jun-ichi Nishimura, Milena Batchvarova, Shahid M. Nimjee, RahimaZenadi, Marilyn J. Telen, Bruce A. Sullenger.Oral Abstract Session 434<strong>Cell</strong> Processing and Vector Production3:00 PM - 5:00 PMRoom: Marriott Ballroom Salon 1668 3:00 PMGMP Production <strong>of</strong> Self-Complementary Serotype 8 AAV Vector forTreatment <strong>of</strong> Hemophilia BJames A. Allay, John S. Coleman, Arthur W. Nienhuis, Andrew David<strong>of</strong>f, Amit C.Nathwani, Susan Sleep, John T. Gray.669 3:15 PMProduction <strong>of</strong> Lentiviral Vectors in Suspension <strong>Cell</strong>s UsingRecombinant BaculovirusesHanna P. Lesch, Anna E. Laitinen, Lauri M. Laitinen, Jere T. Pikkarainen, HarithaSamaranayake, Seppo Ylä-Herttuala, Kari J. Airenne.670 3:30 PMIncreased Therapeutic Effect <strong>of</strong> <strong>Cell</strong> Transplantation for WoundHealing in Diabetic Mice by Prolonging the Survival <strong>of</strong> Transplanted<strong>Cell</strong>s Using Adhesamine, a Synthetic Adhesion MoleculeTetsuya Suehara, Makiya Nishikawa, Yuki Takahashi, Sayumi Yamazoe, MotonariUesugi, Yoshinobu Takakura.671 3:45 PMCharacterization <strong>of</strong> Positioning Effect <strong>of</strong> pol III-shRNA TranscriptionUnit in scAAV Vector Genome on the Packaging Efficiency andFunctionality <strong>of</strong> shRNA SilencingJun Xie, Ran He, Qin Su, Guangping Gao.J. Telen, Bruce A. Sullenger.Saturday, May 22 nd


128<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectorySaturday, May 22, 2010Oral Abstract Session 434 — continued<strong>Cell</strong> Processing and Vector Production3:00 PM - 5:00 PMRoom: Marriott Ballroom Salon 1672 4:00 PMRapid Production <strong>of</strong> High Titer Virus Using a Concentrated RetrovirusExpansion DeviceCeidy Sanchez, Gianpietro Dotti, John Wilson, Cliona M. Rooney, Malcolm K. Brenner,Juan F. Vera.673 4:15 PMA Novel Approach to Developing Recombinant AAV Producing <strong>Cell</strong>LinesJingmin Zhou, Katherine A. High, Guang Qu.674 4:30 PMAn Efficient and Complete Platform for the Production and Use <strong>of</strong>Lentiviral VectorsThomas Quinn, Chris Mello, Lily Lee, Mei Fong, Dwayne Bisgrove, Michael Haugwitz,Hiroaki Sagawa.675 4:45 PMDevelopment, Scale-Up and Technology Transfer for Rapid,Automated, cGMP Compliant, Large-Volume Suspension <strong>Cell</strong> BasedProcess for Lentiviral Vector ProductionJames Brady, Linhong Li, Scott R. Witting, Cornell Allen, Rama Shivakumar, Aparna Jasti,Kenneth G. Cornetta, Madhusudan V. Peshwa.678 3:30 PMUpdate <strong>of</strong> Stem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong> Clinical Trial for ADA-Deficiency inJapanMakoto Otsu, Masafumi Yamada, Yoshihiro Maeyama, Miyuki Kida, Satoru Nakajima,Nariaki Toita, Norikazu Hatano, Ryouji Kobayashi, Osamu Tatsuzawa, Junichi Mineno,Masafumi Onodera, Pawan Bali, Michael S. Hershfield, Fabio Candotti, NobuakiKawamura, Yukio Sakiyama, Tadashi Ariga.679 3:45 PMRescue and Long-Term Outcome <strong>of</strong> a Murine Model <strong>of</strong> MethylmalonicAcidemia Following Liver-Directed, AAV-Mediated <strong>Gene</strong> <strong>Therapy</strong>Randy J. Chandler, Nuria Carillo-Carrasco, Suma Chandrasekaran, Charles P. Venditti.680 4:00 PMAAV Vector Mediated Enzyme Replacement <strong>Therapy</strong> <strong>of</strong>Hypophosphatasia (HPP) Model MiceTae Matsumoto, Seiko Yamamoto, Hideo Orimo, Koichi Miyake, Noriko Miyake, SonokoNarisawa, José Luis Millán, Yoshitaka Fukunaga, Takashi Shimada.681 4:15 PMrDNA-AAV Vectors for the Treatment <strong>of</strong> PhenylketonuriaNick Morcinek, Laura M. Roy, Zhongya Wang, Markus Grompe, Cary Harding.682 4:30 PMSafety and Efficiency <strong>of</strong> Site-Specific Integration AAV-rDNA VectorsZhongya Wang, Mark Kay, Markus Grompe.683 4:45 PMFunctional Improvement <strong>of</strong> LGMD Hamsters by AAV-hMG53-Mediated Sarcolemma RepairBo He, Chunping Qiao, Bin Xiao, Juan Li, Zhenhua Yuan, Christina Mayer, Jianjie Ma,Xiao Xiao.Saturday, May 22 ndOral Abstract Session 435<strong>Gene</strong> <strong>Therapy</strong> for Inborn Errors <strong>of</strong> Metabolism3:00 PM - 5:00 PMRoom: Wilson ABC676 3:00 PMEarly Gestational <strong>Gene</strong> Transfer with Lentiviral Vector Driven bya Liver Specific Promoter Targets the Fetal Liver and ImprovesPhenotype in a Murine Model <strong>of</strong> Wilson’s DiseaseJessica L. Roybal, Masayuki Endo, Antoneta Radu, Carlyn Todorow, Philip W. Zoltick,Alan W. Flake.677 3:15 PMA Combination <strong>of</strong> Pharmacological and Liver-Directed <strong>Gene</strong> <strong>Therapy</strong>Results in Long-Term Phenotypic Correction <strong>of</strong> a Mouse Model <strong>of</strong>Argininosuccinic AciduriaPhilippe M. Campeau, Oleg A. Shchelochkov, Sandesh C. Sreenath Nagamani, NicolaBrunetti-Pierri, Donna Palmer, Muralidhar H. Premkumar, Harsha K. Garg, Anilkumar K.Reddy, Yaoping Tang, Nathan S. Bryan, Ayelet Erez, Brendan H. Lee.Oral Abstract Session 436Late Breaking Science3:00 PM - 5:00 PMRoom: Virginia Suite914 3:00 PMPostexposure Protection <strong>of</strong> Nonhuman Primates against a LethalEbola Virus Challenge by RNA InterferenceMarjorie Robbins, Amy CH Lee, Joan B Geisbert, Anna N Honko, Vandana Sood, Joshua CJohnson, Susan de Jong, Iran Tavakoli, Adam Judge, Thomas W Geisbert, Lisa E Hensley,Ian MacLachlan.915 3:15 PMA Clinical Study to Evaluate the Safety and Efficacy <strong>of</strong> pGM169/GL67A Administered to the Nose and Lungs <strong>of</strong> Individuals with CysticFibrosisStephen C Hyde, Jane C Davies, Uta Griesenbach, Tracy Higgins, Eric WFW Alton, AChristopher Boyd, J Alastair Innes, David Porteous, Deborah R Gill.916 3:30 PMLong-Term Safety and Transgene Expression in MonkeysAdministered Lentiviral and AAV Vectors In UteroAF Tarantal, CCI Lee, C Mall, BJ Byrne, DB Kohn.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010129Abstract DirectorySaturday, May 22, 2010Oral Abstract Session 436 — continuedLate Breaking Science3:00 PM - 5:00 PMRoom: Virginia Suite917 3:45 PMrAAV- Mediated Therapeutic Silencing <strong>of</strong> miR-122 Leads to Rapid andSignificant Reduction <strong>of</strong> LDL in DLDR-/-/Apobec1A-/- MiceJun Xie, Jennifer Broderick, Qing Xie, Hongwei Zhang, Ran He, Chengjian Li, Qin Su, LiZhong, Terence R. Flotte, Phillip D. Zamore, Guangping Gao.918 4:00 PMHyperactive Transposons for <strong>Gene</strong>tic Modification <strong>of</strong> InducedPluripotent and Adult Stem <strong>Cell</strong>s: A Novel Non-Viral Paradigm forCoaxed DifferentiationEyayu Belay, Janka Mátrai, Abel Acosta-Sanchez, Ling Ma, Mattia Quattrocelli, LajosMátés, Pau Sancho-Bru, Martine Geraerts, Bing Yan, Joris Vermeesch, Ermira Samara-Kuko, Zoltán Ivics, Catherine Verfaillie, Maurillio Sampaolesi, Zsuzsanna Izsvák, ThierryVandenDriessche, Marinee K.L. Chuah.919 4:15 PMPromoting the Migration and Integration <strong>of</strong> Schwann <strong>Cell</strong>s afterTransplantation into Lesioned Spinal Cord by Lentiviral MediatedExpression <strong>of</strong> Polysialic AcidJuan Luo, Xuenong Bo, Dongsheng Wu, John Yeh, Peter M Richardson, Yi Zhang.920 4:30 PMRescue <strong>of</strong> the Spinal Muscular Atrophy Phenotype in a Mouse Modelby Early Postnatal Delivery <strong>of</strong> SMNKevin D Foust, Xueyong Wang, Vicki L McGovern, Lyndsey Braun, Adam K Bevan,Amanda M Haidet, Thanh T Le, Pablo R Morales, Mark M Rich, Arthur H M Burghes,Brian K Kaspar.921 4:45 PMAptamer-Mediated Delivery <strong>of</strong> siRNAs and miRNA-Based siRNATriggers to the Nucleus <strong>of</strong> Multiple Cancer <strong>Cell</strong> TypesJonathan Kotula, Bruce Sullenger.Poster Session III6:00 PM - 8:00 PMRoom: Exhibit Hall CRNA Virus Vectors II684Insights on the Mechanisms <strong>of</strong> Measles Virus Entry into <strong>Cell</strong>sMatthew K. Ennis, Stephen J. Russell, David Dingli.685The Principal Function <strong>of</strong> the Lentiviral Central DNA Flap Is DefenseAgainst APOBEC3 Protein RestrictionChunling Hu, Dyana T. Saenz, Hind Fadel, Mary E. Peretz, William Walker, Eric M.Poeschla.686In Vivo Immunization with Integration Deficient Lentiviral VectorsBiliang Hu, Pin Wang.687“Same Day” Ex Vivo Regional <strong>Gene</strong> <strong>Therapy</strong>: A Novel Approach ToEnhance Bone RepairMandeep S. Virk, O. Sugiyama, Sang H. Park, Jay R. Lieberman.688The R Peptide Sequence <strong>of</strong> XMRV Envelope Glycoprotein Determinedthe Pseudotyping Specificity with HIV-1 and MLV VectorsToshie Sakuma, Suk See De Ravin, Harry L. Malech, Yasuhiro Ikeda.689Virus-Receptor Mediated Transduction <strong>of</strong> Dendritic <strong>Cell</strong>s among RossRiver Virus and Semliki Forest Virus Pseudotyped LentivectorsSteven Froelich, April Tai, Katie Stroup, Adnan Zubair, Pin Wang.690Effect <strong>of</strong> TLX1 Homeobox <strong>Gene</strong> Expression on InsertionalImmortalization by an Insulated Self-Inactivating Retroviral VectorLynnsey A. Zweier-Renn, Teresa S. Hawley, Ali Ramezani, Irene Riz, Robert G. Hawley.691Transcriptional Pulsing <strong>of</strong> Retroviral Transgenes in Mouse EmbryonicStem <strong>Cell</strong>sMandy Y. M. Lo, Akitsu Hotta, Sylvie Rival-Gervier, James Ellis.692The <strong>Cell</strong>ular Fibronectin Matrix Is a Key Factor for the Attachment<strong>of</strong> VSV-G Pseudotyped Vector Particles and Its <strong>Cell</strong> Type SpecificComplexity Provides a Mechanism for Delayed <strong>Cell</strong> EntryJosha A. Woodward, Amy M. Skinner, Peter Kurre.693Targeting <strong>of</strong> Lentiviral Vectors and Positioning <strong>of</strong> Transduced <strong>Cell</strong>s byMagnetic Nanoparticles and Magnetic FieldKatrin Zimmermann, Christina Trueck, Olga Mykhaylyk, Ulrich M. Becher, BernhardGleich, Daniela Wenzel, Bernd Fleischmann, Christian Plank, Alexander Pfeifer.694Pseudotyped Hepatitis C Virus Particle Could Be a Liver Target Bio-MissileXiaojing Lin, Yonghui Zhang, Yue Wang.695Regulation <strong>of</strong> Cytokine Expression by Lentivirus-Encoded RNAAntagomirsRasmus O. Bak, Line B. Petersen, Brian Moldt, Karin Stenderup, Cecilia Rosada, LarsAagaard, Tomas N. Dam, Jacob G. Mikkelsen.696Effects <strong>of</strong> Replicators on Retroviral Variegation and ExtinctionMeihong Liu, Maribeth V. Eiden.Saturday, May 22 nd


130<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectorySaturday, May 22 ndSaturday, May 22, 2010Poster Session III — continued6:00 PM - 8:00 PMRoom: Exhibit Hall CAAV Vectors III697Late Gestation Intrauterine <strong>Gene</strong> Transfer <strong>of</strong> AAV9 in Non-HumanPrimates Preferentially Transduces the Central Nervous System,Liver, Skeletal and Cardiac MusculatureC. N. Mattar, N. Johana, A. Fisk, A. Biswas, J. Lu, M. H. Tan, L. G. Tan, A. Rahim, S.Waddington, M. Choolani, J. Chan.698Integrin-Linked Endocytosis Directs AAV2 and AAV1 to a HighlyEfficient Transduction PathwayPaul M. Kaminsky, Nicholas W. Keiser, John F. Engelhardt.699Computationally-Guided Rational Design <strong>of</strong> Tropism-Modified AAVVectorsAdam S. Davis, William C. Ray, Thais Federici, Nicholas M. Boulis, Jeffrey S. Bartlett.700Identification <strong>of</strong> Human Hepatocyte Growth Factor Receptor as a<strong>Cell</strong>ular Receptor/Co-Receptor for AAV3Chen Ling, Yuan Lu, Jasmine K. Kalsi, Giridhara R. Jayandharan, Binbin Cheng, SamanthaW. Y. Gee, Katherine E. McGoogan, Li Zhong, Arun Srivastava.701Rapid Transgene Expression and Long-Term Phenotypic Correction<strong>of</strong> a Lethal Mouse Model <strong>of</strong> Methylmalonic Acidemia Using rAAV9-Mediated <strong>Gene</strong> DeliveryJulien S. Senac, Randy J. Chandler, Charles P. Venditti.702Widespread Transduction <strong>of</strong> Brain Parenchyma after IntraventricularInjection <strong>of</strong> AAV9Annagiusi Gargiulo, Enrico M. Surace.703AAV1.9-3 Exhibiting Significantly Impaired Infectivity andSubstantially Delayed Blood Clearance, Is a Novel DetargetedPlatform <strong>of</strong> AAV Random Peptide Display LibrariesKei Adachi, Nicole M. Kotchey, Kimimasa Tobita, Hiroyuki Nakai.704Development <strong>of</strong> the D[+]-Sequence-Deleted ssAAV2 Vectors:Enhanced Transgene Expression In Vitro and In VivoChen Ling, Giridhara R. Jayandharan, Baozheng Li, Yuan Lu, Wenqin Ma, Arun Srivastava.705Influence <strong>of</strong> Murine Parvovirus on Adeno-Associated Virus Vector-Related Expression in Murine ModelsSara A. Collins, Gerald C. O’Sullivan, Patrick T. Harrison, Martina F. Scallan, MarkTangney.706Involvement <strong>of</strong> the Glucocorticoid Receptor Signaling in AAV2 Vector-Mediated Transgene ExpressionYuan Lu, Chen Ling, Giridhara R. Jayandharan, Angela E. Rivers, Arun Srivastava.707AAV9-Mediated Catalase Expression in Mitochondria ImprovesExercise Performance in mdx MiceDejia Li, Dongsheng Duan.708Development <strong>of</strong> a Liver Specific Tet-On Inducible System for AAVVectorsLucia Vanrell, Laura Blanco, Lilianne Tenenbaum, Jesus Prieto, Gloria González-Aseguinolaza.709Intracortical Delivery <strong>of</strong> an Adeno-Associated Viral Vector Expressingthe Glutamate Transporter (GLT1) Decreases Stroke-InducedExtracellular Glutamate Overflow and Cerebral Infarction afterTransient Middle Cerebral Artery Occlusion in RatsBrandon K. Harvey, Mikko T. Airavaara, Jason Hinzman, Matthew J. Chiocco, Hui Shen,Douglas B. Howard, Greg A. Gerhardt, Barry J. H<strong>of</strong>fer, Yun Wang.710The Potential Role <strong>of</strong> Chromatin Structure in Adeno-Associated VirusType 2 Locus-Specific Integration at AAVS1Victor J. McAlister, Roland A. Owens.711Augmentation <strong>of</strong> Traditional Ad/AAV Helper Plasmids with HPV E1Increases rAAV DNA Replication and Virus Production in 293 <strong>Cell</strong>sSarmistha Bandyopadhyay, Maohua Cao, Yong Liu, Paul L. Hermonat.Adenovirus and Other DNA Virus Vectors III712Hexon-Modifications <strong>of</strong> hAd5 Based Helper-Virus for High-CapacityAdenoviral Vector Production and Limitations Imposed by HVR-Exchanges Utilizing Serotype SwitchMartin A. Hausl, Zsolt Ruzsics, Anja Ehrhardt.713Use <strong>of</strong> <strong>Cell</strong>-Penetrating Peptides To Induce a Bystander EffectYing Shen, William Yu, John G. Hay, Harald Sauth<strong>of</strong>f.714Highly Sensitive Detection <strong>of</strong> Prostate Cancer <strong>Cell</strong>s In Vivo by a DualAdenoviral TSTA Trans-Amplification SystemMakoto Sato, Frédéric Pouliot, Steve Huyn, Lily Wu.715Coagulation Factor X-Mediated Adenoviral Transduction Is Dependenton Heparan Sulfate Proteoglycan SulfationAngela C. Bradshaw, Alan L. Parker, Stuart A. Nicklin, John H. McVey, Andrew H. Baker.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010131Abstract DirectorySaturday, May 22, 2010Poster Session III — continued6:00 PM - 8:00 PMRoom: Exhibit Hall CAdenovirus and Other DNA Virus Vectors III716Towards the <strong>Gene</strong>ration <strong>of</strong> a Baculovirus Display Library for <strong>Cell</strong>TargetingMarcel Westenberg, Helen M. Soedling, Linda J. Nicholson, Derek A. Mann, Colin T.Dolphin.717Broad Spectrum Activity <strong>of</strong> the HSV-2 Vector DRR EnhancesNeuroprotection and Includes Crosstalk between Neurons and Glial<strong>Cell</strong>sJennifer M. Laing, Laure Aurelian.718Enhanced Transduction and Replication <strong>of</strong> Fiber-Modified Adenovirusin Murine Primary T <strong>Cell</strong>sSadhak Sengupta, Ilya V. Ulasov, Atique U. Ahmed, Maciej S. Lesniak.719Cloning <strong>of</strong> the Human Herpesvirus-6A (HHV-6A) Genome into theBacterial Artificial Chromosomes (BACs) towards the Creation <strong>of</strong>Helper-Less Amplicon Vectors for Potential Use in Lymphocyte <strong>Gene</strong><strong>Therapy</strong> and VaccinationNiza Frenkel, Ronen Borenstein, Haim Zeigerman, Adi Museri, Pnina Schlesinger.720Adenovirus-Mediated Decorin Expression Induces <strong>Cell</strong> Death throughActivation <strong>of</strong> p53 and Mitochondrial ApoptosisA-Rum Yoon, Jung-Sun Lee, Chae-Ok Yun.721PEG-Modified Adenovirus Conjugated with Herceptin ImprovesTherapeutic Value by Active Targeting through Her2/neu with anEnhanced Safety Pr<strong>of</strong>ileJoung-Woo Choi.722Study on Testing Method for Adeno-Associated Virus (AAV) <strong>of</strong>Adventitious VirusYejin Choi, Nam Hee Kim, Keum S. Choi, Keerang Park, Younjoo Park.723Ad5-Ad48 Hexon Hypervariable Region Substitutions Affect ViralBiodistribution and Inflammatory Responses In VivoLynda Coughlan, Angela Bradshaw, Laura Denby, Stuart A. Nicklin, Jerome Custers, Nicovan Rooijen, Dan H. Barouch, Andrew H. Baker.724Evaluation <strong>of</strong> a Porcine Adenovirus 3 Vector as a Post-ExposureVaccine CandidateAmi Patel, Suresk K. Tikoo, Gary P. Kobinger.725The Knob Domain Replacement Strategy Is Broadly Applicable to aWide Variety <strong>of</strong> Adenovirus Fiber ProteinsGalina Mikheeva, Natalya Belousova, Victor Krasnykh.DNA Vectorology & <strong>Gene</strong> Targeting726Transcriptional Repression <strong>of</strong> Transgenes Delivered by SleepingBeauty, piggyBac and Tol2 DNA Transposon VectorsNynne Sharma, Rasmus O. Bak, Nicklas H. Staunstrup, Gernot Wolf, Jacob G. Mikkelsen.727Duration <strong>of</strong> Expression from CpG-Free Plasmids FollowingHydrodynamic Delivery to the MouseIan A. Pringle, Mary M. Connolly, Anna E. Lawton, Lee A. Davies, Stephen C. Hyde,Deborah R. Gill.728A Liver-Specific Promoter Enhances Expression <strong>of</strong> alpha-L-Iduronidasein Sleeping Beauty Transposon-Mediated <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> MurineMPS IElena L. Aronovich, Jason B. Bell, Brenda L. Koniar, Perry B. Hackett.729New <strong>Gene</strong>ration, S/MAR Based, Episomal Vectors with HighPotential for <strong>Gene</strong> Transfer into Hematopoietic Progenitor <strong>Cell</strong>sEleana Stavrou, Vassileios Lazaris, Eirini Papapetrou, Aristeidis Giannakopoulos,Dimitra Kokkinou, Louiza Toutouna, Alexandros Spyridonidis, Nikolas Zoumbos, AglaiaAthanassiadou.730High-Yield, Non-Viral Production <strong>of</strong> Engineered <strong>Cell</strong>s throughSleeping Beauty Transposon-Mediated <strong>Gene</strong> Integration Coupled withSelective AmplificationNataly A. Kacherovsky, Maja Zavaljevski, Carl A. Blau, Cecilia M. Giachelli, Suzie H. Pun.731A Combinatorial Approach for Targeted, Systemic Delivery UsingSmall Molecules and Reversible Masking to Bypass Non-SpecificUptake In VivoNancy S. Templeton, Qiaoyun Shin, Andrew T. Nguyen, Yu Angell, Defeng Deng, Chang-Rim Na, Kevin Burgess, David D. Roberts, F. C. Brunicardi, Phillip B. Maples, JohnNemunaitis.732Using Helper Virus-Free Packaged HSV-1/EBV Hybrid Amplicons ToInduce Directed <strong>Cell</strong> Motility toward SDF-1aYasser M. El-Sherbini, Mark Stevenson, Richard Wade-Martins, Len W. Seymour.733A Novel System for the Evaluation <strong>of</strong> Genotoxicity for <strong>Gene</strong> <strong>Therapy</strong>VectorsJun Zhan, Matthew M. Wielgosz, John Gray, Arthur W. Nienhuis.Saturday, May 22 nd


132<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectorySaturday, May 22 ndSaturday, May 22, 2010Poster Session III — continued6:00 PM - 8:00 PMRoom: Exhibit Hall CDNA Vectorology & <strong>Gene</strong> Targeting734A Liver-Directed Targeting Tether Vector System To Increase Uptakein Liver <strong>Cell</strong>sJason B. Bell, Erik Olson, R. Scott McIvor, Perry B. Hackett.735<strong>Cell</strong>s Labeled with Fatty Acid-Oligonucleotide Conjugates asPotentially Suitable for Tissue EngineeringGalina E. Pozmogova, Marina A. Zaitseva, Andrey N. Chuvilin, Grigory G. Borisenko.736An Enhanced Non-Viral Expression Vector (EEV) – For CancerTreatmentPatrick F. Forde, Mira Sadadcharam, Marcel De Kruijf, Slawomir P. Salwa, Michael G.Bourke, Gerald C. O’Sullivan, Declan M. Soden.737Development <strong>of</strong> Post-Translational Tumor-Targeted-Cytokine Vectorfor Systemic Treatment <strong>of</strong> Malignant DiseaseJeffry J. Cutrera, Denada Dibra, Xuexing Xia, Shulin Li.738Improved Antibiotic-Free Plasmid Vector Design by Incorporation <strong>of</strong>Transient Expression EnhancersJeremy M. Luke, Justin M. Vincent, Clague P. Hodgson, James A. Williams.739Genomic DNA Reduction for Therapeutic DNA ManufactureYing Cai, Debra Bailey, Reto P. Bazzani, Henry Hebel, Stephen C. Hyde, StephenRodriguez, Deborah R. Gill.740Preclinical Efficacy and Safety <strong>of</strong> Electroporation-Mediated Delivery<strong>of</strong> a Plasmid Vector Manufactured in the Absence <strong>of</strong> AntibioticSelectionKaren E. Dolter, Cedric Y. Szpirer, Philippe Gabant, Drew Hannaman, Claire F. Evans.741Hepatic Stellate <strong>Cell</strong>-Specific TGF-b1 <strong>Gene</strong> Silencing for Treating LiverFibrosisNingning Yang, Ram I. Mahato.Chemical and Molecular Conjugates III742Nuclear Enclosure <strong>of</strong> Nanospheres in Artificial Nuclei and in Living<strong>Cell</strong>sNathalie Symens, Katrien Remaut, Jo Demeester, Stefaan C. De Smedt.743Analysis <strong>of</strong> Intracellular Trafficking <strong>of</strong> MPC-Coated MultifunctionalEnvelope-Type Nano Device (MPC-MEND) in Isolated HepatocyteMasami Ukawa, Hidetaka Akita, Tomoya Masuda, Yasuhiro Hayashi, Tomohiro Konno,Kazuhiko Ishihara, Hideyoshi Harashima.744Photo-Activated Release <strong>of</strong> DNA from Liposome ComplexesCaroline M. LaManna, Jiazuo H. Feng, Hrvoje Lusic, Mark W. Grinstaff.745A Novel Cationic Lipophosphoramide with Di-Unsaturated LipidChains: Synthesis, Physico-Chemical Properties, and TransfectionActivitiesTony Le Gall, Damien Loiseau, Erwan Picquet, Nathalie Carmoy, Jean Jacques Yaouanc,Laure Burel Deschamps, Pascal Delépine, Philippe Giamarchi, Paul Alain Jaffrès, PierreLehn, Tristan Montier.746Modifying DNA Nanoparticles with Biotin for Versatile LigandSubstitutionWenchao Sun, Pamela B. Davis.747An Integrative Approach To Monitor <strong>Gene</strong> Delivery to AirwayEpithelium by Nanocomplex Vectors and Radioimaging AgentsMaria D. I. Manunta, Aristides Tagalakis, Christopher O’Callaghan, Stephen L. Hart.748Development <strong>of</strong> Small, Homogenous Non-Viral <strong>Gene</strong> Vector UsingMono-Cationic DetergentsRyosuke Suzuki, Yuma Yamada, Hideyoshi Harashima.749Development <strong>of</strong> DNA Complex-Releasing Systems UsingBiodegradable Apatite Cement and Therapeutic Effect in Tumor ModelMiceTomoko Ito, Yoshiyuki Koyama, Makoto Otsuka.750Investigation <strong>of</strong> Cross-Linking Peptides as <strong>Gene</strong> Delivery VectorsAnton V. Kiselev, Anna A. Egorova, Arto O. Urtti, Vladislav S. Baranov.751Rapid and Highly Efficient Magnet<strong>of</strong>ection <strong>of</strong> IL-10 to ExtremelySusceptible Vascular Endothelial <strong>Cell</strong>s Using Hybrid Iron Oxide-PEIConjugateYong Sook Kim, In-Kyu Park, Jin Sook Kwon, Myung Ho Jeong, Youngkeun Ahn.752Cocaine Conjugated to Disrupted Adenovirus – A Synthetic/Viral Protein Hybrid Strategy To Elicit High Titers Against Non-Immunogenic Addictive Small MoleculesBishnu P. De, Martin J. Hicks, Jonathan B. Rosenberg, Stefan Worgall, Stephen M.Kaminsky, Amira Y. Moreno, Kim D. Janda, Ronald G. Crystal.753Bisphosphonate-Mediated <strong>Gene</strong>-Eluting Metal Stent as <strong>Gene</strong> VectorDelivery Using Antibody for Localized <strong>Gene</strong> DeliveryYong Wang, Cunxian Song, Robert J. Levy.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010133Abstract DirectorySaturday, May 22, 2010Poster Session III — continued6:00 PM - 8:00 PMRoom: Exhibit Hall CChemical and Molecular Conjugates III754Optimization <strong>of</strong> Sendai F/HN Viroplexes for Efficient Transfection <strong>of</strong>Plasmid DNA and siRNA to Suspension <strong>Cell</strong>sJung Seok Kim, Yeon Kyung Lee, Jin Ee Baek, Sang Il Park, Hwa Yeon Jeong, Hong SungKim, Keun Sik Kim, Yong Serk Park.755Comparative Evaluation <strong>of</strong> Disulfide-Linked and Hydrophobically-Modified PEI for Plasmid Delivery In VitroK. C. B. Remant, Breanne Landry, Hasan Uludag.756Linear Polyethylenimine Prepared by Partial Acid Hydrolysis <strong>of</strong> Poly(2-ethyl-2-oxazoline) for DNA and siRNA Delivery In VitroJulio C. Fernandes, Xingping Qiu, Francoise M. Winnik, Mohamed Benderdour, XiaolingZhang, Kerong Dai, Qin Shi.757Impact <strong>of</strong> Counterion and Hydrophobic Anchor on PhysicochemicalCharacteristics and Transfection Efficiency for a Series <strong>of</strong> PyridiniumCationic Lipid-Based LipoplexesSushma Savarala, Stephanie L. Wunder, Marc A. Ilies.<strong>Gene</strong>tic and Metabolic Diseases <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> III758AAV-Mediated Liver <strong>Gene</strong> Transfer Results in Long-Term Expression<strong>of</strong> Therapeutic Levels <strong>of</strong> ARSB in MPS VI CatsGabriella Cotugno, Patrizia Annunziata, Anita Capalbo, Thomas O’Malley, PatriciaO’Donnell, Fabio Russo, Elvira De Leonibus, Mark Haskins, Alberto Auricchio.759Beta-Glucuronidase Expressed from AAV Is Poorly Modified withMannose-6-Phosphate Residues and Fails to Completely CorrectMucopolysaccharidosis VII in DogsGuangping Gao, Peter L. Bell, Mark E. Haskins, Lisa A. Kisling, Richard Steet, Luk H.Vandenberghe, Hongwei Yu, Julio C. Sanmiguel, James M. Wilson.760<strong>Gene</strong> Transfer To Induce <strong>Cell</strong>ular Reprogramming for the Treatment <strong>of</strong>DiabetesNeil Phillips, Markus Grompe, Mark A. Kay.761Salivary Gland hPTH <strong>Gene</strong> Transfer: Interesting PossibilitiesJanik Adriaansen, Paola Perez, Changyu Zheng, Michael T. Collins, Bruce J. Baum.762Efficient <strong>Gene</strong> Transfer to the Pancreas Using ssAAV for Prevention<strong>of</strong> Type 1 DiabetesEduard Ayuso, Veronica Jimenez, Judith Agudo, Ariana Salavert, Sergio Muñoz, AlbaCasellas, Merce Obach, Maria Molas, Marta Moya, Xavier Leon, Jesus Ruberte, FatimaBosch.763Tolerance Induction to Coagulation Factor IX Using <strong>Gene</strong> ModifiedPrimary B <strong>Cell</strong>sBabak Moghimi, Irene Zolotukhin, Ou Cao.764 (Moved to Poster Session II after Abstract 422)Microporous Polymer Scaffolds for Islet TransplantationRomie F. Gibly, Lonnie D. Shea.765AAV2- and AAV9 -Mediated <strong>Gene</strong> Transfer <strong>of</strong> LDL Receptor toHypercholesterolemic WHHL RabbitsElisa Vähäkangas, Anniina Laurema, Atsushi Miyanohara, Seppo Ylä-Herttuala.766Hairpin Ribozymes Encoded in Adenoviral Vectors Reduce Drinking inAlcohol Dependent RatsAmalia Sapag, Thergiory Irrazábal, María Elena Quintanilla, Lutske Tampier.767Sustained Therapeutic Levels <strong>of</strong> FVIII Activity from Lentivirally-Engineered Blood Outgrowth Endothelial <strong>Cell</strong>s Requires <strong>Cell</strong> Stabilityand Augmented AngiogenesisHideto Matsui, Margareth Ozelo, Andrea Labelle, Carol Hegadorn, Patrick Thompson,Erin Burnnet, Christine Hough, David Lillicrap.768Effects <strong>of</strong> Neonatal Intracranial AAV and Systemic Lentiviral <strong>Gene</strong><strong>Therapy</strong> in Sanfilippo Syndrome Type B MiceCoy D. Heldermon, Elizabeth Qin, Kevin K. Ohlemiller, Erik Herzog, Carole Vogler, DavidF. Wozniak, Mark S. Sands.769Laminin-111: A Highly Potent Therapeutic Agent To Treat DuchenneMuscular DystrophySebastien Goudenege, Yann Lamarre, Nicolas Dumont, Joel Rousseau, Skuk Daniel,Jacques P. Tremblay.770Layered Scaffolds for Islet Transplantation and <strong>Gene</strong> DeliveryJohn G. Graham, Lonnie D. Shea.771Lentiviral Vector Correction <strong>of</strong> Fabry Disease Encompassing a <strong>Cell</strong>Fate Control Safety ElementMatthew D. Scaife, Sean P. Devine, Anton Neschadim, Natalia A. Pacienza, Jeffrey A.Medin.772Wolman Disease (LIPA p.G87V) Genotype Frequency in Patients <strong>of</strong>Iranian-Jewish DescentYadira Valles-Ayoub, Daniel No, Saghi Esfandiarifard, Zeshan Khokher, Chai Saechao,Marvin Pietruszka, Daniel Darvish.Saturday, May 22 nd


134<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectorySaturday, May 22 ndSaturday, May 22, 2010Poster Session III — continued6:00 PM - 8:00 PMRoom: Exhibit Hall CNeurodegenerative Disorders, Spinal Cord and Trauma773Delayed Inhibition <strong>of</strong> Soluble Tumor Necrosis Factor Signaling Blocksthe Progressive Phase <strong>of</strong> Dopaminergic Neuron Death in a Rat Model<strong>of</strong> Parkinson’s DiseaseAshley S. Harms, Kelly M. Ruhn, Steve Varghese, Isaac Trevino, Malu G. Tansey.774Identification and Analysis <strong>of</strong> a Novel g-Secretase Inhibitory <strong>Gene</strong>Hiroki Hayashi, Hironori Nakagami, Nobutaka Koibuchi, Munehisa Shimamura, YasufumiKaneda.775Effects <strong>of</strong> Alpha 7 Nicotinic Receptor <strong>Gene</strong> Delivery on ProcessesUnderlying <strong>Cell</strong> Viability and <strong>Cell</strong> DifferentiationYan Ren, Lin Zhang, Edwin M. Meyer, Jeffrey A. Hughes, Sihong Song.776Delivery <strong>of</strong> hGDNF to Brain Using DNA Nanoparticles:Neuroprotection <strong>of</strong> the Nigrostriatal PathwayDavid M. Yurek, Anita M. Fletcher, Linas Padegimas, Tomasz H. Kowalczyk, Mark J.Cooper.777ProSavin, a <strong>Gene</strong> <strong>Therapy</strong> for Parkinson’s DiseaseBechir Jarraya, Helene Lepetit, Scott Ralph, James Miskin, Jean-Marc Gurruchaga, MariaVinti, Gilles Fenelon, Pierre Brugiere, K. Abay, I. Gabriel, Sabrina Boulet, Caroline Jan,Susan Kingsman, P. Cesaro, Philippe Hantraye, Philippe Remy, Kyriacos Mitrophanous,Stephane Palfi.778Primate Hippocampal <strong>Gene</strong> Delivery Via Frameless StereotaxisCary H. Leung, Michele A. Kliem, Brooke R. Snyder, Thais Federici, Thomas J. McKown,Scott W. J. McPhee, Nicholas M. Boulis.779Delivery Co-Interventions in <strong>Gene</strong> Transfer and <strong>Cell</strong> Transplantation:A Meta-Analysis <strong>of</strong> Surgical Risk for Trials Involving Inoculation toDeep Brain StructuresJonathan Kimmelman, Katherine Duckworth, Tim Ramsay, Marina Emborg, Tiffini Voss.780Modification <strong>of</strong> the Inhibitory Environment <strong>of</strong> Spinal Cord Injury byLentiviral Vector Containing NT-3 and shRNA for NG2Eleanor M. Donnelly, Nicolas N. Madigan, Gemma E. Rooney, Anthony J. Windebank,Timothy O’Brien, Siobhan S. McMahon.781HSV <strong>Gene</strong> Transfer <strong>of</strong> a Poreless TRPV1 Receptor Reduces Pain-Related Behavior in RodentsJames R. Goss, William F. Goins, Rahul Srinivasan, Joseph C. Glorioso.782A Comparison <strong>of</strong> AAV Serotypes and Routes <strong>of</strong> Administration forDelivery to the Spinal CordBrooke R. Snyder, Thais Federici, Eric Quach, Steven Gray, Cary H. Leung, Nicholas M.Boulis.783Engineered Zinc Finger Protein Improves Microvascular Recovery,Neuronal Sparing and Functional Recovery in Modeled TraumaticBrain Injury in RatsEugene Park, Ishita Siddiq, Elaine Liu, S. Kaye Spratt, Richard Surosky, Gary Lee, DaleAndo, Marty Giedlin, Andrew J. Baker.Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> III784Blocking the Myostatin Signal Improves the Success <strong>of</strong> HumanMyoblast Transplantation in Dystrophic MiceRaouia Fakhfakh, Annick Michaud, Jacques P. Tremblay.785High Force Myosin Correlated with the Early Degeneration <strong>of</strong> JawClosing Muscle in the GRMD Model <strong>of</strong> DMDAndrew F. Mead, Alock Malik, Mihail Petrov, Martin Childers, Marilyn Mitchell, JanetBogan, Joe Kornegay, Brittani Nickens, Hansell Stedman.786Comparison Studies <strong>of</strong> rAAV6 Transduction Efficiency in Adult andAged MusclesPatryk Konieczny, James M. Allen, Eric Finn, Jeffrey S. Chamberlain.787<strong>Gene</strong> Transfer <strong>of</strong> Signals Critical for Allograft Healing UsingImmobilized Freeze Dried Adeno-Associated Viral VectorsMette Koefoed, Alexandra G. Pedersen, Michael Ulrich-Vinther, Thomas G. Jensen, KjeldSoballe.788Rescue from Respiratory Dysfunction by Transduction <strong>of</strong> Full-LengthDystrophin to Diaphragm Via the Peritoneal Cavity in MuscularDystrophy MouseIshizaki Masatoshi, Yasushi Maeda, En Kimura, Makoto Uchino.789Restoration <strong>of</strong> Wild-Type DMD mRNA in an Immortalized HumanFibroblast Line from an Exon 8-9 Duplicated DMD PatientLing Zhao, Gillian Butler-Browne, Vincent Mouly, Soraya Chaouch, Kamel Mamchaoui,Steve D. Wilton, Kevin M. Flanigan.790Expression <strong>of</strong> Micro-Dystrophin and Full-Length Dystrophin in Mouseand Dog MusclesChristophe Pichavant, Pierre Chapdelaine, Joe N. Kornegay, João C. S. Bizario, XiaoXiao, Jacques P. Tremblay.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010135Abstract DirectorySaturday, May 22, 2010Poster Session III — continued6:00 PM - 8:00 PMRoom: Exhibit Hall CMusculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> III791A Comparison <strong>of</strong> Adeno-Associated Virus Mediated <strong>Gene</strong> Transferinto Healthy and Dystrophin-Deficient Skeletal MusclesGilles Moulay, Carole Masurier, Pascal Bigey, Daniel Scherman, Antoine Kichler.792In Vivo Reprogramming into the Myogenic Lineage and ImprovingEngraftment in the mdx Mouse Model <strong>of</strong> DMDLindsey A. Muir, Jeffrey S. Chamberlain.793<strong>Gene</strong> Transfer-Driven Inhibition <strong>of</strong> Cardiac Ankyrin Repeat Protein asa Potential <strong>Therapy</strong> for the Correction <strong>of</strong> Muscular DystrophiesNathalie F. Danièle, Laurence Suel-Petat, Isabelle Richard.794Background Strain and Natural Selection Improves Survival <strong>of</strong> HIBMMouse ModelYadira Valles-Ayoub, Zeshan Khokher, Arman Haghighatgoo, Daniel No, SaghiEsfandiarifard, Chai Saechao, Jennifer Kay, Christopher Creencia, Sheila Darvish, DanielDarvish.795Combining Stem <strong>Cell</strong>s and Exon Skipping Approach To Treat MuscularDystrophiesMirella Meregalli, Daniele Parolini, Andrea Farini, Marzia Belicchi, Giuseppe D’Antona,Simona Maciotta, Paola Razini, Alberto Lerario, Serena Bonfiglio, Roberto Bottinelli, LuisGarcia, Nereo Bresolin, Yvan Torrente.796<strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong> Approaches Tto Treating the DysferlinopathiesBradley A. Williams, Nilah Monnier, Douglas E. Albrecht, Nupur Garg, Laura E. Rufibach,Esther Hwang, Angela Salerno, Plavi Mittal.Infectious Diseases and Vaccines: Non-Vaccine Related <strong>Gene</strong>Transfer Research797Delineation <strong>of</strong> RNA Antisense env Effects on HIV QuasispeciesRecovered from Lexgenleucel-T Treated Subjects: Influences <strong>of</strong>Sequence Similarity <strong>of</strong> the RNA Antisense with the Targeted HIV envTranscript and VRX496-Modified <strong>Cell</strong> PersistenceAlexander Pasternak, Nikolay Korokhov, Vladimir Lukashov, Laurent M. Humeau.798Assessing Preclinical Safety <strong>of</strong> a CCR5-Specific Zinc Finger Nucleasein Human CD4+T <strong>Cell</strong>s Using the NSG Immunodeficient Mouse ModelS. Kaye Spratt, Marty Giedlin, Richard Carroll, Shuguang Jiang, Dawn Maier, GwendolynBinder-Scholl, Gary Lee, Lynn Truong, Bruce Levine, Michael Holmes, Philip Gregory, CarlJune, Dale Ando.799Multivirus-Specific T <strong>Cell</strong> Immunotherapy To Prevent or TreatInfections <strong>of</strong> Allogeneic Stem <strong>Cell</strong> Transplant RecipientsAnn M. Leen, Ulrike Gerdemann, Carlos A. Ramos, Tamara Tripic, Malcolm K. Brenner,Cliona M. Rooney, Juan F. Vera, Helen E. Heslop.800Inhibition <strong>of</strong> HIV-1 Replication in VRX496-Modified CD4 T <strong>Cell</strong>s:Importance <strong>of</strong> Cytoplasmic dsRNA ComponentsRandall Merling, Brit J. Hart, Ziping C. Chen, Laurent M. Humeau, Nikolay Korokhov.801Engineering Resistance to CXCR4-Tropic HIV: A Cutting EdgeApproachCraig B. Wilen, Michael C. Holmes, Jianbin Wang, Philip D. Gregory, Carl H. June, RobertW. Doms.802Development <strong>of</strong> an Unbiased Method for Characterizing ViralPopulations Using Next <strong>Gene</strong>ration Sequencing TechnologyStephanie M. Willerth, Helder L. Pedro, Laurent M. Humeau, Adam P. Arkin, David V.Schaffer.803Long Term Persistence <strong>of</strong> VRX496-Modified CD4+ T LymphocytesFollowing Lexgenleucel-T Infusions for the Treatment <strong>of</strong> HIV/AIDS:Results from a Phase 2 Clinical TrialLaurent M. Humeau, Kris Andre, Karen Bengtson, Shuyuan Zhang, Gerard McGarrity,Tessio Rebello.804<strong>Gene</strong>ration <strong>of</strong> Restriction Factor-Transgenic Cats Using LentiviralTransgenesisPimprapar Wongsrikeao, Dyana T. Saenz, Thomas A. Rinkoski, Takeshige Otoi, Eric M.Poeschla.805Combinatorial Latency Reactivation for HIV-1 Subtypes and VariantsJohn C. Burnett, John J. Rossi, Kwang-il Kim, Adam P. Arkin, David V. Schaffer.806Highly Efficient Expression and Purification <strong>of</strong> Hepatitis C Virus CoreProtein in E.coliYixin Bian, Guozhen Qiu, Tingting Li, Zixuan Chen, Wenjing Wang, Chengyao Li.807Molecular Epidemiology <strong>of</strong> HCV Infection among Recovery andChronic Blood Donors in Guangdong, ChinaTingting Li, Jinfeng Zeng, Zixuan Chen, Lifang Shuai, Anqi Wang, Chengyao Li.Saturday, May 22 nd


136<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectorySaturday, May 22 ndSaturday, May 22, 2010Poster Session III — continued6:00 PM - 8:00 PMRoom: Exhibit Hall CInfectious Diseases and Vaccines: Non-Vaccine Related <strong>Gene</strong>Transfer Research808Occult Hepatitis B Virus Infection in Shenzhen, ChinaXin Zheng, Yixin Bian, Ling Zhang, Wenjing Wang, Lifang Shuai, Anqi Wang, Jean-PierreAllain, Daniel Candotti, Chengyao Li.Cancer – Immunotherapy III809Time for TEA: T <strong>Cell</strong>s Expanded on Artificial Antigen Presenting <strong>Cell</strong>s?Hillary N. Gibbons, Carrie Yuen, Sergio Giralt, Carl H. June, Laurence J. N. Cooper.810Oxaliplatin Increases the Antitumor Effect <strong>of</strong> Vector-DeliveredInterleukin-12 Expression in a Murine Model <strong>of</strong> Hepatic ColorectalCancer MetastasesManuela Gonzalez-Aparicio, Itsaso Mauleon, Pilar Alzuguren, Sandra Hervas-Stubbs,Uxua Manchengo, Julien Crettaz, Gloria González-Aseguinolaza, Jose Medina-Echeverz,Pedro Berraondo, Jesus Prieto, Ruben Hernandez-Alcoceba.811Optimizing Dendritic <strong>Cell</strong> Vaccination by Combination with OncolyticAdenovirus Coexpressing Interleukin-12 and Granulocyte-Macrophage Colony Stimulating FactorSong-Nan Zhang, Jing-Hua Huang, Ji Young Yoo, Kyung-Ju Choi, Chae-Ok Yun.812Bacterial Vector Mediated Vaccination for Prostate CancerGarrett Casey, Sarfraz Ahmad, Michelle Cronin, Simon Rajendran, Mark Tangney, GeraldC. O’Sullivan.813Evaluation <strong>of</strong> Long-Term Transgene Expression in piggyBac-ModifiedHuman T LymphocytesYozo Nakazawa, Sunandan Saha, Daniel Galvan, Cliona M. Rooney, Matthew H. Wilson.814Engineered Drug Resistant Immunocompetent <strong>Cell</strong> <strong>Therapy</strong> forNeuroblastomaAnindya Dasgupta, David McCarty, H. Trent Spencer.815<strong>Gene</strong>tically Mediated Constitutive Expression <strong>of</strong> CD40L by T <strong>Cell</strong>sWill Induce Enhanced Immunogenicity <strong>of</strong> B-<strong>Cell</strong> MalignanciesKevin J. Curran, Raymond Yeh, Beatrijs Seinstra, Yan Nikhamin, Yelena Usachenko,Renier J. Brentjens.816Tumor Antigen Delivery to Secondary Lymphoid Organs byInterleukin-12 <strong>Gene</strong> Modified T <strong>Cell</strong>sAdham S. Bear, Xiatong Song, Meghan E. Turnis, Stephen M. Gotschalk, Cliona M.Rooney, Aaron E. Foster.817Pluronic Based Formulations Are Promising Anticancer DNA VaccinesCaroline Roques, Zagit Gaymalov, Jedd D. Wolchok, Alexander V. Kabanov.818Efficient Induction <strong>of</strong> Humoral and <strong>Cell</strong>ular Immune ResponsesAgainst Her-2/neu by Combination <strong>of</strong> Naked DNA and AdenoviralVectors Expressing <strong>Gene</strong>tically Engineered Her-2/neuJae-Gyun Jeong, Dong-Sik Kim, Sung-Won Lim, Kyu-Woong Shim, Hwang-Jae Lee, Sung-Min Oh, Jin-A Chae, Yeon-Sook Cha, Seong-Hyun Ho, Youngtae Hong, Woong Hahn,Sujeong Kim, Jong-Mook Kim, Seungshin Yu, Chang-Yuil Kang, Sunyoung Kim.819Autocrine, Not Paracrine, Interferon-Gamma <strong>Gene</strong> Delivery EnhancesEx Vivo Antigen-Specific Cytotoxic T Lymphocyte Stimulation andKillingDazhi Zhang, Liu Yong, Chang Xuan You, Rongcheng Lou, Paul L. Hermonat.820Potent Transfer <strong>of</strong> Human CD40L to Chronic Myeloid Leukemia Stem<strong>Cell</strong>s Via an AdCD40L <strong>Gene</strong>tically Engineered Bystander <strong>Cell</strong> LineLisa H. Christiansson, Lina Liljenfeldt, Bengt Simonsson, Ulla Olsson-Strömberg, AngelicaS. I. Loskog.822Preclinical Efficacy Data <strong>of</strong> MGN1601, a Tumor Vaccine Comprising4-Fold <strong>Gene</strong>-Modified and Irradiated Allogeneic Tumor <strong>Cell</strong>s inCombination with a DNA-Based Immunomodulator for the Treatment<strong>of</strong> Metastatic Renal CarcinomaBaerbel Volz, Manuel Schmidt, Kerstin Kapp, Matthias Schr<strong>of</strong>f, Marina Tschaika,Burghardt Wittig.823Electrically Mediated Delivery <strong>of</strong> Plasmid DNA-Encoding Cytokines forthe <strong>Gene</strong>ration <strong>of</strong> an Enhanced Immune Response Against MelanomaBernadette Marerro, Shawna Shirley, Loree Heller, Richard Heller.824Cytokine-Based Log-Scale Expansion <strong>of</strong> Functional Human Dendritic<strong>Cell</strong>sYui Harada, Yasuji Ueda, Hiroaki Kinoh, Aki Furuya, Makoto Inoue, Mamoru Hasegawa,Tomohiko Ichikawa, Yoshikazu Yonemitsu.825Small Plasmid/PEI/Anionic Polysaccharide Ternary ComplexParticles: Toxicity and Animal Clinical StudyYoshiyuki Koyama, Masahiko Tojyo, Chieko Yoshihara, Katsuyuki Hamada, Tomoko Ito.826Imiquimod, a TLR7 Agonist, Delays Brain Tumor Growth throughSuppression <strong>of</strong> Treg in BrainZhengming Xiong, Shoko Toma, John Ohlfest.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010137Abstract DirectorySaturday, May 22, 2010Poster Session III — continued6:00 PM - 8:00 PMRoom: Exhibit Hall CCancer – Immunotherapy III827Strategy Escalation: An Emerging Paradigm for Safe ClinicalDevelopment <strong>of</strong> T <strong>Cell</strong> <strong>Gene</strong> TherapiesR. P. Junghans.Cancer-Oncolytic Viruses III828Combination <strong>of</strong> Promoter and microRNA Detargeting Is GreatlyAdvantageous in Attenuation <strong>of</strong> Adenoviral Replication in Hepatic<strong>Cell</strong>sJustyna Leja, Berith Nilsson, Di Yu, Magnus Essand.829Combining Oncolytic Herpes Simplex Virus and Chemotherapy: G47Dand Temozolomide Synergize in Killing Glioma Stem <strong>Cell</strong>sRyuichi Kanai, Hiroaki Wakimoto, Robert L. Martuza, Samuel D. Rabkin.830Transcriptional Targeting <strong>of</strong> an Oncolytic HSV-1 to Midkine-PositiveTumorsArturo R. Maldonado, Chuck Klanke, Anil G. Jegga, Bruce J. Aronow, Yonatan Y. Mahller,Timothy P. Cripe, Timothy M. Crombleholme.831Preclinical Studies Support the Efficacy <strong>of</strong> Prodrug Activator <strong>Gene</strong>Transfer in Experimental Glioma Models by Enhanced Replication-Competent Retrovirus VectorAkihito Inagaki, Kei Hiraoka, Christopher R. Logg, Hiroshi Matsumoto, Joan Robbins,Omar Perez, Harry Gruber, Douglas Jolly, Noriyuki Kasahara.832Intrapleural Administration <strong>of</strong> Third <strong>Gene</strong>ration Oncolytic HSV-1 (G47D) Is Highly Efficacious in Orthotopic Malignant PleuralMesothelioma ModelsMasamichi Takahashi, Yasushi Ino, Nobuhito Saito, Tomoki Todo.833Engineered Measles Viruses for Oncolytic <strong>Therapy</strong> <strong>of</strong> PancreaticCancerSascha Bossow, Christian Grossardt, Stefanie Sawall, Christoph Springfeld, AchimTemme, Roberto Cattaneo, Christ<strong>of</strong> von Kalle, Guy Ungerechts.834Incorporation <strong>of</strong> a Mutated E1A and a Chimeric 5/3 Fiber Improvedthe Therapeutic Efficacy <strong>of</strong> a Novel CRAd (Ad-F512) on DisseminatedOvarian CancerVeronica M. Lopez, Angel A. Rivera, Lorena Benedetti, Diego L. Viale, Kristopher J.Kimball, Minghui Wang, Alicia J. Bravo, Christine Dorvault, Joanne Douglas, Zeng B. Zhu,Ronald D. Alvarez, David T. Curiel, Osvaldo L. Podhajcer.835Inhibition <strong>of</strong> Autophagy Has Negative Impact on Virus ProductionIlya V. Ulasov, Atique U. Ahmed, Bart Thaci, Natalya V. Kaverina, Maciej S. Lesniak.836Armed and Transcriptionally Driven Oncolytic HSV-1 EnhancesAntitumor Efficacy In Vitro and In VivoJi-Young Yoo, Amy Haseley, Anna Bratasz, Kimerly Powell, Balveen Kaur.837Determining Adenovirus Titers Following Extraction <strong>of</strong> the Virus fromTumors in the Presence <strong>of</strong> Anti-Adenovirus AntibodiesDebanjan Dhar, Karoly Toth, William S. M. Wold.838 Abstract Withdrawn839The Use <strong>of</strong> Third <strong>Gene</strong>ration Oncolytic HSV-1 Expressing Luciferasefor Demonstration <strong>of</strong> Real-Time Biodistribution and DynamicsYihan Wu, Yasushi Ino, Tomoki Todo.840Highly Attenuated Vaccinia Virus as a Potential Oncolytic Agent forCancer VirotherapyTakafumi Nakamura, Minoru Kidokoro, Mina Hikichi, Hisatoshi Shida, Hideaki Tahara.841Enhanced Tumor Killing by a Mortalin Targeting Modified OncolyticAdenovirusJung-Sun Lee, Ji Young Yoo, Jihoon Ryu, Tomoko Yaguchi, Sunil C. Kaul, Renu Wadhwa,Chae-Ok Yun.842Increased Oncolytic Ability <strong>of</strong> a Double RGD-Modified Adenovirus inOvarian CancerLena J. Gamble, Qiana L. Matthews, Anton V. Borovjagin, David T. Curiel.Cancer-Targeted <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> III843Increasing the Efficacy <strong>of</strong> Monoclonal Antibody <strong>Therapy</strong> <strong>of</strong> BreastCancer through Transient Remodeling <strong>of</strong> Intercellular JunctionsJonas Persson, Max Drescher, ZongYi Li, Roma Yumul, Ying Liu, Hongjie Wang, AkseliHemminki, Pascal Fender, Andre Lieber.844Improvement <strong>of</strong> Cancer <strong>Gene</strong> <strong>Therapy</strong> by Incorporating a ChickenHypersensitive Site-4 Insulator into TSTA-Based Tumor-TargetingVectorChien-Fu Chen, Yu-Ping Sher, Mien-Chie Hung, Chia-Ling Hsieh.Saturday, May 22 nd


138<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectorySaturday, May 22 ndSaturday, May 22, 2010Poster Session III — continued6:00 PM - 8:00 PMRoom: Exhibit Hall CCancer-Targeted <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> III845Cytokine Induced Killer (CIK) <strong>Cell</strong>s for <strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> AcuteMyeloid Leukemia (AML): Improvement <strong>of</strong> Their Immune Activity byExpression <strong>of</strong> CD33-Specific Chimeric Receptors (CR)Virna Marin, Irene Pizzitola, Valentina Agostoni, Helene Finney, Alastair Lawson, MartinPule, Raphael Rousseau, Andrea Biondi, Ettore Biagi.846Exosome-Mediated Suicide <strong>Gene</strong> <strong>Therapy</strong> for Brain TumorsArda Mizrak, Senol Ozlem, Baris G. Ozdener, Leonora Balaj, Johan Skog, Xandra O.Breakefield, Okay Saydam.847Eradication <strong>of</strong> <strong>Therapy</strong>-Resistant Human Prostate Tumors Using anUltrasound Guided Site-Specific Cancer Terminator Virus DeliveryApproachPier Paolo Claudio, Rounak Nande, Mary A. Teter, Paul B. Fisher, Sarkar Denavand, LuigiClaudio, Adelaide Greco, Candace Howard.848Systemic Tumour Targeting with Orally Administered Non-PathogenicBacteriaMichelle Cronin, David Morrissey, Simon Rajendran, Douwe van Sinderen, Gerald C.O’Sullivan, Mark Tangney.849Transient Intratumoral Relaxin Expression Results in Tumor StromaDegradation and Improves Breast Cancer <strong>Therapy</strong> by HerceptinZongYi Li, Ying Liu, Roma Yumul, Max Drescher, Jonas Persson, Ines Beyer, Andre Lieber.850Developing a Multi-Modal <strong>Gene</strong>-Based Imaging and <strong>Therapy</strong> To TreatDisseminated Hormone Refractory Prostate CancerZiyue Jiang, Makoto Sato, Lily Wu.851Adenoviruses-Mediated Expression and <strong>Cell</strong>-Mediated Delivery <strong>of</strong>Interferon-Lambda Produced Anti-Tumor Effects to Human EsophagealCarcinoma In VivoMasatoshi Tagawa, Quanhai Li, Kiyoko Kawamura, Shinya Okamoto, Shan Yang, HiroshiKobayashi, Muneo Numasaki, Yuji Tada, Kenzo Hiroshima, Hideaki Shimada.852Inhibition <strong>of</strong> Angiogenesis and Suppression <strong>of</strong> Colorectal CancerMetastatic to the Liver Using the Sleeping Beauty Transposon SystemLalitha R. Belur, Kelly M. Podetz-Petersen, Brent Sorenson, Josh Parker, Cathy Carlson,Daniel Saltzman, Ramakrishnan Sundaram, R. Scott McIvor.853Plasmid AMEP, a New Efficient Antitumor <strong>Gene</strong> Transfer Product forthe Treatment <strong>of</strong> Metastatic MelanomasCeline Bouquet, Veronique Trochon-Joseph, Valerie Polard, Lluis M. Mir, MichelPerricaudet, Sophie Lebel-Binay.854Adenovirus-Delivered Short Hairpin-Structured RNA for MicrophageMigration Inhibitory Factor Silencing Inhibits Human Prostate Cancer<strong>Cell</strong> ProliferationLing Zhang, Anqi Wang, Chengyao Li, Hongwei Li, Leah N Braseth, Arseniy E Shabashvili,Colin Sumners.855Monitoring <strong>of</strong> Electroporation-Mediated Diphtheria Toxin AExpression in Prostate Cancer <strong>Cell</strong>s Grown in SCID MiceChristine Goepfert, Felix J. Frey, Brigitte M. Frey.856Clinical <strong>Gene</strong> Transfer in the Community: Ten Years <strong>of</strong> Local IBCReviewJan P. Vleck.Hematologic and Immunologic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> III857High Incidence <strong>of</strong> Vector Integration near Cancer-Related <strong>Gene</strong>swithin Primitive Hematopoietic Stem <strong>Cell</strong>s (HSC) after Fetal <strong>Gene</strong>Transfer with g-Retroviral VectorsJoe Tellez, Josh Wood, Graça Almeida-Porada, Chris D. Porada.858Successful Treatment <strong>of</strong> Canine Leukocyte Adhesion Deficiency Usinga MSCV-Promoter Based Foamy Viral Vector and Low-Dose BusulfanConditioningThomas R. Bauer, Jr., Erik M. Olson, Laura M. Tuschong, James M. Allen, Tanya H.Burkholder, David W. Russell, Dennis D. Hickstein.859<strong>Gene</strong>tically Engineered VIP-Expressing Dendritic <strong>Cell</strong>s Are Therapeuticin the Autoimmune Mouse Model <strong>of</strong> Experimental AutoimmuneEncephalomyelitisMiguel G. Toscano, Mario Skarica, Weiming Kong, Doina Ganea.860Long-Term Preclinical Evaluation <strong>of</strong> Lentiviral Vectors for SCID-X1<strong>Gene</strong> <strong>Therapy</strong> in a SCID-X1 Mouse ModelSheng Zhou, Suk See DeRavin, Zhijun Ma, Taihe Lu, John T. Gray, Harry L. Malech, BrianP. Sorrentino.861Transduction <strong>of</strong> Human CD34+ Derived NOG Repopulating <strong>Cell</strong>s withan Insulated SIN Lentiviral Vector for SCID-X1 Produced from aStable Producer <strong>Cell</strong> LineMichael R. Greene, Yoon-Sang Kim, Timothy Lockey, Paolo Fagone, Robert E. Throm,John T. Gray, Derek A. Persons, Brian P. Sorrentino.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010139Abstract DirectorySaturday, May 22, 2010Poster Session III — continued6:00 PM - 8:00 PMRoom: Exhibit Hall CHematologic and Immunologic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> III862Prevention <strong>of</strong> Atopic Dermatitis by Plasmid-Based SustainedTransgene Expression <strong>of</strong> Interferon g in NC/NGA MiceKayoko Hattori, Makiya Nishikawa, Kanitta Watcharanurak, Akihiko Ikoma, KenjiKabashima, Hiroyasu Toyota, Yuki Takahashi, Rei Takahashi, Yoshihiko Watanabe,Yoshinobu Takakura.863Expression <strong>of</strong> an Antibody Against CTLA4 from Tumor InfiltratingLeukocytes Induces Efficient Anti-Tumor Immune Responses in aMouse Model for Breast CancerJonas Persson, Ying Liu, Roma Yumul, ZongYi Li, Steve R<strong>of</strong>fler, Andre Lieber.864Foamy Virus Vectors with MGMT-P140K Mediate Efficient Selectionin a Canine Pyruvate Kinase Deficiency Model for HematopoieticStem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong>Brian C. Beard, Robert A. Wu, Christina Ironside, Punam Malik, Kiem Hans-Peter,Trobridge D. Grant.865Modeling <strong>of</strong> Congenital Erythropoietic Porphyria by RNA-Interference: A New Tool for Preclinical <strong>Gene</strong> <strong>Therapy</strong> EvaluationHubert de Verneuil, Elodie Robert-Richard, Magalie Lalanne, Isabelle Lamrissi-Garcia,Véronique Guyonnet-Duperat, Emmanuel Richard, Frédéric Mazurier, Cécile Ged, FrançoisMoreau-Gaudry.866<strong>Gene</strong> Corrected Neutrophils from Induced Pluripotent Stem <strong>Cell</strong>sDerived from an X-CGD Disease ModelSayandip Mukherjee, Giorgia Santilli, Michael P. Blundell, Elena Almarza, Juan Bueren,Adrian J. Thrasher.867The Comparison <strong>of</strong> Various Promoters for Wiskott Aldrich Syndrome<strong>Gene</strong> <strong>Therapy</strong> Using Insulated Lentiviral VectorsRachel M. Koldej, Deepti Banka, Gael Carney, Hui Fen Zhao, Derek Persons, Arthur W.Nienhuis.868Sleeping Beauty-Mediated Therapeutic <strong>Gene</strong> Transfer intoHematopoietic Stem <strong>Cell</strong>sKendra A. Hyland, Erik R. Olson, Xianzheng Zhou, R. Scott McIvor.869 (Moved to Poster Session I after Abstract 233)Myelospecific Self-Inactivating Gammaretroviral Vectors for p47 phoxCGD <strong>Gene</strong> <strong>Therapy</strong>Vital Wohlgensinger, Christian Brendel, Axel Schambach, Manuel Grez, JanineReichenbach, Reinhard A. Seger, Ulrich Siler.870Delivery <strong>of</strong> Soluble ICAM-1 in NOD Salivary Glands Can Result in B<strong>Cell</strong> Proliferation and Immunoglobulin ProductionJelle L. Vosters, Nienke Roescher, Paul P. Tak, John A. Chiorini.871Modulation <strong>of</strong> CXCR4 Expression by Novel Growth Factor Cocktail onStem <strong>Cell</strong>sSomayeh Shahrokhi, Kamran Alimoghaddam, Massoumeh Ebtekar, Zohre Sharifi,Seyed Hamidolah Ghaffari, Ali Akbar Pourfathollah, Mahboobeh Mohseni, ArdeshirGhavamzadeh.Lung and Respiratory Disease <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>872Efficient <strong>Gene</strong> Delivery to Pig Airways Using Helper-DependentAdenoviral VectorsHuibi Cao, Jonathan C. Yeung, Cathleen Duan, Allan Coates, Kitty Leung, Shaf Keshavjee,Jim Hu.873SIV Vector Pseudotyped with SeV-F/HN Envelope Proteins ProducesLong Lasting Expression in the Murine Lung, Is Readministrable andTransfects Human Airway ModelsUta Griesenbach, Makoto Inoue, Cuixiang Meng, Andrea M. Brum, Raymond Farley,Nikki Newman, Eiji Akiba, Mamoru Hasegawa, Eric W. F. W. Alton.874Airway Lentiviral <strong>Gene</strong> Transfer In MarmosetsDavid Parsons, Richard Bright, Darren Miller, Don Anson.875Pulmonary Delivery <strong>of</strong> mRNA: In Vitro and In Vivo EvaluationOliwia Najder, Joanna Rejman, Stefaan De Smedt, Joseph Demeester, Mario VanPoucke, Luc Peelman, Niek N. Sanders.876Inhibiting Bap31 by Dominant Negative p20 Does Not Promote CFTRTranslocation to the <strong>Cell</strong> MembraneYadong Zhang, Wei Zhang, Min Li, T. C. Hwang, Yihong Ye, Dongsheng Duan.877Targeted siRNA Delivery to Lung Epithelia Reduces Mucus Secretionin a Mouse Model for Allergic AsthmaHyo Sung Choi, Jae Wook Jung, Hong Seok Ban, Nahyun Kim, Priti Kumar, Sang kyungLee.878The T1a Promoter Mediates Nuclear Import <strong>of</strong> Plasmid DNA intoAlveolar Epithelial Type I <strong>Cell</strong>sLynn F. Gottfried, David A. Dean.Saturday, May 22 nd


140<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectorySaturday, May 22 ndSaturday, May 22, 2010Poster Session III — continued6:00 PM - 8:00 PMRoom: Exhibit Hall CLung and Respiratory Disease <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>879Effects <strong>of</strong> LPC on Lung Mechanics in Non-Human PrimatesP. Hiatt, R. McConnell, N. Grove, D. Dang, N. Brunetti-Pierri, D. Palmer, A. Beaudet, P.Ng.880Repeat Aerosol Delivery <strong>of</strong> Concentrated PEI/pDNA to the SheepLungGerry McLachlan, Lee A. Davies, Cath M. Gordon, Christina Vrettou, Eilidh Baker, PeterTennant, Alison Baker, Rebecca L. Coles, Dominique McCormick, Stephanie G. Sumner-Jones, Stephen C. Hyde, Deborah R. Gill, D. David S. Collie.881Pseudotype Switching as a Strategy To Permit Efficient Re-Administration <strong>of</strong> Lentiviral Vectors to Mouse AirwaysJohn C. Olsen, Angela Giddings, Manij Patel.Oligonucleotide & RNAi Therapeutics III882Antinociceptive Potentiation and Attenuation <strong>of</strong> Tolerance byIntrathecal b-Arrestin 2 siRNA in RatsChung-Ren Lin, Kuan-Hung Chen, Chih-Hsien Wu, Yi-Sen Chen.883siRNA Cationic Delivery Systems for Therapeutic ApplicationsAnne-Laure D. Bolcato-Bellemin, Marie-Elise Bonnet, Gaëlle Deglane, Nathalie Lenne,Patrick Neuberg, Clément Paris, Jean-Paul Behr, Patrick Erbacher.884Therapeutic Effect <strong>of</strong> Intravenous Administration <strong>of</strong> New Type HybridStaple-Modified NFkB Decoy Oligonucleotides on InflammatoryBowel Disease Model MiceKazunari Ozaki, Hir<strong>of</strong>umi Makino, Takashi Miyake, Yasushi Takeya, Motokuni Aoki,Hiromi Rakugi, Ryuichi Morishita.885Artificial miRNA Clusters for Targeting Multiple Regions <strong>of</strong> the HCVGenomeXiao Yang, Virginia Haurigot, Shangzhen Zhou, Linda B. Couto.886Design and Pre-Clinical Evaluation <strong>of</strong> Adeno-Associated Viral (AAV)Vectors for Therapeutic Inhibition <strong>of</strong> Disease-Associated miRNAsNina Schürmann, Ellen H<strong>of</strong>fmann, Dirk Grimm.887Safety Results <strong>of</strong> a Phase 1 Clinical Study for Treatment <strong>of</strong> Patientswith Advanced Solid Tumors with the TLR9 Agonist MGN1703Max E. Scheulen, Manuel Schmidt, Michael S. von Bergwelt-Baildon, Ulrich Hacker,Alexander Shimabukuro-Vornhagen, Heike Richly, Bahar Nokay, Marina Tschaika,Burghardt Wittig, Martin R. Weihrauch.888Parallel Assessment <strong>of</strong> Silencing Potency <strong>of</strong> Conventional SiRNAs vs.SiRNA VariantsNicholas M. Snead, Min-sun Song, Kumi Sakurai, John J. Rossi.889Inhibition <strong>of</strong> Viral Hepatitis with Non-Coding RNAsJovanna Gonzalez-Rojas, Amaya Abad, Lorea Blazquez, Nerea Razquin, Xabier Abad,Puri Fortes.890Screening <strong>of</strong> Nature Compounds for Enhancing AntisenseOligonucleotide-Induced Exon Skipping <strong>of</strong> Dystrophin <strong>Gene</strong> In Vitroand In VivoBo Wu, Caryn Cloer, Peijuan Lu, Mingxing Wang, Wei Li, Kazuo Koike, Qi Long Lu.891A Combination <strong>of</strong> PHD2 Prolyl Hydroxylase Silencing andBiocompatible Polyplex Nanocarrier Enhances TherapeuticAngiogenesisShourong Wu, Nobuhiro Nishiyama, Keiji Itaka, Takuya Hashimoto, Hiroyuki Koyama,Mitsunobu R Kano, Kazunori Kataoka.892Development <strong>of</strong> a Novel, RNAi-Based Therapeutic TargetingPancreatic Duodenal Homebox-1 (PDX-1) for Insulinoma andPancreatic CancerZhaohui Wang, Shi-he Liu, Donald D. Rao, Phillip B. Maples, Neil Senzer, JohnNemunaitis, F. C. Brunicardi.893Systemic Delivery <strong>of</strong> Highly Active Functionalized Lipopolyamine-Formulated siRNA to Murine Lung with Reduction <strong>of</strong> Inflammation byDexamethasone Pre-TreatmentJeff Sparks, Kevin J. Polach, Richard T. Congo, Gregory Slobodkin, Angela Rea-Ramsey,Casey Pence, Majed Matar, Diane McClure, Jennifer Rice, David Ulkoski, ElaineBrunhoeber, Leslie Wilkinson, Khursheed Anwer, Jason G. Fewell.894Ornithine and Tryptophan as an Efficient Polycation for siRNADelivery to Tumor <strong>Cell</strong>sYusuke Sato, Hiroto Hatakeyama, Hideyoshi Harashima.895Influence <strong>of</strong> 5’ Terminal Triphosphate in Short Hairpin RNA TargetingHCV Replicon on Antiviral Activity and Innate Immune ResponseKyungbo Kim, Bokhui Lee, Soo-Ryoon Ryoo, Dal-Hee Min, Dong-Eun Kim.896Enhanced Delivery <strong>of</strong> GFP siRNA Using New Amino Lipid-BasedCationic LiposomesHyun-Ki Kim, Enkhzaya Davaa, Chang-seon Myung, Jeong-Sook Park.EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010141Abstract DirectorySaturday, May 22, 2010Poster Session III — continued6:00 PM - 8:00 PMRoom: Exhibit Hall COligonucleotide & RNAi Therapeutics III897<strong>Gene</strong> Editing Activity in C. elegans Directed by Single-StrandedOligonucleotidesKerry A. Falgowski, Eric B. Kmiec.Stem <strong>Cell</strong> Therapies898Engraftment into the Liver <strong>of</strong> Transplanted Adipose Tissue-DerivedStromal <strong>Cell</strong>s Detected by Noninvasive Bioluminescence ImagingGiuliana Di Rocco, Antonietta Gentile, Annalisa Antonini, Maurizio C. Capogrossi, GabrieleToietta.899Establishment <strong>of</strong> iPS <strong>Cell</strong>s Derived from the New World Monkey <strong>of</strong>Common MarmosetSaori Yamaguchi, Tomotoshi Marumoto, Hirotaka Kawano, Shinya Shimoda, YoshieMiura, Tomoko Inoue, Ryo Kurita, Hiroyuki Miyoshi, Erika Sasaki, Atsushi Takahashi,Kenzaburo Tani.900Umbilical Cord Blood-Derived Mesenchymal Stem <strong>Cell</strong>s AcquireCardiac Phenotype with Oxytocin Treatment and Enhance CardiacRecovery after Myocardial InfarctionYong Sook Kim, Jin Sook Kwon, In-Kyu Park, Myung Ho Jeong, Youngkeun Ahn.901Ex Vivo Expansion and Enrichment <strong>of</strong> Murine Adult Neural Stem <strong>Cell</strong>sfor <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> Neurogenetic DiseasesSushma Chaubey, John H. Wolfe.902Dual Effect <strong>of</strong> Mesenchymal Stem <strong>Cell</strong>s <strong>Gene</strong>tically Modified with aSingle-Chain Antibody Against EGFRvIII on the Targeting and Growth<strong>of</strong> EGFRvIII Expressing Experimental Glial TumorsIrina V. Balyasnikova, Sherise D. Ferguson, Yu Han, Maciej S. Lesniak.903Expression <strong>of</strong> Coagulation Factors from Murine iPS-Derived Liver<strong>Cell</strong>sKohei Tatsumi, Yoshihiko Sakurai, Kazuo Ohashi, Shogo Kasuda, Junko Kato, TeruoOkano, Midori Shima.904The Granules Isolated from Mast <strong>Cell</strong>s Have Increased the Survival<strong>of</strong> Bone Marrow-Derived Mesenchymal Stem <strong>Cell</strong>sJin Sook Kwon, Yong Sook Kim, In-Kyu Park, Myung Ho Jeong, Youngkeun Ahn.905Large-Scale Expansion <strong>of</strong> Undifferentiated Human Embryonic Stem<strong>Cell</strong>s in Serum-Free Suspension CultureVincent C. Chen, Sylvana Couture, Jonathan Auerbach, Araz Toumadje, Lara Ausubel,David Hsu, Larry Couture.906Enrichment <strong>of</strong> Bone Marrow Stem <strong>Cell</strong>s In Vivo by Dissociation- andProliferation-Inducing Compounds Augments Bone Growth in aMouse Segmental Bone Defect ModelSanjay Kumar, Selvarangan Ponnazhagan.907A Delayed Treatment <strong>of</strong> Mesenchymal Stem <strong>Cell</strong>s Is Effective inRescuing Mice with Established LPS-Induced Acute Lung InjuryShirley H. J. Mei, Colleen H. Parker, Conrad Liles, Duncan J. Stewart.908Lentiviral Vector-Mediated Transduction in Murine SMA EmbryonicStem <strong>Cell</strong>s Derived from Spinal Muscular Atrophy (SMAI) MiceArianna Malgieri, Andrea Luchetti, Massimo Sanchez, Maria Bellocchi, Paola Spitalieri,Douglas A. Kerr, Giuseppe Novelli, Federica Sangiuolo.909Development <strong>of</strong> a Reporter System for I-SceI-Mediated GenomeEditing in Canine Hematopoietic Stem <strong>Cell</strong>sNina M. Muñoz, Grant D. Trobridge, Brian C. Beard, Byoung Y. Ryu, Andrew M.Scharenberg, Hans-Peter Kiem.910Liver Disease Correction with <strong>Cell</strong>s Isolated from Cadaveric LiverLaura Erker, Hisaya Azuma, Laura Eaton, Eric Benedetti, Bryan Jensen, Andrew Y. Lee,Milton Finegold, Holger Willenbring, Markus Grompe.911Efficient <strong>Gene</strong> Transfer into CD34+ Hematopoietic Stem <strong>Cell</strong>s UsingClinical Grade Lentiviral Vector Expressing the Human Beta-Globin<strong>Gene</strong>Clare Taylor, Leda Ferro, Jason Plotkin, Xiuyan Wang, Michel Sadelain, Isabelle Rivière1.912Embryonic Stem <strong>Cell</strong> <strong>Therapy</strong> Enhances Tendon Regeneration in aLarge Animal Flexor Tendonitis ModelAlan J. Nixon, Ashlee A. Watts, Oleg Kopyov.913<strong>Gene</strong>ration <strong>of</strong> Cystic Fibrosis Induced Pluripotent Stem (iPS) <strong>Cell</strong>s for<strong>Gene</strong> <strong>Therapy</strong>R. G. Sargent, Y. Yang, A. Lee, S. Kim, L. Ye, J. Chang, D. C. Gruenert.Saturday, May 22 nd


142<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectorySaturday, May 22 ndSaturday, May 22, 2010Poster Session III — continued6:00 PM - 8:00 PMRoom: Exhibit Hall CLate Breaking Science III938Blockage <strong>of</strong> Viral RNA Transport <strong>of</strong> REV- and CTE-DependentLentiviral Vectors Encoding NUP98 Fusion <strong>Gene</strong>sYildirim Dogan, David C Dorn, Sutapa Sahay, Anna Franceschino, Malcolm AS Moore.939Novel Helper-Independent piggyBac Plasmids for <strong>Gene</strong> DeliveryApproachesJohann Urschitz, Chad Walton, Jesse Owens, Miyuri Kawasumi, Hideaki Yamashiro, IlkoStoytchev, Joel Marh, Craig J. Coates, Joseph M. Kaminski, Pawel Pelczar, Ralph V.Shohet, Stefan Moisyadi940Transgene Trafficking to Nuclear Periphery is a Barrier to Transfectionin a Lipid Substituted Nonviral Cationic PolymerCharlie Y-M Hsu, Hasan Uludag.941Studies on the Biodistribution Evaluation <strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong> Productsusing the Real Time-PCRSong Hyun, Choi Min Joung, Seo Su Jin, Han Sung Yeol, Lee Yeon Ho, Park Youn Joo,Ahn Kwang Soo, Kim Tae Geun.942Activity, Integration Preferences, and Localization <strong>of</strong> TcBuster; aNovel hAT Transposon SystemLauren E. Woodard, Aparna Kaja, Robert H. Hice, Peter W. Atkinson, Nancy L. Craig,Matthew H. Wilson.943Overcoming the Lip<strong>of</strong>ection Resistance <strong>of</strong> Oral Cancer <strong>Cell</strong>s:Polyplexes and AdenovirusesSenait Gebremedhin, Krystyna Konopka, Ilya Ulasov, Maciej Lesniak, Nejat Düzgünes.944Association <strong>of</strong> CD44 Expression with Hormone Receptor Status inBreast CancerP Olivera, E Ortiz, R Romero, A Palacios, R Morales, G Cervantes, S Galván, E Langley,A García-Carrancá.945Systemic Apoptotic <strong>Gene</strong> <strong>Therapy</strong> for Pancreatic Cancer: Multi-DoseToxicity Study <strong>of</strong> the BikDD NanoparticleK Barnhart, M Javle, S Miller, J Hsu, J McMannis, P Smith, J Abbruzzese, MC Hung.946Phosphatidylserine Immobilization <strong>of</strong> Lentivirus for Localized <strong>Gene</strong>TransferSeungjin Shin, Hannah M. Tuinstra, David M. Salvay, Lonnie D. Shea.947Engineering Adenovirus Serotype 5 Vectors to Increase HepatocyteTransduction for Liver <strong>Gene</strong> <strong>Therapy</strong>Reeti Khare, Michael A. BarryEXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 143Abstract Author IndexAAagaard, Lars. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 695Aalbers, Caroline J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 66Aartsma-Rus, Annemieke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 153Abad, Amaya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 889Abad, Xabier . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 889Abay, K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777Abbasi, Meysam. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 562, 568Abdelbary, Hesham . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39Abdulrasul, Fouzia. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 349Abrameto, Javier A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616Abu-Asab, Mones . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 135Acharya, Bishnu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 216Adachi, Kei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 7, 703Adair, Jennifer E.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 294Adamo, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 323Adamo, Richard F.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 425Adams, Marvin E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 584Adams, Samuel J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 622Adriaansen, Janik . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 761Affin, Lulit A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 125Aflaki, Pejman . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 237Agbandje-McKenna, Mavis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 65Agerholm-Larsen, Birgit . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 109Agkatsev, Sarina. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 602Agostoni, Valentina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 845Agudo, Judith. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 140, 762Ahlén, Gustaf . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 111Ahlen, Gustaf . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 289Ahlzadeh, Garbielle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 475Ahmad, Farizan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 212, 512Ahmad, Sarfraz . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 812Ahmed, Atique U. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 191, 718, 835Ahmed, Nabil . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 179, 470, 471Ahmed, Sherif O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 451Ahn, Miwon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 52Ahn, Youngkeun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 399, 751, 900, 904Ahouansou, Ornella. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19Ahrens, Eric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 271Ahtiainen, Laura . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 197, 291Aimi, Junko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 35Airavaara, Mikko T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 709Airenne, Kari J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 669Aiuti, Alessandro. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225, 229, 276,. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295, 345Akbulut, Hakan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 85Akiba, Eiji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 873Akita, Hidetaka . . . . . . . . . . . . . . . . . . . . . . . . . . . . 246, 253, 401, 591, 743Akkina, Ramesh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 287Alba, Raul . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 380, 573Albelda, Stephen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 611Alberici, Luca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 644Albrecht, Douglas E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 796Al-Dahhak, Roula . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155Alden, Anderson . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 181Aldhamen, Yasser A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 638, 651Aldrich, Carla J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 332Alemany, Ramon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 291, 496Alexander, Ian E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 409Alfaro, Carlos . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42Alferiev, Ivan S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 425Ali, Nowrouzi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 138Alimoghaddam, Kamran. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 871Allain, Jean-Pierre . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 808Allay, James A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 668Allen, Cornell . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 675Allen, Cory. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 490, 499Allen, Henry. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 536, 586Allen, James M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 234, 459, 786, 858Allen, Lee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 576Allen, Michael H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 122, 125Allen, Phil . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 392Allimperti, Stella . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 358Allocca, Mariacarmela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 64Almarza, David. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 351Almarza, Elena. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 866Almeida-Porada, Graça. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 551, 857Alton, Eric W. F. W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 873Alvarez, Ronald D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36, 503, 507, 834Alves, Paula M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 278, 570, 579Alzadeh, Gabrielle. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 181Alzuguren, Pilar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 810Amalfitano, Andrea . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 638, 651Amaral, Ana I. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 278Amarnath, Shoba . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 341Ambrosi, Alessandro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 345, 346Amendola, Mario . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 413An, Weijun. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 603Anagnostopoulos, Achilles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137Anagnou, Nicholas P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 338Anderson, Jonathan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279Anderson, Joseph . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 281Anderson, Susan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 300Ando, Dale. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 783, 798Andre, Kris. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 41, 803Andreadis, Stelios . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 359Andreadis, Stelios T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 358, 282Andreani, Marco . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 319


144<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexAndresen, Thomas L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 511Angell, Yu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 731Anguela, Xavier M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 140Anikster, Yair . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 144Annan, Anand C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 503Annoni, Andrea. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 545Annunziata, Patrizia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 758Anson, Don . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 874Anson, Donald . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 621Ansorge, Sven . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 361Antonini, Annalisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 898Antoniou, Michael N.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 531Anwer, Khursheed . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893Aoki, Motokuni. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 884Aoki, Yoshitsugu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 565Appledorn, Daniel M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 638, 651Aramaki, Yukihiko. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 102Aras, Rahul A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 426, 438Arbuthnot, Patrick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 82, 563Ardiani, Andressa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 330Arens, Anne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 286, 305, 347Ariga, Tadashi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678Arkin, Adam P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 802, 805Arman, A. Cyrus . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 21Armaroli, Annarita. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258Armstrong, Adam . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 633Arnett, Andrea L.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 461Arning, E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139Arntz, Onno J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 559, 627Aronovich, Elena L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 325, 728Aronow, Bruce J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 830Arruda, Valder R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 308, 551, 614, 615Arsenau, James . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 35Arshad, Shazia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 530Arstila, Petteri. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 538Arun, Srivastava . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 68Arzberger, Silke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312Asakawa, Nagisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 410Asdourian, George. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 444Asefa, Benyam. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 165Aslanidi, George . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 363, 370, 378Asokan, Aravind . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 62, 160, 364, 367, 403Assi, Hikmat . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26, 181Astone, Dalila. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 435Astrakhan, Alexander. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 343, 529Athanassiadou, Aglaia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 340, 729Atkins, Harry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39Aubert, Dominique . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 138Aubourg, Patrick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19Audet, Sarah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 402Auerbach, Jonathan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 905Aurelian, Laure . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 717Auricchio, Alberto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59, 64, 443, 624, 758Aurigemma, Rosemarie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36Ausubel, Lara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 905Ausubel, Lara J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279Aviles, Misael O.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 121Ayuso, Eduard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 762Azuma, Hideki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 397Azuma, Hisaya. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 910bBacci, Maria Laura . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59Back, Jin Ee. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 128Badding, Melissa A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 588Baek, Jin Ee. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 406, 754Bagarazzi, Mark. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 477Bahar, Bahar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 664Bahou, Wadie F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 372Bailey, Debra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 739Bais, Anthony J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 27Bajzer, Zeljko. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 208Bak, Rasmus O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 695, 726Bakay, Marina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 425Baker, Alison . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 880Baker, Andrew H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 380, 715, 723Baker, Andrew J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 783Baker, Eilidh. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 880Baker, K. Scott . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 232Balaj, Leonora . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 846Baldim, Victor . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 485Balducci, Anthony G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 271Bali, Deeksha. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 136Bali, Pawan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678Ball, Claudia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612Ballmaier, Matthias . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 318Balyasnikova, Irina V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 902Balyasnikova, Irina Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 191Balzer, Frederick. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156, 454Ban, Hiroshi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 303, 634Ban, Hong Seok . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 877Ban, Hong-Seok . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 407Bandyopadhyay, Sarmistha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 711Banfi, Andrea . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 430, 597Banka, Deepti . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 867Banks, Glen B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 459, 584Banwell, Brenda . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155Baranov, Vladimir . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 57EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 145Abstract Author IndexBaranov, Vladislav S.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 750Baranyi, Lajos . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 527Barbas, III, Carlos . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 340Barbas, III, Carlos F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 94, 339Barber, Glen. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 604Bareja, Akshay . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 466Barlow, Robert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17Barnes, Justin A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 503Barohn, Richard J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155Baron, Stéphanie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 226Barouch, Dan H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 723Barron, Valerie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 439Barry, Frank. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 270Barry, Michael A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 81Bartek, Jiri. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 265Bartholomae, Cynthia C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297,. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 298, 345, 346, 347Bartido, Shirley . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 32Bartlett, Jeffrey S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 547, 699Bartoli, Marc . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 48Barve, Minal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 292, 335Barzel, Adi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16Basner-Tschakarjan, Etiena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 43Bassi, Elena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 666Basu, Gaurav . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 107Batchu, Ramesh B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250Batchvarova, Milena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 667Batshaw, Mark L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 416Batsis, Ioannis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137Bauer, Gerhard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 281Bauer, Jr., Thomas R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 234, 858Bauerschmitz, Gerd. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 193Baum, Bruce J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 74, 77, 143, 761Baum, Christopher . . . . . . . . . . . . . . . . . . . . . . 284, 302, 304, 318, 528, 619Baumhover, Nicholas J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 117, 594Bazzani, Reto P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 739Beal, Nikia L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445Bear, Adham . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 469Bear, Adham S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 816Beard, Brian C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 294, 864, 909Beaudet, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 879Beaudet, Arthur . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 639Beaudoin, Esther L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 27Becher, Ulrich M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 693Bechinger, Burkhard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 400Becker, Pamela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137, 294Bedell, Cynthia. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 292, 335, 536Beheshti, Ben. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 217Behr, Jean-Paul. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 883Behr, Michael. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 381Belicchi, Marzia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 460, 464, 795Bell, Christie L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 65Bell, Jason B.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 325, 728, 734Bell, John C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33, 37, 39Bell, Peter L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 45, 412, 416, 543, 759Bellavia, Diego . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 599Bellayr, Ian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 465Bello, Alexander J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 64Bellocchi, Maria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 908Beloglazova, Irina. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 434Belousova, Natalya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 725Belur, Lalitha R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 20, 852Benati, Claudia. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276Benderdour, Mohamed . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 756Bendjelloul, Mehdi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 463Benedetti, Eric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 910Benedetti, Lorena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 834Benedicenti, Fabrizio. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297, 298Benes, Vladimir . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 270Bengtson, Karen. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 803Benito, Alberto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42Bennett, C. F.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55Bennett, C. Frank . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 557Bennink, Miranda B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 559, 627Benvegnu, Thierry. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120Benza, Raymond L.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 104Berger, Carolina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 618Bergmann, Juri. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 237Bernard, Robert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162Berraondo, Pedro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 485, 810Berry, Charles C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 13Bertin, Terry K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 642Bertolotti, Roger . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 244Bertrand, Emilie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 395Besarab, Anatole . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616Besharse, Joseph C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17Betts, Michael R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 164Beuerlein, Michele . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 280Beyer, Ines . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 264, 849Bhasvar, Janak. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 269Bhatnagar, Rakesh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 562Bhavsar, Janak. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 272Biagi, Ettore. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 845Bian, Ang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 389Bian, Yixin. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 806, 808Bianco, Paolo. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 133Biasco, Luca. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225, 345Bienemann, Alison . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 394


146<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexBien-Willner, Ricardo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155Biffi, Alessandra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276, 313, 315, 316Bigey, Pascal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 791Bikram, Malavosklish . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 405Billiet, Ludivine. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 395Binder-Scholl, Gwendolyn. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 798Bindra, Ranjit S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 50, 285Biondi, Andrea . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 845Bird, Andrew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 142Bisgrove, Dwayne. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 674Bish, Lawrence. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 433, 625Bisordi, Katie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 630Biswas, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223, 697Bizario, João C. S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 790Black, Margaret E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 330Blake, Bonita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 355Blake, Bonita L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 2Blanchard, Jan E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39Blanco, Laura. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 485, 708Blasczyk, Rainer . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612Blau, Carl A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 730Blazar, Bruce R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 221Blazquez, Lorea . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 889Blechingberg, Jenny . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 91Blick, Gary. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 646Blits, Bas. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 249Blondeau, Johanna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 229Blundell, Michael P.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 866Blundell, Mike . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 229Boccalatte, Francesco . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 337Bochner, Bernard H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 334Boczkowski, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 561Bodine, David M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 521Bogan, Janet . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 454, 458, 467, 785Böhm, Marie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612Boisgérault, Florence. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 48Bolcato-Bellemin,Anne-Laure D.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 883Bollard, C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30Bollard, Catherine . . . . . . . . . . . . . . . . . . . . . . . . . . . 29, 288, 293, 471, 473Bolund, Lars. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 91Bolyard, Chelsea M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 547Bomsztyk, Karol . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 93Bondanza, Attilio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295, 654Bonetti, Ciro. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 624Bonfiglio, Serena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 795Bonini, Chiara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 88, 295, 346, 632, 654Bonner, Melissa A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 569Bonnet, Marie-Elise . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 883Bonten, Eric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 449Bookay, Haddas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616Bordignon, Claudio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276, 295, 654Borenstein, Ronen. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 719Borisenko, Grigory G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 735Borovjagin, Anton V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 842Borquez-Ojeda, Oriana. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 32, 184Bortolanza, Sergia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 494Bosch, Assumpció . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 573Bosch, Fàtima . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 140Bosch, Fatima . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 762Bosma, Piter J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 138Boss, Hannah. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 27Bossow, Sascha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 833Bosticardo, Marita. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225Bostick, Brian P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 601Botchkina, Galina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 266Bottiglieri, T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139Bottinelli, Roberto. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 795Boulet, Sabrina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777Boulis, Nicholas M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 699, 778, 782Bouquet, Celine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19, 110, 113, 853Bourdel, Alix . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 87Bourke, Michael G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 736Boutin, Sylvie. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 67Bovolenta, Chiara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 644Bovolenta, Matteo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258Bowers, William J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 18, 23, 76Bowles, Dawn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 581Bowles, Dawn E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 366Boyd, Surina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 164Boyden, Edward S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 21Boye, Sanford L.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17, 71Boye, Shannon. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 71Boye, Shannon E.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17, 21Boyer, Jean . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 477Boyle, Kristy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 267, 461Boztug, Kaan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612Bradley, Allan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 15Bradshaw, Angela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 723Bradshaw, Angela C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 380, 715Brady, James . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 675Brady, Troy L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 13Brambilla, Riccardo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 315Bramson, Jonathan L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 79Braseth, Leah N.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 518Braseth, Leah N . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 854Bratasz, Anna. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 836Braun, Stephen E.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 645EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 147Abstract Author IndexBravo, Alicia J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 834Brayman, Andrew A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 326Breakefield, Xandra O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 57, 665, 846Bredius, Robbert. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225Breitbach, Caroline . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33Bremer, William . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149Bremer, William G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 290Brendan, Lee H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 642Brendel, Christian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 869Brenner, Malcolm K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 28, 29, 30,. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 178, 288, 293, 672, 799Brennig, Sebastian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 528Brentjens, Renier J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 32, 183, 815Breous, Ekaterina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 543Bresolin, Nereo. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 464, 795Breuzard, Gilles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 395Bridges, Charles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156, 454Bridges, Charles R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 600Bright, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 874Brigida, Immacolata . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295, 345Brioschi, Simona. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258Brittebo, Eva . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 453Broccoli, Vania . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 632Broderick, Jennifer . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 362Broderick, Kate E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 103, 115, 327Brown, Brian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 613Brown, Eric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 391Brown, Eric D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39Brown, Eric J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 497Brown, Ryan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39Brown, Talmage T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 142Browne, Andrew W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 201Broxtermann, Mathias. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312Brugiere, Pierre . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777Brugman, Martijn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284Brugman, Martijn H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 619Brum, Andrea M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 873Brumbach, Jonathan H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 126Brunetti-Pierri, N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 879Brunetti-Pierri, Nicola. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 75, 388, 639, 677Brunhoeber, Elaine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893Brunicardi, F. C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 247, 564, 731, 892Bryan, Nathan S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 677Bryant, Christopher S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250Bu, Jie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55Buchlis, George . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 546Buening, Hildegard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312Bueren, Juan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 866Büning, Hildegard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 5Bunnell, Bruce . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314Bunnell, Bruce A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 411Buñuales, Maria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 494Burel Deschamps, Laure. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 745Burgess, Kevin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 731Burke, James. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33Burke, James M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 35Burke, Kathleen C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11Burkholder, Tanya H.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 234, 524, 858Burnett, John C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 599, 805Burnette, Angela D.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 667Burnnet, Erin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 767Burris, Clark A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 76Bushman, Frederic D. . . . . . . . . . . . . . . . . . . . . . . . . . 13, 228, 283, 308, 346Bussadori, Giulio. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 308Busuttil, Ronald W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 44, 224, 373Butler, Greg . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 488Butler-Browne, Gillian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 789Byrne, Barry J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 290, 371, 378, 596Byrnes, Andrew P.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 542CCaccavelli, Laure. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 229Cafferata, Eduardo G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 609Cai, Hancheng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 200Cai, Shangde . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 185Cai, Ying . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 739Calabrese, Christopher . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 449Calcedo, Roberto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 45, 164Callejas, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 140Campbell, Jarryd M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 599Campbell, Katherine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149, 581Campbell, Katherine J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155, 290Campeau, Philippe M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 677Campion, C. A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 194Cancelas, Jose A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 526Candolfi, Marianela. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26, 181, 475Candotti, Daniel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 808Candotti, Fabio. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 227, 296, 678Cannon, Paula M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11Cantore, Alessio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297, 545, 613Cantz, Tobias . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284, 528Cao, Huibi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 872Cao, Maohua . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 429, 436, 437, 711Cao, Ou. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 146, 763Capalbo, Anita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 758Capogrossi, Maurizio C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 898Cappelli, Barbara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225Carbonaro, Denise A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296, 414


148<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexCardoso, Rosana. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 349Carillo-Carrasco, Nuria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 679Carlson, Cathy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 852Carmoy, Nathalie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120, 131, 745Carnathan, Diane G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 164Carnes, Aaron E.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 572, 575Carney, Gael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 867Carpenter, Kevin H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 409Carr, Bob. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 577Carragher, Bridget. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 393Carrer, Alessandro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 431Carroll, Richard. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 798Carrondo, Manuel J. T.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 278, 579Carrum, George . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288, 293Cartier, Nathalie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19Carvalho, Leonardo P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 440Carville, Angela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 645Casellas, Alba. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 140, 762Casey, Garrett . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 812Cashman, Siobhan M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 447, 448Casimir, Colin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 349Casorati, Giulia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 654Cassani, Barbara. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 617Castiello, Maria C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225Castro, Maria G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26, 181, 475Cataldi, Marcela P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 307Cataliotti, Alessandro. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 599Cathomen, Toni . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16, 95Cattaneo, Roberto. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 168, 833Cattoglio, Claudia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 346, 523Cavazzana-Calvo, Marina. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 13, 229, 617Cawley, Niamh X. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 143Cela, Racel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 46Cela, Racel G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 224, 642Cerri, Federica . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 315Cersosimo, Stefania . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 133Certo, Michael T.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 90Cerullo, Vincenzo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 177, 193, 197,. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204, 291, 538, 549, 642, 658Cervio, E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 360Cesana, Daniela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297, 298, 632Cesaro, P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777Chéradame, Hervé . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 395Cha, Yeon-Sook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818Chae, Jin-A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818Chamala, Sreedhar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250Chamberlain, Jeffery . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 553Chamberlain, Jeffrey S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 267, 459,. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 461, 466, 468, 584, 786, 792Chamberlain, Marc . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 294Champlin, Richard E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479Chan, Emma . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 647Chan, J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223, 697Chan, Janet . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 640Chan, Jerry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 531Chand, Allan R.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 64Chanda, Diptiman . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 63Chandler, Randy J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 135, 679, 701Chandra, Pallavi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 127Chandra, Ravi K.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 262Chandrasekaran, Suma . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 679Chandrashekran, Anil. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 349Chang, Chung-Che. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 293Chang, Esther. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 536Chang, J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 913Chang, Yu-Han . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 583Channon, Keith M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 602Chaouch, Soraya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 789Chapdelaine, Pierre . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 462, 790Charan, Rakshita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 455Charrier, Sabine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 229Chase, Lucas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 268Chatterjee, Anindo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 127Chatterjee, Saswati. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 3Chaubey, Sushma . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 901Chekmasova, Alena A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 183Chen, Baihong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 219Chen, Chien-Fu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 844Chen, Christopher Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 81Chen, Dong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 605Chen, Fang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 130Chen, Haifeng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 72Chen, Heng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 389Chen, Hong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 172Chen, Jesse . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 467Chen, Jessie. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156Chen, Jianzhu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 531Chen, Kuan-Hung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 882Chen, Li. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 574Chen, Liping. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 326Chen, Margaret . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 289Chen, Shu-Jen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 625Chen, Thomas C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 200Chen, Vincent C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 905Chen, Xizhe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 427Chen, Y.-T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 136Chen, Yifeng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 548Chen, Yi-Sen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 882EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 149Abstract Author IndexChen, Zhi-Ying . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 9, 14Chen, Ziping C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 422, 800Chen, Zixuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 171, 806, 807Chenajovsky, Yuti . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 609Cheng, Binbin. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 700Cheng, Kun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 567Cheng, Linzhao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 220Cheng, Long . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 130Cheng, Seng H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55, 557Cheng, Woei Ping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 254Cherel, Yan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19Cherqui, Stephanie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 408Chester, John . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 606Cheung, Roland . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 367Chicoine, Louis G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149Childers, Martin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 454, 458, 467, 785Chillón, Miguel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 573Chinnasamy, Nachimuthu. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 190Chino, Takenao. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 628Chiocca, E. A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 491Chiocca, E. Antonio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 498Chiocco, Matthew J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 709Chiorini, John A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 483, 870Cho, Hanna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 560Choi, Connie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156Choi, Hye Jin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 198Choi, Hyo Sung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 877Choi, Il-Kyu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 198, 202, 242, 516Choi, Joung-Woo. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 383, 721Choi, Keum S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 722Choi, Kyoung-ju . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 188Choi, Kyung-Ju . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 202, 811Choi, Min Joung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 112Choi, Uimook. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 220, 354Choi, Vivian M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 98Choi, Yejin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 722Choi, Yeong “Christopher” . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296Chong, Heung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 606Chong, Mark . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 531Choolani, M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223, 697Choolani, Mahesh. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 531Chopra, Manisha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 328Chorny, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 323, 425Chou, Bin-Kuan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 220Chow, Laura. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33Chowdhury, Wasim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 382Chowdhury, Wasim H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 390Christensen, Camilla L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 206, 210, 511Christensen, Rikke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 91Christiansson, Lisa H.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 486, 820Christin, Anne S.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 29Christopher, Logg R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 492Chu, Baocheng. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 553Chu, Kirk . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 310Chulay, Jeffrey D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 377Chun, Kyung-Hee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 384Chung, Samuel. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 405Chuvilin, Andrey N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 735Ciccone, Carla . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 144Ciceri, Fabio. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295Ciré, Séverine. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 552Ciuffreda, C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 360Clark, K. Reed . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149, 290, 581Clarke, Christian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 642Claudio, Luigi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 847Claudio, Pier Paolo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 847Clay, Timothy C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 47Clemens, Paula R.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 455, 457, 580Clevenger, Joshua R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 96Cline, Amanda . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 521Cloer, Caryn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 890Cloutier, Denise A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 596Cmielewski, Patricia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 621Coady, Tristan H.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 626Coates, Allan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 872Cockrell, Adam S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 355, 534Cody, James J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 507C<strong>of</strong>fey, Matt. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 606Cohen, Justus B.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 640Cohen, Reuben. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 274Colella, Pasqualina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 443Coleman, John S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 668Coles, Rebecca L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 880Coley, Brian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 581Coley, Brian D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149, 290Collantes, María . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42Colle, Marie-Anne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19Collie, D. David S.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 880Collins, J. K.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 194Collins, Margaret H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 498Collins, Michael T.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 761Collins, Sara A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 705Compte-Grau, Marta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 481Condomines, Maud . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 655Congo, Richard T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893Conlon, Thomas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 290Connolly, Mary M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 727Constantinou, Varnavas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137


150<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexConti, Peter S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 200Convertine, Anthony C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 558Cooper, Laurence J. N.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479, 809Cooper, Mark J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445, 622, 776Cornetta, Kenneth G.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 353, 356, 675Coroadinha, Ana S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 278, 570Cosset, Francois-Loic . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 336Costa, Caroline . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 336Cotrim, Ana P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 74, 77, 143Cots, Dan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 573Cotugno, Gabriella . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 758Coughlan, Lynda. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 380, 723Couto, Linda . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 69Couto, Linda B.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 885Couture, Larry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279, 905Couture, Sylvana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 905Cowan, Morton J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 221Cox, Josephine. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162Cox, Julie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39Craig, Anthony T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 70Craig, Nancy L.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 15Cramer, Frederik. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 206, 210, 511Crasta, Manuela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59Creencia, Christopher. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 794Crettaz, Julien . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 810Cripe, Timothy P.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 201, 498, 830Criswell, Hugh E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 2Crombleholme, Timothy M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 830Crome, Sarah Q.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 79Cromley, Debra. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 412Cronin, Michelle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 398, 812, 848Crowley, Jim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 439Crowther, Carol. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 82Croyle, Maria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 435Cruz, Conrad R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 473Cruz, Pedro E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 579Cruz, Russell . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288Cruz, Russell C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 29Crystal, Ronald G.. . . . . . . . . . . . . . . . . . . . . . . . . . . . 19, 161, 163, 452, 752Cunningham, Sharon C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 409Cunningham, Tshaka . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 70Curiel, David T. . . . . . . . . . . . . . . . . . . . . . . . . . . 36, 379, 497, 503, 834, 842Curran, Kevin J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 815Currier, Mark A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 201, 498Curtin, James F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 181Custers, Jerome . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 723Cutler-Strom, Richelle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 558Cutrera, Jeffry J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 737Dda Silva Bizario, Joao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 464Dae, Hyun Mi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 386D’Agostino, Karla . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 50Dai, Jihong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 310Dai, Kerong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 756Dalkara, Deniz . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 442Dally, Len . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162Dam, Elisabeth. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 110Dam, Tomas N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 695Dane, Allison P.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 409Daneshmand, Manijeh. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33Dang, D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 879Danièle, Nathalie F.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 793Daniel, Skuk . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 769Danieli, P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 360Danos, Olivier. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 87, 617D’Antona, Giuseppe. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 795Dantzer, Jessica . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 353, 356Dar, Gulam H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 404Darocha, Sylvie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 48Darvish, Daniel. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 144, 772, 794Darvish, Sheila . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 794Dasgupta, Anindya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 814Dasouki, Majed . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155Dassie, Justin P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 662, 663Davaa, Enkhzaya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 896David, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223David<strong>of</strong>f, A. M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223David<strong>of</strong>f, Andrew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 668Davidson, Jesse T.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 452Davies, Kay E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 466Davies, Lee A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 123, 727, 880Davila, J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139Davis, Adam S.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 699Davis, Nancy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149Davis, Pamela B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 746Davydova, Julia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 80, 391, 497d’Azzo, Alessandra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 449D’Costa, Jenice. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 422De, Bishnu P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 161, 452, 752De Angelis, B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 178de Boer, Helen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 617de Cortie, Karin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 231De Falco, Elena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 229De Groot, Anne S.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 43De Kruijf, Marcel. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 736De Leonibus, Elvira . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 758De Palma, Michele . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 331EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 151Abstract Author IndexDe Ridder, Corrina M. A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 506de Silva, Suresh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 76De Smedt, Stefaan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 875De Smedt, Stefaan C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 742de Verneuil, Hubert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 865de Winter, Christa L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 153Dean, David A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 588, 878Deglane, Gaëlle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 883Deisseroth, Albert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 85Del Campo, Jacob. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 285del Campo, Jake. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 50Del Carro, Ubaldo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 313, 315Delépine, Pascal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 745Delai, Stefania . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 315Delalande, Anthony. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 324Delisle, Josie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 649Della Corte, Michele . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59Demaster, Amanda K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 142Demeester, Jo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 742Demeester, Joseph . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 261, 875Denapoli, Priscila M. A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 440Denaro, Maria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 228Denavand, Sarkar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 847Denby, Laura . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 723Deng, Defeng. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 731Deng, Yao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 78, 172Denisenko, Oleg . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 93Denman, Cecele J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479Denning, Gabriela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 527Denovan-Wright, Eileen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 147Dent, Paul . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 503DeRavin, Suk See. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 354, 860Derecho, Ivy. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279Derendorf, Hartmut. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 256DeRossi, Charles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 348, 650Desai, Maya K.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 18Desmedt, Stefaan S. D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 261Dev, Jyoti . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 208Devine, Sean P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 509, 771DeWeese, Theodore L.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 251Dhar, Debanjan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 489, 837Di, Wei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 158Di Domenico, Carmela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 315Di Girolamo, Ilaria. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 316, 413Di Natale, Paola . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 315Di Paolo, Nelson C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 541Di Pasquale, Giovanni . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 483Di Rocco, Giuliana. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 898Di Scala, Marianna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 485Di Serio, Clelia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 345, 346Di Stasi, Antonio. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288Di Stefano, Bruno . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 632Di Vicino, Umberto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59, 443, 624Diaconu, Iulia. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 193, 204, 538, 549Diago, Oscar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 492Diallo, Jean-Simon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39Dias, João D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204Diaz, Deanna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 477Diaz, Rosa. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 175, 481, 606, 607, 610Dibb, Nick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 349Dibra, Denada . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 737Dieci, Marco. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276Diepolder, Helmut M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 289Dietz, Allan B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 488Dighe, N.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223Dighe, Niraja . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 531Dinauer, Mary . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 356Dinauer, Mary C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 353, 354Dinculescu, Astra. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 61Dindot, Scott . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 75Dines, Karla A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445, 622Dingli, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 208, 684Diouf, Oumar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288DiPasquale, Betsy A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 498Discher, Dennis E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 357Dixon, Tracy. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 157, 514, 515Dmitrieva, Nina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 199Doering, Christopher B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 230, 527Doglioni, Claudio. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297Doherty, Joseph E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 15Dohmann, Hans F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 440Dolphin, Colin T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 716Dolter, Karen E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162, 740Doms, Robert W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 801Donate, Amy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 107Donate, Amy L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 174Dondero, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 218Dong, Biao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 310Dong, John Y.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 504, 643Dong, Xiaoyan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 376Donnelly, Eleanor M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 780Donovan, Kathleen A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 218Donsante, Anthony . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 24Dorer, Dominik E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 387, 641Doria, Monica. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59, 64Dorko, K.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139Doronin, Konstantin. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 496Doroudchi, Mehdi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 21Dorvault, Christine. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 834Dotti, G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30, 178


152<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexDotti, Gianpietro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 28, 288, 293, 472, 672Douglas, Joanne. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 834Douglas, Joanne T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 507Dowdy, Sean C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 488Down, Julian D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 228Downey, Harre . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 546Downing, Brandon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 353Doyon, Yannick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 98Dragneva, Galina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159Drescher, Charles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 265Drescher, Max . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 843, 849Dropulic, Boro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 527Du, Shi-Suo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 211Duan, Cathleen. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 872Duan, Dongsheng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 150, 466,. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 587, 601, 707, 876Duan, Jiachuan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 433Duan, Xunbao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 310Dubrot, Juan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42Duckworth, Katherine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 779Dudley, M. E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31Dudley, Mark E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 25Duffy, Angela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 424Duffy, Aoife . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 427Dugin, Daniel P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162Dumont, Nicolas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 769Dunbar, Cynthia E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 96Dunfee, Ashley E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 96Dunn, Diane M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 152Dunn, Robert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 181Dunphy, Nina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279Durett, April . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288Duskey, Jason . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 594Dwyer, Markryan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 645Dylewski, Dawna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 487EEagle, Michelle. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314, 411Eaton, Laura . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 910Ebtekar, Massoumeh. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 871Edelman, Gerald . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 292Edmonson, Shyrie. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 548Edmonson, Shyrie E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 308Edwards, Paul C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 104Eerola, Kim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 145Eghtesad, Saman . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 580Egorova, Anna A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 750Ehrhardt, Anja . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 92, 141, 385, 636, 712Eiden, Maribeth V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 696Eiermann, Jessica . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314, 411Einfeld, David A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 504El Andaloussi, Samir . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 556Elgstrand Wettergren, Erika . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 239Elhalel, Michal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616Eliopoulos, Aristides G.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 486Ellis, James . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 300, 691El-Sherbini, Yasser M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 732Ely, Abdullah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 82Emborg, Marina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 779Eming, Sabine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 5Emmanouil, Kalyopy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 405Enbersen, Johan F. J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 126Encinas, Tony . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 274Endo, Masayuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 320, 344, 676Engelhardt, John F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 4, 620, 698Engler, Tatjana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 637Ennis, Matthew K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 684Enoto, Kaoru . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 591Enríquez de Salamanca,. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Rafael42Enright, Annette . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 270Epperly, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 157, 514, 515Epperly, Michael W.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 580Erbacher, Patrick. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 883Erbs, Philippe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204Erez, Ayelet . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 677Erger, Kirsten . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 371Erger, Kirsten E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 378Eriksen, Jens . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 101Eriksson, El<strong>of</strong> . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 237Eriksson, Minna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204Erker, Laura . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 910Ertl, Hildegund C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 389Escutenaire, Sophie. . . . . . . . . . . . . . . . . . 177, 197, 204, 291, 538, 549, 658Esfandiarifard, Saghi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 772, 794Essand, Magnus . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 501, 828Esser, Knud . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312Evans, Claire F.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162, 740Everhart, Drew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17Ewert, Kai K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 393FFabris, Marina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258, 666Fadel, Hind . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 685Fagone, Paolo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 861Fakhfakh, Raouia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 784Falgowski, Kerry A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 897EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 153Abstract Author IndexFalk, Darin J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 596Falls, Theresa. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39Falzarano, S<strong>of</strong>ia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258, 666Fan, Bai . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 510Fan, Zheng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 328Farag, Jacqueline . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156, 454Fargnoli, Anthony . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 600Farini, Andrea. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 460, 464, 795Farley, Raymond. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 873Fassas, Athanasios . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137Fast, Patricia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162Favaro, Patricia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 614Federici, Thais . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 699, 778, 782Feder<strong>of</strong>f, Howard J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 23Federspiel, Mark J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 490, 499Feduska, Joseph M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 629Feijen, Jan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 126Feldman, Steven A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 25, 190Fellenberg, Kurt . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 641Fender, Pascal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 843Fenelon, Gilles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777Feng, Jiazuo H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 744Ferguson, Sherise D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 902Ferlini, Alessandra. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258, 666Fernandes, Julio C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 756Fernandez, Christian A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 117, 594Ferrand, Maxime . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 48Ferrari, Giuliana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 319, 523Ferro, Leda . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 911Ferry, Nicolas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 138Fewell, Jason G.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893Fidler, Jonathan A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 557Filipczyk, Adam . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284Filippova, Elena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 444Finegold, Milton . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 910Finn, Eric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 461, 786Finn, Jonathan D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 43, 551, 614, 615Finney, Helene . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 845Fire, Andrew Z.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 14Firozi, Pervez. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 574Fischbeck, Kenneth H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 235Fischer, Alain . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 229Fishbein, Ariel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 425Fishbein, Ilia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 323, 425Fisher, John T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 620Fisher, Paul B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 503, 847Fisher-Perkins, Jeanne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 411Fisk, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 697Fisk, N.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223Flaat, Mette. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314, 411Flageul, Maude . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 138Flake, Alan W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 320, 344, 676Flanagan, Mike . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 105Flanigan, Kevin M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 152, 155, 789Flannery, John G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 442Fleischhauer, Katharina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295Fleischmann, Bernd. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 693Fletcher, Anita M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 776Florence, Julaine M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 152Flotte, Terence R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 51, 53Flotte, Terrence. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 444Flotte, Terry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58Foley, Jason. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 659Follenzi, Antonia. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 134Foloppe, Johann. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204Fong, Mei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 674Fons, Michael. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 289Fontanellas, Antonio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42Forde, Patrick F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 736Forman, Stephen J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 555Fortes, Puri . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 249, 889Foster, Aaron E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 469, 816Foster, Edmund . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 451Foulad, David. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 181, 475Fowler, Daniel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 341Fowler, Daniel H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 333, 509, 659Fracchia, Sergio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276Fraix, Aurore . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 131Franicola, Darcy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 514Frank, Michael M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 638Frankel, Timothy L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 190Fransson, Moa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 176, 453Frattini, Mark. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 32Frecha, Cecilia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 336Frelin, Lars. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 111, 289Frenkel, Niza . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 719Frey, Brigitte M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 855Frey, Felix J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 855Friedman, Ge<strong>of</strong>frey . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11Froehner, Stanely C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 584Froelich, Steven . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 689Fu, Yang-Xin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 517Fujita, Yuriko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288Fujiwara, Hiroshi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 652Fukudome, Takayasu. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 585Fukunaga, Yoshitaka. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 680Furuya, Aki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 824Fusaki, Noemi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 303, 634Futaki, Shiroh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 246, 253, 401


154<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexGGabant, Philippe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 740Gabriel, I. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777Gabriel, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 286, 347Gahan, Cormac G. M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 398Gahl, William A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 144Gaj, Thom . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 94Galanis, Evanthia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 170, 490, 499Galivo, Feorillo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 175, 481, 607, 610Galla, Melanie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284, 302, 304, 619Galletti, Margherita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 523Galloway, Gloria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 290, 581Galton, Bruce . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 218Galun, Eithan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16, 616Galvan, Daniel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 12, 813Galy, Anne. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 48, 225, 229, 533, 552Gamble, Lena J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 842Ganea, Doina. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 859Gangadharan, Bagirath . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 230, 527Gangeswaran, Rathi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 605Gangopadhyay, Anu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 659Ganguli, Munia. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 127, 132Ganly, Sandra. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 439Gao, Dongling . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 605Gao, Guangping . . . . . . . . . . . . . . . . . . . . . . . . . 58, 362, 433, 450, 671, 759Gao, Ran. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 242Gao, Yongxin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 219, 518Garabedian, Elizabeth . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296Garcia, Louis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 460Garcia, Luis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 154, 464, 795Garcia-Aragoncillo, Eva. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 494Gardiner, David F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162Gardmark, Truls . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 176Gardner, Matthew. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 608Gardner, Rebecca A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 618Garg, Harsha K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 677Garg, Nupur . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 796Gargiulo, Annagiusi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 702Garibov, Mikhail . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 90, 529Gatti, Alessandra. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276Gautney, Richard. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 527Gavrikova, Tatyana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 497Gay-Antaki, Carlos. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 317Gaymalov, Zagit . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 817Ged, Cécile . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 865Gedeon, Christopher R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445, 622Gee, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30Gee, Adrian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288Gee, Adrian P.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 293Gee, Samantha W. Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 700Geguchadze, Ramaz . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 371, 378Gehl, Julie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 101, 106, 109Geisert, Eldon E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 441Genovese, Pietro. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10, 88, 286, 632, 654Gentile, Antonietta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 898Gentner, Bernhard. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 313, 331, 337Gerdemann, Ulrike . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 29, 473, 799Gerhardt, Greg A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 709Germain, Sean A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 596Gerritsen, Winald R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 40, 502Gersbach, Charles A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 94Geurts, Jeroen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 559, 627Ghaffari, Seyed Hamidolah. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 871Ghavamzadeh, Ardeshir . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 871Ghazi, Alexia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 470, 471Ghio, Domenico . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295Giacca, Mauro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 311, 423, 431Giachelli, Cecilia M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 730Giamarchi, Philippe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 745Giangrande, Paloma H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 662, 663Giannakopoulos, Aristeidis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 729Gianni-Barrera, Roberto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 430, 597Giannoni, Francesca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 522Gibbons, Hillary N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 809Gibly, Romie F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 764Gibson, K. M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139Gibson, Soojung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 417Giddings, Angela. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 881Giedlin, Marty . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 783, 798Giffear, Mary . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 477Giladi, Hilla . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16Gilbert, Rénald . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 463Gilbert, Richard A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 116Gill, Deborah R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 123, 727, 739, 880Gill, Steven . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 394Gillet, Danielle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120Gillet-Legrand, Beatrix . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 228Gilmour, Jill . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162Gimble, Jeffrey. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314Gimble, Jeffry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 105Ginns, Edward I . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 418Giorgio, Todd D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 592Giovannoni, Roberto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59Giralt, Sergio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 809Giridhara, Jayandharan R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 68Gissel, Hanne. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 106Giunti, Massimo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59Giustacchini, Alice . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 337EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 155Abstract Author IndexGjetting, Torben . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 206, 511Gkirtzimanaki, Katerina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 486Glazer, Peter M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 50, 285Gleich, Bernhard. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 693Glimm, Hanno . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 347, 612Glorioso, Joseph C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 640, 781Glowacki, Julie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 157Gnatenko, Dmitri . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 372Gnecchi, M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 360Godbehere, Sarah S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 638, 651Goepfert, Christine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 855Goerlich, Corbin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 243Goetzmann, Jason . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58G<strong>of</strong>f, Julie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 514Goins, William F.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 640, 781Gokhale, Abhay . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 157Goldschlager, Gregg . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 38Goltzmann, Haim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616Gomero, Elida . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 449Gomez Almarza, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 5González-Aseguinolaza, Gloria . . . . . . . . . . . . . . . . . . . . . . . . . . 485, 708, 810Gonzalez-Aparicio, Manuela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 810Gonzalez-Rojas, Jovanna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 889Good, Alec S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 125Goodheart, Michael J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 620Gopal, Kapil . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156, 454Gopal, Vijaya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 404Gorbatyuk, Marina S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 446Gorbatyuk, Oleg . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 378Gordley, Russell M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 94Gordon, Cath M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 880Goss, James R.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 781Gothelf, Anita. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 101Gotoh, Akinobu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 216Gotoh, Mitsukazu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 419Gotschalk, Stephen M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 816Gottfried, Lynn F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 878Gottschalk, Stephen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 179, 293, 471, 656Gottschalk, Stephen M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288Gottschalk, Steven . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 470Goudenege, Sebastien. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 769Gould, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 609Gräslund, Torbjörn. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 239Gröhn, Olli . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159Gracey, Lia E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 14Graham, John G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 770Grande, Alexis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 523Grandi, Paola . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 640Grant, Rebecca L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 45Grant, Steven. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 503Grant, Trobridge D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 864Grassman, Elke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 526Gray, John . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 222, 449, 733Gray, John T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 354, 668, 860, 861Gray, Steven . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 581, 782Gray, Steven J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 2Greco, Adelaide . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 847Green, Matthew D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 122, 125Greenberg, Philip D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 88, 654Greenberg, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 646Greenberger, Benjamin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 515Greenberger, Joel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 157, 514, 515Greene, Michael R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 861Gregory, Philip . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 798Gregory, Philip D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10, 11, 88, 98,. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 286, 415, 618, 632, 650, 654, 801Greiser, Udo. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 439Grez, Manuel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 869Grieger, Joshua C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 160Griener, Dale . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 285Griesenbach, Uta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 873Griffith, Graziella. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 154Griffiths, Duncan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 577Grilley, Bambi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288Grimm, Brad . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 571Grimm, Dirk . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 660, 886Grinstaff, Mark W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 392, 744Gritti, Angela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 316, 413, 632Grompe, Markus. . . . . . . . . . . . . . . . . . . . . . . . . 99, 306, 681, 682, 760, 910Gross, David-Alexandre . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 48Grossardt, Christian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 833Grossman, Robert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 471Grove, N.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 879Grove, Nathan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 75, 388Grove, Nathan C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 639Grover, Rajat . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 63Gruber, Harry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 492, 831Gruenert, D. C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 913Gu, Shuo. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 54Gu, Yachuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 566Guégan, Philippe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 395Guérin, Claude . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 463Gualandi, Francesca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258, 666Guan, Jie. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 78, 114Gubinelli, Francesco . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 644Guedan Carrio, Sonia. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 496Guerin, Karen. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 442Gui, Tong. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 534


156<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexGunset, Gertrude . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 655Gunther, William R.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 595Guo, Jianfeng. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 254Guo, Siqi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 107Gupta, Sanjeev. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 134Gürke, Lorenz. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 430, 597Gurram, Sweta. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 221Gurruchaga, Jean-Marc . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777Guse, Kilian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204Gussing, Fredrik . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 239, 350Guttridge, Denis C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 635Guyonnet-Duperat, Véronique . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 865HHa, Yoon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 260Haas, Andrew. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33Haavisto, Elina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 177, 193, 197, 538, 658Habib, Bianca. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 603Hacein-Bey Abina, Salima . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 229Hacein-Bey-Abina, Salima. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 13Hacker, Ulrich. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 664, 887Hackett, Neil R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19, 452Hackett, Perry B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 325, 728, 734Hackl, Florian. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 237Hadac, Elizabeth. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 604Haddock, Courtney . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 213, 564, 578Haga, Kazunori. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 334Haghighatgoo, Arman . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 794Hahn, Woong. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818Hai, Li. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 484Hai-Ying, Zeng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 73Hajjar, Roger J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 601Hakkarainen, Tanja . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204Halbert, Christine L.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 623Hall, Ge<strong>of</strong>f. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 606Halle, Stephan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284Hallek, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 5, 312Hamada, Hiromi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 554Hamada, Katsuyuki. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 216, 493, 825Hamakawa, Hiroyuki. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 493Hamilton, Shari R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 308Han, Sang W.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 440Han, Seung Ryul. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 560Han, Sung Yeol . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 112Han, Tae Hee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 85Han, Yu. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 902Hannah, William. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 534Hannaman, Drew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162, 740Hans, Skovgaard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 206Hansel, M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139Hansford, Loen. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 498Hans-Peter, Kiem . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 864Hantraye, Philippe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777Harada, Yui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 824Haralambieva, Iana. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 170Harashima, Hideyoshi . . . . . . . . . 97, 240, 246, 253, 401, 591, 743, 748, 894Harding, Cary. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 681Hargrove, Phillip W.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 338, 339, 525Harms, Ashley S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 773Harper, John . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 158Harrington, Kevin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 606Harris, Drea . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 477Harris, Raymond D.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36Harrison, Frank. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 408Harrison, Patrick T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 705Harrow, Faith. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 521Hart, Brit J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 800Hart, Stephen L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 394, 747Hartman, Zachary C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 638Harty, C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 194Harvey, Brandon K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 709Hasegawa, Mamoru . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 303, 634, 824, 873Haseley, Amy. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 836Hashimoto, Takuya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 891Haskins, Mark . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 758Haskins, Mark E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 598, 759Hatakeyama, Hiroto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 246, 253, 894Hatano, Norikazu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678Hatefi, Arash . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 329Hattori, Kayoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 862Hauer, Julia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 617Haugwitz, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 674Haurigot, Virginia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 546, 885Hausl, Martin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 636Hausl, Martin A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 385, 712Hauswirth, Wiliam W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 417Hauswirth, William W.. . . . . . . . . . . . . . . . . . . . . . . . . . . . 17, 21, 61, 71, 446Hawkins, Troy B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 353, 356Hawley, Robert G.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 342, 690Hawley, Teresa S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 342, 690Haworth, Kevin G.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11Hay, John G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 713Hayashi, Hiroki. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 774Hayashi, Yasuhiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 743Hayes, John. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155He, Bo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 683He, Cheng-Yi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 9He, Ran. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58, 362, 450, 671EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 157Abstract Author IndexHe, Shuhui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 130He, Xianghui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 186, 566Hearing, Patrick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 372, 573Hebel, Henry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 576, 739Heberer, Michael. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 430, 597Heckl, Dirk. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 318Heemskerk, Hans A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 153Hegadorn, Carol . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 767Hegge, Julia. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 160Heinonen, Suvi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159Heinz, Niels . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 302, 619Heldermon, Coy D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 768Helfer, Brooke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 271Heller, Loree . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 116, 823Heller, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 107, 174, 823Helminen, Andreas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 197Hemmi, Silvio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 193Hemminki, Akseli . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 177, 193, 197,. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204, 291, 538, 549, 657, 658, 843Hemminki, Otto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 193Hensel, Jonathan A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 63Heo, Kyun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 386Hermonat, Paul L.. . . . . . . . . . . . . . . . . . . . . . . . . . . 429, 436, 437, 711, 819Hernandez-Alcoceba,. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .Ruben 494, 810Hershfield, Michael S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296, 678Hervás-Stubbs, Sandra. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42Hervas-Stubbs, Sandra. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 810Herzog, Erik. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 768Herzog, Roland W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 309, 363, 539, 540Heslop, Helen E.. . . . . . . . . . . 28, 29, 30, 178, 288, 293, 470, 471, 473, 799Hewitt, Stephen M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 659Hiatt, P.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 879Hibbitt, Olivia C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 602Hicks, Martin J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 452, 752Hicks, Martin L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 161Hickstein, Dennis D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 234, 524, 858Higashimoto, Tomoyasu. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 526Higgins, Luke J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 214Higgs, Jerome T.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 63High, Katherine A.. . . . . . . . . . . 8, 43, 69, 308, 546, 548, 614, 615, 630, 673Hikichi, Mina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 34, 840Hinderer, Christian. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 412Hintze, Thomas H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 423Hinzman, Jason . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 709Hiraoka, Kei. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 334, 492, 831Hiroshima, Kenzo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 851Hirsch, Matthew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 2Hiscott, John . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39Ho, David D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162Ho, Seong-Hyun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818Hodge, Gregory . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 168Hodgson, Clague P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 166, 572, 575, 738Hoel, Brian D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 504Hoesel, Marianna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312H<strong>of</strong>fer, Barry J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 709H<strong>of</strong>fman, Brad E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 539H<strong>of</strong>fman, Eric. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 56H<strong>of</strong>fmann, Ellen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 886H<strong>of</strong>fmann, Victoria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 135H<strong>of</strong>mann, Lawrence V.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 214Hoheisel, Jörg . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 641Hojman, Pernille. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 101, 106Hole, Patrick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 577Hollis, Roger . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 522Holmes, Michael. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 798Holmes, Michael C.. . . . . . 10, 11, 88, 98, 286, 415, 618, 632, 650, 654, 801Holt, Nathalia G.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11Holtrup, Frank . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 641Holtz, R. B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 213Hondmann, Dirk H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 332Hong, Seung Hwa. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 112Hong, Youngtae . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818Hoogstraten-Miller, Shelley. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 144Horowitz, Eric D.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 403Horsager, Alan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 21Horton, Terzah M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 473Horvath, Staci . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 196Hosoyama, Sachiko O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 151Hoti, Naser Uddin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 382Hotta, Akitsu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 691Hou, Linan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 186Hough, Christine. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 767Hovhannisyan, Ruben . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 625Howard, Candace . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 847Howard, Douglas B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 709Howard, Linda . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 270Howard, Jr, James . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 328Hricak, Hedvig . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 185Hsieh, Chia-Ling . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 844Hsieh, Matthew M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 352Hsieh, Ming Kun. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 173Hsu, David. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279, 905Hu, Biliang. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 686Hu, Chuhong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 44, 224, 373Hu, Chunling . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 685Hu, Chunyan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 433Hu, Huimin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 449


158<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexHu, Jim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 872Hu, Yu-Chen. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 583Hua, Yimin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55Huang, Chienjin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 173Huang, Jing-Hua . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 202, 811Huang, Yaoxing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162Huard, Johnny . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 456, 629, 635Hughes, Jeffrey A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 256, 775Hui, Daniel J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 43, 546Huizing, Marjan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 144Huls, Mary H.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479Humala, Nelson . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 38Humblet-Baron, Stephanie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 343, 529Humeau, Laurent . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 41Humeau, Laurent M. . . . . . . . . . . . . . . . . . . . . . 165, 422, 797, 800, 802, 803Humphries, Margaret. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 444Humphries, R. Keith . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 631Hung, <strong>Gene</strong> . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55, 557Hung, Mien-Chie. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 844Hunter, Michael J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 524Hurley, Arlene. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162Hurton, Lenka . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479Huston, Marshall W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 530Hutson, Thomas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 451Huye, Leslie E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 472Huyn, Steve. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 714Hwang, Esther . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 796Hwang, Gloria L.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 214Hwang, Mark E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 119, 595Hwang, T. C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 876Hwang, Taeho . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33Hyatt, Susannah L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445, 622Hyde, Stephen C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 123, 727, 739, 880Hyland, Kendra A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 868Hynes, Sean O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 439Hyvärinen, Anna. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 212, 512IIacobelli, Nicholas. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 615Iankov, Ianko D.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 170, 490Ichikawa, Tomohiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 824Iida, Akihiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 303Ikeda, Alan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296Ikeda, Hiroaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 652Ikeda, Yasuhiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 599, 633, 688Ikoma, Akihiko. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 862Ilett, Elizabeth . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 481Ilies, Marc A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 757Inagaki, Akihito . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 334, 492, 831Ino, Yasushi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 832, 839Inoue, Hiroyuki. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 192, 263Inoue, Makoto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 303, 634, 824, 873Inoue, Tomoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263, 899Iodice, Carolina. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 443Ironside, Christina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 864Irrazábal, Thergiory. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 766Isaksson, Christina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 350Ise, Kazuya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 419Ishida, Eri . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 513Ishihara, Kazuhiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 743Ishii, Takehiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 118Ishii, Yuko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 102Ishiwata, Akira . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 421, 544Ismail Nounou, Mohamed M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 405Itaka, Keiji. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 118, 891Ito, Erika . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 253Ito, Mikako . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 585Ito, Takayuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 428Ito, Tomoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 749, 825Ivanovska, Irena. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 357Ivics, Zoltan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 302Iwamoto, Mari . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 350Iwata-Kajihara, Tomoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 480Izmiryan, Araksya. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 87Izsvak, Zsuzsanna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 302JJaffrès, Paul-Alain . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 131, 745Jagadeesh, Jayashree . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296James, Scott E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 558Jan, Caroline . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777Janda, Kim D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 161, 452, 752Janicki, Hanna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312Janik, J. E.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31Janjic, Jelena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 271Jarraya, Bechir . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777Jasti, Aparna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 353, 675Jay, Chris. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 144Jay, Chris M. . . . . . . . . . . . . . . . . . . 148, 245, 247, 252, 532, 564, 578, 586Jayandharan, Giridhara R. . . . . . . . . . . . . . . . . . 363, 369, 370, 700, 704, 706Jegga, Anil G.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 830Jenkins, Kristy M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 79Jensen, Bryan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 910Jensen, Michael C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 618Jensen, Thomas G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 787Jeong, Hwa Yeon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 128, 406, 754Jeong, Jae-Gyun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818Jeong, Jin-Sook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 560EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 159Abstract Author IndexJeong, Myung Ho . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 399, 751, 900, 904Jian, Zhou . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 73Jiang, Guozhong. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 605Jiang, Haixiang. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 638Jiang, Jiangjang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 420Jiang, Shuguang. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 798Jiang, Xuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 124, 321Jiang, Ziyue . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 850Jibly, Tamar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616Jikimoto, Takumi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 167Jimenez, Veronica. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 762Jin, Jiankang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 243Jin, Xiangyang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 414Jno-Charles, Odella . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 625Joensuu, Tiimo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 538Joensuu, Timo . . . . . . . . . . . . . . . . . . . . . . . . . 177, 193, 197, 291, 549, 658Joergensen, Mette . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 109Joh, Ju . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 163Johana, N.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 697John von Freyend, Miriam . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312Johnson, Adam J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 330Johnson, R. Paul. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 645Johnson, Shane . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 576Johnston, Jennifer M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 230Johnston, Julie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 625Jollet, Arnaud. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 87, 617Jolly, Douglas. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 492, 831Jones, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 416Jonker, Derek. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33Jonlin, Erica . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137Joung, J. Keith . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 95Judge, Luke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 466Judge, Luke M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 459Juliano, Rudy L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 62June, Carl H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 341, 798, 801, 809Jung, Jae Wook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 877Jung, Kyeong Cheon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 406Jung, Maria C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 289Junghans, R. P.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 827Junghans, Richard P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 27, 182Jungmann, Andreas. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 241KKabanov, Alexander V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 817Kabashima, Kenji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 862Kabir, Farruk M. L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 379Kacherovsky, Nataly A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 730Kaeppel, Christine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 138, 305Kafri, Tal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 355, 534Kaikkonen, Minna U.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 209Kaja, Aparna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 12Kakeda, Minoru . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 236Kakimoto, Shinji. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 397Kaler, Stephen G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 24Kalsi, Jasmine K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 700Kaluza, Karen. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 175Kamau, Erick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 645Kamen, Amine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 361Kamijima, Shuichi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 334Kamimura, Kenya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 322Kaminsky, Paul M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 698Kaminsky, Stephen M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 161, 452, 752Kamiya, Hiroyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 97, 240Kanai, Ryuichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 608, 829Kanda, Genki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 240Kaneda, Yasufumi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 187, 628, 774Kanerva, Anna . . . . . . . . . . . . . . . . . . . . . . . . . 193, 204, 291, 538, 549, 658Kang, Bum-Yong. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 437Kang, Chang-Yuil . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818Kang, Elizabeth M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 354Kangasniemi, Lotta . . . . . . . . . . . . . . . . . . . . . . . . . . 177, 291, 538, 549, 658Kano, Mitsunobu R.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 891Kao, Chinghai. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 334Kaplan, David. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 498Kapoor, Neena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296Kapp, Kerstin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 189, 822Kappel, C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223Karandish, Sara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 342Karioja-Kallio, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 538Karlen, Andrea . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 221Karlen, Andrea D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 232Karli, Eerika . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 658Karlsson, Hannah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 83Karpati, George . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 463Karponi, Garyfalia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137Kasahara, Nori . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 200Kasahara, Noriyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 334, 492, 500, 831Kasahara, Yuko N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 151, 582Kaskenpää, Nina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159Kassim, Sadik H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 1, 412Kasuda, Shogo. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 903Kataoka, Kazunori . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 118, 891Katayama, Kazufumi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 537Katayama, Takane . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 167Kato, Junko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 903Katoono, Ryo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 401Katovich, Michael J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 518Katus, Hugo A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 241, 432


160<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexKatz, Michael G.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 600Katzenberg, Regina H.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 214Kaul, Sunil C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 242, 841Kaur, Balveen. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 199, 836Kaushal, Shalesh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 444Kauss, Ariel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 3Kaverina, Natalya V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 835Kawabata, Kenji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 537Kawabata, Masato . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 167Kawakami, Yutaka . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 480Kawamura, Kiyoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 851Kawamura, Nobuaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678Kawanishi, Yu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 513Kawano, Hirotaka. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263, 899Kawasaki, Yoshiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 500Kay, Jennifer . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 794Kay, Mark A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 9, 14, 54, 306, 682, 760Kaynor, G. Campbell . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 517Kaza, Niroop . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 63Kazuki, Yasuhiro. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 236Keating, M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30Kehry, Marilyn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 550Keiser, Nicholas W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 4, 698Kemmerrer, Stephen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 103Kemmerrer, Steve. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 327Kennedy-Nasser, Alana. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288Kerkard, Sid P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 478Kerns, Hannah M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 343Kerr, Douglas A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 908Keshavjee, Shaf . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 872Keswani, Rahul K.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 119Kettlun, Claudia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 12Kew, Yvonne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 471Khan, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 648Khan, Amir. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 103, 115, 477Khan, Junaid A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 429, 436, 437Khanduri, Richa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 132Khargharia, Sanjib . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 117, 594Khazi, Fayaz . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 310Khim, Jit . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 529Khokher, Zeshan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 772, 794Kholová, Ivana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159Khoury, Maroun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 531Kichler, Antoine. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 67, 400, 791Kida, Miyuki. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678Kidokoro, Minoru . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 34, 840Kiem, Hans-Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 294, 631, 909Kim, Dong-Eun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 895Kim, Dongho . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 257Kim, Dong-Sik . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818Kim, Eun Ok . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 386Kim, Hong Sung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 406, 754Kim, Hyun-Ki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 896Kim, In-Hoo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 384, 386Kim, Inwook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 242Kim, Ji-seong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 188Kim, Jong-Mook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818Kim, Joo-Hang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 202, 257Kim, Jung Seok . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 128, 406, 754Kim, Kenneth . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11Kim, Kenneth H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36, 503Kim, Keun Sik . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 406, 754Kim, KeungNyun. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 260Kim, Keun-Sik . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 128Kim, Kwang-il. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 805Kim, Kyungbo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 895Kim, Min jung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 560Kim, Nahyun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 407, 877Kim, Nam Hee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 722Kim, Pyung-Hwan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 86Kim, S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 913Kim, So Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 257Kim, Sue Kyung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 386Kim, Sujeong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818Kim, Sung Wan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 86, 126Kim, Sunyoung. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818Kim, Tae-il . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 86Kim, Yong Oock . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 516Kim, Yong Sook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 399, 751, 900, 904Kim, Yoon-Sang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 338, 339, 861Kim, Yun-Hee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 386Kimball, Kristopher J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 834Kimmelman, Jonathan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 779Kimura, En. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 468, 788Kimura, Takahiro. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 334Kinder, Clifford . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 646King, Gwendalyn D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26King, Wendy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155Kingsman, Susan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777Kinoh, Hiroaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 824Kinoh, Hiromi H.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 151, 582Kinoshita, Shohiro. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 167Kirn, David H.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33, 39Kiselev, Anton V.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 750Kisling, Lisa A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 759Klanke, Chuck . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 830Klein, Christoph . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612Kleinschmidt, Jürgen A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 241, 432EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 161Abstract Author IndexKleuss, Christiane . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 189Kliem, Michele A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 778Klimczak, Ryan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 299Klimczak, Ryan R.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 442Klump, Hannes. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284Kmiec, Eric B.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 569, 897Knoblauch, Christine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279Knolle, Percy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312Knop, David R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 377, 571Kobasa, Darwyn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 169Kobayashi, Hiroshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 851Kobayashi, Ryouji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678Kobinger, G.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 648Kobinger, Gary . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 64Kobinger, Gary P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 169, 724Koch, Walter J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 600Kochanek, Stefan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 637Kochenderfer, J. N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31Kocis, Keith . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 328Koeberl, Dwight D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 47, 136, 142Koefoed, Mette . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 787Koerber, James T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 8K<strong>of</strong>od-Olsen, Emil . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 91Kohn, Donald B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296, 414, 522Koibuchi, Nobutaka. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 774Koide, tatsuya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 468Koike, Kazuo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 890Kok, Cindy Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 409Kokkinou, Dimitra. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 729Koldej, Rachel M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 867Kole, Ryszard. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 56Kolstad, Kathleen D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 442Komanduri, Paavani . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 515Komatsu, Norio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263Kondratenko, Irina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612Kong, Seogkyoung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 182Kong, Weiming. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 859Koniar, Brenda L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 728Konieczny, Patryk . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 786Konno, Tomohiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 743Konstantinova, Pavlina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 249, 255Kontermann, Roland . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 387Koorneef, Annemart . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 255Koornneef, Annemart . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 249Kopp, Lisa M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479Kopyov, Oleg . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 912Kornegay, Joe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 454, 467, 785Kornegay, Joe N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 790Kornegay, Joseph . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 458Korokhov, Nikolay. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 165, 422, 797, 800Korpisalo, Petra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159Koski, Anniina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 177Kota, Janaiah. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155Kotchey, Nicole M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 7, 703Kothary, Nishita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 214Kottke, Timothy . . . . . . . . . . . . . . . . 175, 481, 496, 606, 607, 610, 611, 653Kouyama, Emi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 537Kowalczyk, Tomasz H.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445, 622, 776Koyama, Hiroyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 891Koyama, Taro. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 237Koyama, Yoshiyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 749, 825Kozink, Daniel M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 142Kozyak, Benjamin. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156, 454Kraaij, Robert. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 506Krahn, M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 460Krainer, Adrian R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55Krance, Robert A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288Krasnykh, Victor . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 725Krause, Anja. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 163Krause, Johanna. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 318Krejci, Eric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 585Kremer, Eric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 570Kremer, Eric J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 573Kreppel, Florian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 637Krichevsky, Anna M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 665Kroeger, Kurt . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 181Kroeger, Kurt M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26, 475Kron, Matthias W.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 637Ksienzyk, Jan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 432Kuang, Chenzhong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 332Kuball, Jurgen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 88, 654Kubo, Shuji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 500Kudrolli, Tarana A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 390Kudva, Yogish . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 633Kuehle, Johannes . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284Kühlke, Klaus . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612Kuhn, Joseph . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 275, 292, 335, 532Kulkeaw, Kasem. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263Kumagai, Izumi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 640Kumar, Padmasini. . . . . . . . . . . . . . . . . . . 245, 275, 292, 335, 487, 532, 564Kumar, Priti . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 285, 407, 877Kumar, Sanjay . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 906Kumar, Sanjeev . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250Kumar-Singh, Rajendra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 447, 448Kume, Akihiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 207, 421, 428, 544, 554Kupiec, Martin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16Kurachi, Shinnosuke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 537Kurita, Ryo. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263, 899


162<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexKurre, Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 692Kurtova, Antonina V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 469Kusano, Eiji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 428Kushmeric, Martin. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 553Kustikova, Olga S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 619Kuwahara, Hiroya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 259Kwon, Jin Sook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 399, 751, 900, 904Kwon, Oh Seok . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 112Kyrtatos, Panos . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 394LLab, McIvor . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 221Labelle, Andrea. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 767Labruna, Giuseppe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 435Lachmann, Nico . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 528Laflamme, Karina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 521Lagadinou, Eleni . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 340Lago, Natalia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 451Lagresle-Peyrou, Chantal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 617Laguzzi Larcher, Fernando . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 5Lai, Frances . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39Lai, Yi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 150, 466, 587Laidinen, Svetlana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159Lainé, Céline . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120Laing, Jennifer M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 273, 717Laitinen, Anna E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 669Laitinen, Lauri M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 669Lakshmipathy, Uma . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 268Lalanne, Magalie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 865LaManna, Caroline M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 744Lamarre, Yann . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 769Lamm, Don . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 35Lamrissi-Garcia, Isabelle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 865Landry, Breanne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 129, 755Langan, Russell C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 25Langel, Ülo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 556Lapteva, Natalia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 474Larcher, Fernando . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 158, 351Larochelle, Andre . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 96Larochelle, Nancy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 463Lattanzi, Annalisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 413Lau, Ashley . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 306Laure, Aurelian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 273Laurema, Anniina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 765Lavasani, Mitra. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 629, 635Lavasanifar, Afsaneh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 568Lavie, Arnon. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 333Lavilla-Alonso, Sergio. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 193Lavitrano, Marialuisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59Lawler, Sean . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 498Lawson, Alastair . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 845Lawton, Anna E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 727Lay, Fides D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 44, 224, 373Layman, John M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 125Lazaris, Vassileios . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 729Le, Yun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 417Le Boeuf, Fabrice . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 37, 39Le Gall, Tony . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120, 131, 395, 745Le Guiner, Caroline . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 305, 365Le Hir, Maëva. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 154Lebel-Binay, Sophie. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 110, 113, 853LeBlanc, Katarina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 453Leborgne, Christian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 400Leboulch, Philippe. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 13, 228Leddon, Jennifer L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 201Lee, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 913Lee, Andrew Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 910Lee, Bokhui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 895Lee, Brendan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 46, 224, 309Lee, Brendan H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 677Lee, Chang J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 522Lee, Dean A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479Lee, Donghoon. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 553Lee, Fan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 558Lee, Gabsang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 283Lee, Gary. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 783, 798Lee, Hwang-Jae . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818Lee, Il-Kyu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 516Lee, Jessica . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 477Lee, Jung-Sun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 720, 841Lee, Kyung-Mi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 167Lee, Lily . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 674Lee, Meng Horng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 282Lee, Mi-Hyang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 384Lee, Mingul . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 202Lee, Sang kyung. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 877Lee, Sang-Jin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 384Lee, Sang-Kyung. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 407Lee, Seong-Wook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 560Lee, Suhung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 257Lee, Won Il . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 560Lee, Yeon Jyung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 406Lee, Yeon Kyung. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 128, 754Lee, Yong J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 257Lee, Young Ju . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 560Lee, Young Won . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 126Lee, Young-Sook. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 198Leen, Ann . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 179, 471EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 163Abstract Author IndexLeen, Ann M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 29, 473, 799Leger, Adrien . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 305, 365Leggiero, Eleonora . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 435Lehn, Pierre . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120, 131, 395, 745Lei, Pedro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 358, 359Lei-Butters, Diana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 4Leif, Jean . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 285Leja, Justyna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 501, 828Lekka, Malgorzata . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 533Lemiale, Franck . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 41, 165Lemiegre, Loic . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120Lemoine, Nick R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 605Lenander, Andrew W.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 20Lenne, Nathalie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 883Leon, Xavier. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 762Leonard, Helen Paige . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 411Leonard, Vincent. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 168Leong, Derek . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 321Lepetit, Helene. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777Lerario, Alberto. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 795Lesch, Hanna P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 209, 669Lesniak, Maciej S.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 191, 718, 835, 902Leung, Anders . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 169Leung, Cary H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 778, 782Leung, Kitty . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 872Levine, Bruce . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 341, 798Levings, Megan K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 79Lévis, Nicolas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 460Levy, Camille . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 336Levy, Robert J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 323, 425, 753Lewin, Alfred . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 61Lewin, Alfred S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 417, 446Lewis, Sarah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155, 290, 581Leyfman, Yan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 266Li, Baozheng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 369, 704Li, Chengwen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 160, 366, 367Li, Chengwin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 581Li, Chengyao . . . . . . . . . . . . . . . . . . . . . . . . . . 171, 518, 806, 807, 808, 854Li, Dejia. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 466, 707Li, Haitang. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 287Li, Hojun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 308Li, Hongtao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 203Li, Hongwei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 219, 518, 854Li, Hui. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 412Li, Jianfeng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 484Li, Juan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 6, 160, 420, 581, 683Li, Linhong. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 675Li, Mengxin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58, 369Li, Min . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 876Li, Pingchuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 85Li, Quanhai . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 851Li, Shulin. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 105, 484, 737Li, Songtao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 142Li, Tingting. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 171, 806, 807Li, Wei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 890Li, Weidong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 186Li, Wuping. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 2Li, Xiaohong. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 130Li, Xiaozhu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 605Li, Yan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 603Li, Yi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 234Li, Yong. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 456, 465Li, Yong-Fu. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 49Li, Yuedan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 617Li, Zhixiong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 619Li, Zhong. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 68Li, Zong. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 657Li, ZongYi . . . . . . . . . . . . . . . . . . . . . . . . . . . . 180, 264, 265, 843, 849, 863Lichty, Brian D.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39Lidonnici, Maria Rosa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 523Lieber, Andre . . . . . . . . . . . . . . . 93, 180, 264, 265, 415, 657, 843, 849, 863Lieberman, Jay R.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 687Liesner, Raphael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 92Liggitt, Denny. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 343Liikanen, Ilkka . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204Likar, Yury . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 185Liles, Conrad . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 907Liljenfeldt, Lina. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 820Liljenfeldt, Lina S. E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 486Lillicrap, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 767Lim, Arlene T.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 418Lim, Carol S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 205Lim, Kwang-il . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 299Lim, Sung-Won. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818Lima de Silva, Maria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479Limberis, Maria P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 625Lin, Amy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 411Lin, Chao. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 126Lin, Chin-Yu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 583Lin, Chung-Ren . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 882Lin, Feng. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 103, 115, 327Lin, Kun-Ju. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 583Lin, Patricia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26Lin, Xiaojing. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 694Lindberg, Mattias . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120, 131Lindholm, Leif . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 506Lindqvist, Camilla . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 83Lindqvist, Camilla A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 176


164<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexLindsey, Matt . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 281Ling, Chen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 369, 700, 704, 706Linton, Gilda M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 354Lionetti, Vincenzo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 423Lipshutz, Gerald S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 44, 224, 373Lisowski, Leszek. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 306Liu, Chao-Lien. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 535Liu, Chunyan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 181Liu, Dexi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 100, 108, 322, 484Liu, E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30Liu, Elaine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 783Liu, Enli. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288, 293Liu, Hao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288, 293Liu, Jian W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 417Liu, Jian Wen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 21Liu, Joyce C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 651Liu, Meihong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 696Liu, MengLu. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 260Liu, Minzhi M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 390Liu, Pei-Qi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 88Liu, Shihe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 247, 564Liu, Shi-he . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 892Liu, Xiaoming. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 620Liu, Xiaoxuan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 287Liu, Xiuying . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 662Liu, Ying . . . . . . . . . . . . . . . . . . . . . 180, 264, 265, 415, 657, 843, 849, 863Liu, Yong. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 429, 436, 711Liu, Yunfan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 238Liu, Zhao-Jun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 603Liu, Zhongda . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 218Llewellyn, J. Anthony . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 116Lo, Mandy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 300Lo, Mandy Y. M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 691Lo, Wen-Yi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 583Lock, Martin W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 45Lockey, Timothy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 861Logg, Christopher R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 334, 831Loh, Y. P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 143Loiler, Scott A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 66, 231Loiseau, Damien. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 745Lollar, Pete . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 527Lombardo, Angelo. . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10, 88, 286, 632, 654Long, Timothy E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 122, 125Lopez, Patricia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279Lopez, Teresita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288Lopez, Veronica M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 609, 834Lorain, Stéphanie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 154Lorilleux, Christophe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 131Lorini, Alessandro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276Lorson, Christian L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 626Loskog, Angelica S. I. . . . . . . . . . . . . . . . . . . . . . . . . . 83, 176, 453, 486, 820Lotta, Louis T.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 76Lou, Rongcheng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 819Louie, Stan F.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 522Lovric, Jasmina. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 311Low, David. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156Low, Walter C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 20Lowe, Andrew S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 394Lowe, Michelle C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 592Lowenstein, Pedro R.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26, 181, 475Lu, Aiping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 629, 635Lu, An. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 469Lu, Hui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 310Lu, J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 697Lu, Jian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 114Lu, Jing. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 65Lu, Peijuan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 890Lu, Qi Long . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 890Lu, Taihe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 222, 860Lu, Yi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 217Lu, You . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 433Lu, Yuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 700, 704, 706Lu, Yuanqing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 375Lu, Zhuoyuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 405Lucarelli, Guido. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 319Luchetti, Andrea . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 908Lukacs, Gergely . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 620Lukashov, Vladimir . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 797Luke, Jeremy M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 166, 572, 575, 738Lulay, Christina. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612Lundberg, Cecilia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 239, 350Lundberg, Kathryn. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 107Luo, Chia-Hsin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 583Luo, Mathew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279Luo, Meihui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 620Luo, Min . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 504Luo, Xianghua . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 497Luo, Xiaoyan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 142Luo, Yonglun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 91Lupo Stanghellini, Maria Teresa. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295Lupold, Shawn E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 251, 390Lusic, Hrvoje . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 744Lusky, Monika . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204Lust, John A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 218Lüttge, Doreen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 528Lv, Chengyu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 566Lyon, Deborah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288Lythgoe, Mark . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 394EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 165Abstract Author IndexMMa, Hong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 355, 534Ma, Jianjie. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 683Ma, Qiangzhong. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 27Ma, Wenqin. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 369, 704Ma, Yongjie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 100Ma, Zhijun. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 860Määttä, Ann-Marie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 209Macauley-Rambach, Shannon L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 22Machen, Laurie. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 104Machleidt, Thomas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 268Maciotta, Simona . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 464, 795MacTavish, Heather. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39Maczuga, Piotr. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 255Mader, Emily K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 488Maderna, Claudio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 413Madigan, Nicolas N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 780Madoiwa, Seiji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 421, 544Madtes, David K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 623Maeda, Michiko W.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 151Maeda, Tadao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445Maeda, Yasushi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 468, 788Maeder, Morgan L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 95Maetzig, Tobias . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284, 302, 619Maeyama, Yoshihiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678Magee, Mitchell . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 292Magnani, Zulma . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 88, 654Magnusson, Maria K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 506Maguire, Casey A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 57Maguire-Zeuss, Kathleen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 23Mah, Cathryn S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 596Mahan, John D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155Mahato, Ram I. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 84, 741Mahller, Yonatan Y.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 830Maier, Dawn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 798Maisner, Andrea . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 336Maiti, Sourindra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479Makarevich, Pavel. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 434Makino, Hir<strong>of</strong>umi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 884Malani, Nirav . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 13, 228, 283, 308, 346Maldonado, Arturo R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 830Malech, Harry L.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 220, 354, 688, 860Malgieri, Arianna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 908Malik, Alock. . . . . . . . . . . . . . . . . . . . . . . . . . . 156, 374, 454, 458, 467, 785Malik, Punam. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 280, 526, 864Malik, Vinod . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149, 155, 290, 581Malloy, Andrew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 577Malmstrom, Per-Uno . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 176Mamchaoui, Kamel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 789Mamdani, Mohammed . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 423Mancheño, Uxua . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42Manchengo, Uxua. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 810Mandel, Ron . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 147Mandelli, Giacomo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 319, 523Mangopoulos, Kathryn. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 280Mangsbo, Sara M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 176Mangus, Courtney. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 341Mani, Sendurai. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 243Maniar, Jay M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 14Mann, Anita. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 132Mann, Derek A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 716Mano, Miguel. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 311Manoli, Irini . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 135Mansfield, S. Gary . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 422Manunta, Maria D. I.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 747Mao, Hai-Quan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 124, 321Mao, Haoyu. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 446Maples, Phil. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 144Maples, Phillip B. . . . . . . . . . . . . . . . . . . 148, 213, 245, 247, 248, 252, 275,. . . . . . . . . . . . . . . 292, 335, 487, 520, 532, 564, 574, 578, 586, 731, 892Maródi, Laszló . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612Marano, Giuseppina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276Marasco, Wayne A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 645Marc, Lavertu. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 396March, Keith L.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 630Marchadier, Dawn. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 412Marcucci, Katherine T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 630Marerro, Bernadette . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 823Marianowski, Rémi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120Maric, I. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31Marigo, Valeria. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 624Marin, Virna. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 845Marinkovich, Peter M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 320Marion, Patricia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 82Marjomäki, Varpu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159Markley, John C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 655Marktel, Sarah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 319Markusic, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 371Markusic, David M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 540Marongiu, F.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139Marrocco, Elena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 624Martelli, Letizia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276Martin, Fernando L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 599Martin, William . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 43Martinez, Caridad . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288Martino, Ashley . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 540Martino, Ashley T.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 309, 363, 539Martino, Sabata . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 313, 316, 413


166<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexMartuza, Robert L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 608, 829Maruggi, Giulietta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10, 319, 346Marumoto, Tomotoshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263, 899Maruyama, Kazuo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 102Masatoshi, Ishizaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 788Maslyar, Daniel. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 35Mason, A. James . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 400Massariello, Paola. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276Massey, Paul . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 659Massie, Bernard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 463Mastrangelo, Michael A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 18Masuda, Akio. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 585Masuda, Tomoya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 591, 743Masurier, Carole . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 67, 791Matar, Majed . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893Mathiesen, Iacob . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 289Matsui, Chihiro. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 320Matsui, Hideto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 767Matsumoto, Hiroshi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 492, 831Matsumoto, Tae . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 680Mattar, C. N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223, 697Mattar, Citra. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 531Matteo, Benjamin C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 21Matthews, Qiana L.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 842Mätzig, Tobias . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 304Mauleón, Itsaso . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42Mauleon, Itsaso . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 810Mavilio, Fulvio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10, 319, 346, 351, 523Mayer, Christina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 683Maynard, Jonathan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 85Mays, Lauren E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 1Mazur, Curt . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 557Mazurier, Frédéric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 865Mazzaccara, Cristina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 435Mazzieri, Roberta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 331McAlister, Victor J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 70, 710McCants, Marjorie. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314, 411McCarty, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 814McCarty, Douglas M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 307McChesney, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 168McClure, Diane. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893McConnell, R.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 879McConnell-Smith, Audrey . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 89McCormick, Dominique . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 880McCown, Thomas J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 2, 355, 366McCoy, Jay . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 103, 327McCullagh, Karl . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 439McDonald, Robert A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 380McDonough, Samantha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 80, 505McGarrity, Gerard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 41, 646, 803McGarrity, Gerard J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 274, 422McGinley, Lisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 427McGoogan, Katherine E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 700McHugh, Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 439McIntosh, J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223McIvor, R. Scott . . . . . . . . . . . . . . . . . . . . . 868, 20, 221, 232, 325, 734, 852Mckenna, Mavis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58Mckiernan, James. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 35McKown, Thomas J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 778McLachlan, Gerry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 880McMahon, Jill. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 427McMahon, Ross . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279McMahon, Siobhan S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 780McMahon, Stephen B.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 451McMenamin, Deirdre. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 1McNamara, II, James O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 662, 663McNeer, Nicole Ali. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 285McPhee, Scott . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 581McPhee, Scott W. J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 160, 778McVey, John H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 715Mead, Andrew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156, 374, 458, 467Mead, Andrew F.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 785Mead, Andy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 454Medin, Jeffrey . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 185, 341Medin, Jeffrey A. . . . . . . . . . . . . . . . . . . . . . . . . 79, 333, 508, 509, 659, 771Medina-Echeverz, Jose. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 810Medina-Jaszek, C. Angelica . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 164Meehan, Anne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 301Mehta, Jawahar L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 437Mei, Shirley H. J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 907Mei, Z. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30Mejido, Josef . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 482Melcher, Alan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 481, 606, 607, 610, 653Melero, Ignacio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42Mello, Chris . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 674Mendell, Jerry R. . . . . . . . . . . . . . . . . . . . . . . . . . . . 149, 152, 155, 290, 581Mendoza, Janess . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 115, 327Meng, Cuixiang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 873Meng, Max . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 35Meng, Xin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 192Mercer, Andrew C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 94Meregalli, Mirella . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 460, 464, 795Merlin, Simone. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 134Merling, Randall . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 800Merlini, Luciano . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258, 666Mermod, Nicolas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 533Meroni, Germana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 624Meseck, Marcia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 38, 277EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 167Abstract Author IndexMetzger, Joseph M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 584Metzger, Michael J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 89Meunier, Isabelle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 169Meyer, Edwin M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 775Meyer, Johann . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 318Meyerholz, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 620Mezzadra, Riccardo. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 319Miao, Carol H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 326, 535, 550Miari, Reem. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616Michael, Buschmann . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 396Michaud, Annick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 784Midoux, Patrick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 324, 395Mikheeva, Galina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 725Mikkelsen, Jacob G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 695, 726Miliaresis, Jennifer . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 164Milinkeviciute, Giedre . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 349Millán, José Luis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 680Miller, A. Dusty. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 89, 623Miller, Ashley M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 380Miller, Darren . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 874Miller, Timothy J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 426, 438Millili, Peter G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 590Mims, M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30Mimuro, Jun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 421, 544Min, Dal-Hee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 895Min, Seok H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 61Mincheva-Nilsson, Lucia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 57Mineharu, Yohei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26Mineno, Junichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 652, 678Ming, Xin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 62Mingozzi, Federico . . . . . . . . . . . . . . . . . . . . . . . . . . 8, 43, 66, 546, 548, 614Mingozzi, Federico M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 308Mir, Lluis M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 853Miranda, Paula S. M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 138Miselli, Francesca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10Miskin, James . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777Mitchell, James B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 74, 77Mitchell, Marilyn. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156, 374, 454, 785Mitrophanous, Kyriacos . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777Mittal, Plavi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 796Miura, Yoshie. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263, 899Miyake, Koichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 410, 510, 680Miyake, Noriko. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 410, 510, 680Miyake, Takashi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 884Miyamoto, Shohei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 192Miyanohara, Atsushi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 765Miyoshi, Hiroyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 899Mizrak, Arda . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 665, 846Mizuguchi, Hiroyuki . . . . . . . . . . . . . . . . . . . . . . . . . 207, 216, 480, 537, 591Mizukami, Hiroaki . . . . . . . . . . . . . . . . . . . . . . . . . . 207, 421, 428, 544, 554Mizusawa, Hidehiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 259Mockaitis, Keithanne. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 353, 356Mockenhaupt, Stefan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 660Modlich, Ute . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 304, 318, 619Mody, Disha A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 222Moen, Robert C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445, 622Moghimi, Babak. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 146, 763Mohseni, Mahboobeh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 871Moimas, Silvia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 423, 431Molas, Maria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 762Moldt, Brian. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 695Monahan, Paul E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 534Monnier, Nilah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 796Monsma, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 487Montazami-Astaneh, Kaweh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 95Montgomery, Chrystal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149Montier, Tristan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120, 131, 395, 745Montini, Eugenio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297, 298, 613Moon, Anne. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33Moon, Ju Young . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 386Moon, Lawrence. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 451Mooney, Michael A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 235Mooney, Sean . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 353, 356Moore, Nicole M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 589Moot, Robby . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 230Morcinek, Nick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 681Morcos, Paul A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 49Moreau-Gaudry, François . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 865Moreno, Amira Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 161, 452, 752Moreno, Peter J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 272Morgan, R. A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31Morgan, Richard A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 190, 476, 478Morgello, Susan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 38Morishita, Ryuichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 884Moritz, Thomas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 528Morizono, Hiroki. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 416Mornet, Emmanuel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120Moroz, Maxim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 185Morral, Nuria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 52Morris, Caroline . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 483Morris, John C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 80, 483, 505Morris, Robert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250Morrissey, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 398, 848Morrow, <strong>Gene</strong>vieve . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 242Morrow, M. P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 648Morrow, Matthew. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 103Mossalam, Mohanad. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 205Mossoba, Miriam . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 659


168<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexMoulay, Gilles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 67, 791Moullier, Philippe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19, 305, 365Mouly, Vincent . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 789Mowa, Betty . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 82Moya, Marta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 762Mrak, Emanuela. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 315Mrugala, Maciej . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 294Mu, Xiaodong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 456, 465Muñoz, Nina M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 909Muñoz, Sergio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 762Mueller, Christian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 51, 53Mueller, Steffen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 372Muenzer, Joseph . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 328Muether, Nadine. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 636Muir, Lindsey A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 792Mujagic, Edin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 430Mukherjee, Sayandip . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 866Mukhopadhyay, Debabrata. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 606Müller, Oliver J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 241, 432Mullins, Charles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 639Multhaup, Megan M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 221, 232Munden, Regina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 1Muniz Pello, Oscar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 88, 654Murakami, Akikazu. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 645Muralidharan, Sujatha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 28Murillas, Rodolfo. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 351Murphy, Michael P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 269, 272, 272Museri, Adi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 719Mussolino, Claudio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59, 624Mustafa, Sadaf. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 382Muthumani, K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 115Muul, Linda . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296Mykhaylyk, Olga . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 693Myung, Chang-seon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 896NNa, Chang-Rim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 731Nace, Rebecca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 604Nagasaki, Takeshi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 397Nagasaki, Yukio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 246, 253Nagata, Keiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 236Nagata, Tetsuya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 56, 565Naik, Rangeetha. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 132Naik, Rangeetha J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 127Naik, Shruthi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 604Naik, Ulhas P.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 590Nair, Smita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 561Najder, Oliwia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 875Nakagami, Hironori. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 774Nakai, Hiroyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 7, 141, 580, 703Nakajima, Satoru . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678Nakamura, Akinori . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 56, 151, 565Nakamura, Masakuni . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 167Nakamura, Takafumi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 34, 192, 840Nakamura, Takashi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 401Nakano, Kenji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 640Nakashima, Hiroshi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 491Nakashiro, Koh-ichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 493Nakatsu, Yoshimichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 97Nakazawa, Yozo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 179, 813Nalam, Madhusudhana R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 404Nalbantoglu, Josephine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 463Naldini, Luigi . . . . . . . . . . . . . . . . . . . . . . . . . . . 10, 88, 225, 276, 286, 297,. . . . . . . . . . . . . . . 298, 313, 315, 316, 331, 337, 413, 545, 613, 632, 654Nan, Zhenhong. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 20Nande, Rounak . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 847Narayanan, Priyadharshini . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 474Narisawa, Sonoko. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 680Narrow, Wade . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 23Narrow, Wade C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 18Nathwani, A. C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223Nathwani, Amit C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 668Naundorf, Sonja . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612Navarro, Claire . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 460Neeley, Andy W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17Neely, Andrew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 71Neff, Preston C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 287Negishi, Yoichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 102Neglia, Simona. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59Negre, Didier . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 336Negre, Olivier. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 228Nemunaitis, Gregory . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 586Nemunaitis, John . . . . . . . . . . . . . . . 35, 144, 148, 196, 213, 245, 247, 248,. . . 252, 275, 292, 335, 487, 520, 532, 536, 564, 574, 578, 586, 731, 892Neri, Marcella. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258Neri, Margherita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 316, 413, 632Neschadim, Anton. . . . . . . . . . . . . . . . . . . . . . . . . . . 185, 333, 508, 509, 771Nettelbeck, Dirk M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 381, 387, 641Neuberg, Patrick. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 883Newman, Nikki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 873Ng, P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 879Ng, Philip . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 388, 542, 616, 639Nguyen, Andrew T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 731Ni, Xiahoua . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 251Nichols, Timothy. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 613Nichols, Timothy C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 615Nicholson, Linda J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 716Nickens, Brittani . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 785EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 169Abstract Author IndexNickerson, Michael L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 365Nicklin, Stuart A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 380, 715, 723Nicoletti, Roberto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295Nicolson, Sarah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 2Niedernh<strong>of</strong>er, Laura J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 629, 635Nielsen, Peter E.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 109Nienhuis, Arthur W. . . . . . . . . . . . . . . . . . . . . . . 338, 339, 449, 668, 733, 867Nieva, Jorge. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33Nikhamin, Yan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 815Nikolskaya, Alena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 402Nilsson, Berith . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 501, 828Nimesh, Surendra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 396Nimjee, Shahid M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 667Nishikawa, Makiya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 670, 862Nishimura, Jun-ichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 667Nishina, Kazutaka. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 259Nishiyama, Akiyo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 151Nishiyama, Nobuhiro. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 891Nissenson, Allen R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616Niu, Guoqin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 256Nixon, Alan J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 912No, Daniel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 772, 794Noble, Misty L.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 326Nokay, Bahar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 887Nokisalmi, Petri . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 291Nolta, Jan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 281Nordheim, Alfred . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 241Nordling, Diana L.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 280Noske, Nadja . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 92Novelli, Giuseppe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 908Noviello, Maddalena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295Nowrouzi, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223Nowrouzi, Ali . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 305, 612Numasaki, Muneo. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 851Nunan, Robert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 536Nunez-Alonso, Graciela A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 123OObach, Mercè. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 140Obach, Merce. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 762O’Brien, Timothy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 424, 427, 439, 780O’Callaghan, Christopher . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 747Ochiai, Hiroshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 240Ochs, Hans . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 529Odom, Guy L.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 461, 584O’Donnell, Patricia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 598, 758O’Driscoll, Caitriona. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 254Oehler, Elise A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 599Oette, Sharon M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445, 622Offord, Chetan P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 208Ofstein, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 269Ogura, Manabu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 428Oh, Il Ung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 112Oh, Seeun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 257Oh, Sung-Min. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818Ohashi, Kazuo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 141, 419, 903Ohlemiller, Kevin K.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 768Ohlfest, John . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 826Ohmori, Tsukasa. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 421, 544Ohno, Kinji. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 585Oikawa, Tatsuo. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263Oishi, Motoi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 246, 253Ojima, Iwao. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 266Okada, Hironori . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 151, 582Okada, Michiyo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263Okada, Takashi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 151, 582, 585Okamoto, Sachiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 652Okamoto, Shinya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 851Okamura, Haruki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 500Okano, Kiichiro. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445Okano, Teruo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 141, 419, 903Okazaki, Akiyo. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 236Okazawa, Hitoshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 565Oksanen, Minna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 193, 538, 658Okun, Alex. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616Olivares, Jairo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 292Olivares, Simon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479Oliveira, Giacomo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295Olsen, John . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 355Olsen, John C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 881Olson, Erik. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 734Olson, Erik M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 234, 858Olson, Erik R.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 325, 868Olsson-Strömberg, Ulla . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 83, 820Olszewska, Malgorzata . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 32O’Malley, Janis. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36O’Malley, Thomas. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 758Oneal, Michael J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 80, 505O’Neill, Ann Marie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 379Onishi, Akira . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 428Onodera, Masafumi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678Orimo, Hideo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 680Orkin, Stuart H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 338Orlacchio, Aldo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 413Osawa, Kanako . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 236Oshimura, Mitsuo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 236O’Sullivan, G. C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 194O’Sullivan, Gerald C. . . . . . . . . . . . . . . . . . . . . . . . . . 398, 705, 736, 812, 848


170<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexO’Sullivan Murphy, Bryan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 51, 53Otoi, Takeshige. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 804O’Toole, Daniel. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 427Otsu, Makoto. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678Otsuka, Makoto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 749Ou, Wu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 649Owen, Carina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 602Owens, Roland A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 70, 710Ozaki, Kazunari . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 884Ozawa, Keiya. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 207, 421, 428, 544, 554Ozdener, Baris G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 846Ozelo, Margareth . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 767Ozlem, Senol . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 846PPacholska, Agnieszka . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 212, 512Pacienza, Natalia A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 509, 771Pack, Daniel W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 119, 595Padegimas, Linas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445, 622, 776Padmashali, Roshan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 282, 359Pagliara, D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 178Palanca-Wessels, Maria C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 558Palazón, Asis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42Palczewski, Krzyszt<strong>of</strong> . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445Paldor, Mor . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16Palfi, Stephane. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777Palmer, D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 879Palmer, Donna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 75, 388, 639, 677Palmer, Donna J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 542Pan, Deng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 321Pan, Haiying . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 465Pandha, Hardev . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 606Paneda, Astrid . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 485Pang, Jijing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17Pankhong, Panyupa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 477Papanikolaou, Eleni. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 338Papapetrou, Eirini . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 729Papapetrou, Eirini P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 283Papayannopoulou, Thalia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137Pappen, Beena O.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 275, 292, 487, 532Pâques, Frédéric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 87, 462Parato, Kelley. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33Parfyonova, Elena. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 434Pariétti, Véronique . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 533Paris, Clément . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 883Park, Eugene . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 783Park, Harriet . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162Park, In-Kyu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 399, 751, 900, 904Park, Jeong-Sook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 896Park, Keerang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 722Park, Ryan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 200Park, Sang H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 687Park, Sang Il . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 128, 406, 754Park, Yeon Hee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 85Park, Yong Serk . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 128, 406, 754Park, Younjoo. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 722Parker, Alan L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 380, 715Parker, Colleen H.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 907Parker, Ernest T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 527Parker, Josh. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 852Parkhurst, Maria R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 25, 190Parolini, Daniele . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 460, 464, 795Parsons, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 874Parsons, David W.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 621Partridge, Terence . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 56Paruzynski, Anna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612Pasceri, Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 300Passineau, Michael J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 104Passini, Marco A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55Pasternak, Alexander. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 797Pastore, Joseph M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 426, 438Pastore, Lucio. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 435Patel, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 648Patel, Ami . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 169, 724Patel, Manij . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 881Patel, Priti M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 639Patrene, Ken . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 157Pattarini, Lucia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 431Paulk, Nicole K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 99Pavelko, Kevin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 607Payen, Emmanuel. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 228Peñuelas, Iván . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42Pearlman, Andrew L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616Pease, Larry. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 607Peccate, Cécile . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 154Peccatori, Jacopo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295Pedersen, Alexandra G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 787Pedro, Helder L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 802Peebles, D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223Peelman, Luc . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 875Pei-Qi, Liu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 654Pellin, Danilo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 345, 346Peluffo, Hugo. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 451Pelusio, Adina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33Penaud-Budloo, Magalie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 305, 365Pence, Casey . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893Peng, Baowei. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 535, 550Peng, Hairong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 456EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 171Abstract Author IndexPeng, Kah-Whye. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 488Peng, Liaomin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 517Peng, Ling . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 287Penn, Marc S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 426, 438Pepe, Martino . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 423Perabo, Luca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 5Peretz, Mary E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 301, 685Perez, Omar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 492, 831Perez, Paola . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 761Perini, Giovanni . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258Perkins, Jeanne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314Perna, S.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 178Perna, Serena Kimi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 346Perricaudet, Michel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 853Perrin, George Q. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 363, 370Perrone, Daniela. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 666Persons, Derek . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 867Persons, Derek A. . . . . . . . . . . . . . . . . . . . . . . . . . . . 338, 339, 524, 525, 861Persson, Jonas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 180, 264, 843, 849, 863Peshwa, Madhusudan V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 675Pesonen, Sari. . . . . . . . . . . . . . . . . . 177, 193, 197, 204, 291, 538, 549, 658Pestina, Tamara I . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 525Pestronk, Alan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 152Peters, Brandon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 356Petersen, Line . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 85Petersen, Line B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 695Peterson, Kenneth. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 340Petrov, Mihail. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156, 454, 458, 600, 785Petrov, Mike . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 374Petrs-Silva, Hilda. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 61Petry, Harald . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 249, 255Pfaff, Nils . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 528Pfeifer, Alexander . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 693Phadke, Anagha P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 148, 196, 252, 586Phalon, Connor. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 245Pham, Thu. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 405Phillips, Jana L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 364, 367, 403Phillips, Neil. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 760Phillips, Terry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 482Piao, Jingshu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 97Piao, Wenying . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 259Piccolo, Pasquale . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 75Pichavant, Christophe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 462, 790Pichon, Chantal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 324, 395Picquet, Erwan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 745Pien, Gary . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 43Piersanti, Stefania. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 133Pierson, Tyler Mark . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 235Pietruszka, Marvin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 772Piguet, Françoise . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19Pikkarainen, Jere T.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 209, 669Pilla, James J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 600Pillet, Stephane . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 169Piras, Elena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 453Piraud, Monique . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19Pisano, F.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 360Pizzitola, Irene . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 845Plank, Christian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 693Plotkin, Jason. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 184, 655, 911Podetz-Pedersen, Kelly. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 221Podetz-Pedersen, Kelly M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 232Podetz-Petersen, Kelly M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 852Podhajcer, Osvaldo L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 609, 834Podsak<strong>of</strong>f, Gregory M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 8Poeschla, Eric M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 301, 685, 804Polach, Kevin J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893Polard, Valerie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 110, 113, 853Politi, Letterio S.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 313, 315Ponder, Katherine P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 315, 598Ponnazhagan, Selvarangan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 63, 507, 906Ponomarev, Vladimir . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 184, 185, 333Ponzoni, Maurilio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297Pope, M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 648Porada, Chris D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 857Porada, Christopher D.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 551Porcellini, Simona . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 644Postema, Michiel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 324Potter, Clinton S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 393Pouliot, Frédéric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 714Poulsen, Hans S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 210, 511Poupiot, Jérôme . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 48Pourfathollah, Ali Akbar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 871Powell, Kimerly . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 836Powell, Suzanne. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 471Power, Anthony T.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 37Powers, William . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 328Pozmogova, Galina E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 735Pozza, Chiara. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276Pratico, Elizabeth D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 561Pratt, William D.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 643Premkumar, Muralidhar H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 677Press, Michael F.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 558Press, Oliver W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 558Preston, Thomas J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149Preuss, Meredith A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36, 503Prieto, Jesús . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42Prieto, Jesus . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 485, 494, 708, 810Pringle, Ian A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 727


172<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexPristinski, Denis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 589Privman, Eyal. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16Proto, Jonathan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 635Protzer, Ulrike . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312Provasi, Elena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 88, 632, 654Pucci, Ferdinando . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 331Pule, Martin. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 845Pulicherla, Nagesh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 364Pulido, Jose . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 606, 607, 610, 653Puligadda, Uday . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 423Pun, Suzie H.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 730Pupko, Tal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16Puttaraju, Madaiah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 422QQanud, Khaled . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 423Qasim, Waseem . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 158, 647Qazi, Aamer M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250Qi, Feng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 566Qi, Yanfei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 518Qiang, Ming. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 211Qiao, Chunping. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 6, 420, 683Qiao, Jian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 195, 495Qin, Bin. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 567Qin, Elizabeth. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 768Qin, Qiuyue . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 164Qin, Rui. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 505Qiu, Guozhen. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 806Qiu, Xiaohong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 402Qiu, Xingping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 756Qiujie, Jiang. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 320Qu, Guang. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 69, 673Qu, Jinrong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 184Quach, Eric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 782Quattrini, Angelo. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 313, 315Quezada, Valerie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279Quinn, Thomas. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 674Quintanilla, María Elena. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 766Quintino, Luis. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 350Quiocho, Florante A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 574Quispe, Joel. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 393RRaake, Philip W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 432Rabinowitz, Joseph . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 600Rabinowitz, Joseph E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 366Rabkin, Samuel D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 608, 829Rachinel, Nicholas. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 336Radd, Katherine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 422Rader, Daniel J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 412Radrizzani, Marina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225, 276, 613Radu, Antenata . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 320Radu, Antoneta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 344, 676Raffeld, M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31Raggi, Geno. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 457Rah, Dong Kyun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 516Rahim, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 697Rainusso, Nino . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 470Rajagopalan, Malolan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 514Rajecki, Maria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 197, 291Rajendran, Simon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 398, 812, 848Rajput, Kamal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 242Raki, Mari . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 197, 291, 549, 658Rakugi, Hiromi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 884Ralph, Scott. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777Ramesh, Rajagopal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 243Rameswaran, Rameswari. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 576Ramezani, Ali. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 342, 690Ramirez, Sean M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 125Ramlogan, Charmaine A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 483Ramo, Kasmir . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 448Ramos, C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30Ramos, Carlos A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288, 799Ramsay, Tim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 779Rantanen, Ville. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204Ranzani, Marco . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297, 298, 613Rao, Donald D.. . . . . . . . . . . . . . . . . . . . . 245, 247, 248, 252, 532, 564, 892Rao, Mahendra S.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 268Rao, Thapi D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 183Raoul, Sylvie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19Rapp, Brian M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 269, 272Rasmusson, Ida . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 453Rathod, Trushar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 143Ratiglia, Roberto. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59Ratner, Nancy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 201Rauschhuber, Christina. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 385Raut, Sanj . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 134Ravin, Suk See De . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 688Rawlings, David J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . 90, 226, 343, 529, 550Ray, William C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 699Razini, Paola . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 460, 464, 795Razquin, Nerea. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 889Read, Sarah P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 447Readhead, Carol . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 349Rea-Ramsey, Angela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893Reay, Daniel P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 457Rebar, Edward . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 12Rebello, Tessio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 41, 646, 803Recchia, Alessandra. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10, 346EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 173Abstract Author IndexRecchia, Fabio A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 423Reddy, Anilkumar K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 677Reeves, Lilith R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 280Reginato, Silvia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 597Reichenbach, Janine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 233, 869Reik, Andreas. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 88, 618, 654Rejman, Joanna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 261, 875Remant, K. C. B.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 129, 755Remaut, Katrien . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 742Remoli, Cristina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 133Remy, Philippe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777Ren, Yan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 775Ren, Yong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 124Restifo, Nichaolas P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 478Restifo, Nicholas P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 190Rex, Tonia S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 441Reyes-del Valle, Jorge . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 168Ricca, Alessandra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 316Rice, Jennifer . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893Rice, Kevin G.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 117, 594Richard, Emmanuel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 865Richard, Isabelle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 48, 793Richards, Amy M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55, 557Richly, Heike . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 664, 887Riddell, Stainly . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 553Riddell, Stanley R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 618Riley, James. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 341Riley, John P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 190Rimessi, Paola . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258, 666Riminucci, Mara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 133Rimler, Avi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616Rindt, Hansjorg. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 626Rinkoski, Thomas A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 804Ripamonti, Anna. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225Ripoll, Cynthia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314, 411Rising, Aaron C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 147Ritsema, Tita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 249, 255Rival-Gervier, Sylvie. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 300, 691Rivera, Angel A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 834Rivers, Angela E.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 363, 370, 706Rivière, Isabelle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 184Rivière1, Isabelle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 911Riviere, Isabelle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 32, 283Rivkin, Ludmila. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16Riz, Irene . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 690Rizzardi, Gian-Paolo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 644Robbins, Joan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 492, 831Robbins, Paul D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 457, 629, 635Robbins, Paul F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 25, 190Robert-Richard, Elodie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 865Roberts, David D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 731Roberts, Marie S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 22Robinson, Andria R.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 629Roca, Carles. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 140Roccio, M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 360Rockey, William M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 662, 663Rockhill, Jason . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 294Rodgers, John . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 46Rodino-Klapac, Louise R. . . . . . . . . . . . . . . . . . . . . . . . . . . 149, 155, 290, 581Rodrigues, Ana F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 278Rodriguez, Luis. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 184Rodriguez, Ronald. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 382, 390Rodriguez, Stephen. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 739Roescher, Nienke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 870R<strong>of</strong>fler, Steve . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 180, 863Rogers, Faye . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 285Rojas, Juanjo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 291Roland, Herzog W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 68Rollins, Lisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 656Rommelfanger, Diana M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 208Roncarolo, Maria G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225Roncarolo, Maria Grazia. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 345, 545Ronen, Keshet . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 228Roode, Luke. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 420Roodman, G. David. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 157Rooney, C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30, 178Rooney, Cliona M. . . 28, 29, 179, 288, 293, 472, 473, 656, 672, 799, 813, 816Rooney, Gemma E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 780Roques, Caroline. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 817Rosada, Cecilia. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 695Rosales-Quintero, Xiomara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 290Roselli, Emanuela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 319Rosen, Elliot D.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 630Rosenberg, Jonathan B.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 161, 452, 752Rosenberg, S. A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31Rosenberg, Steven A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 25, 190, 476, 478Rossetti, Francesca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276Rossi, Claudia. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 319Rossi, John J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 287, 555, 661, 805, 888Rossi, Settimio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59Rostomily, Robert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 294Roujeau, Thomas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19Rousseau, Joel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 462, 769Rousseau, Raphael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 845Rowzee, Anne M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 74, 77, 143Roy, Andre J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 527Roy, Dominic G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 37Roy, Laura M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 681


174<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexRoy, Soumitra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 164Roybal, Jessica L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 344, 676Ruan, Li . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 114, 172Ruberte, Jesus . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 762Rubinacci, Alessandro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 315Rufibach, Laura E.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 796Ruhn, Kelly M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 773Ruiz, Rafaela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 52Russell, David W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 234, 858Russell, Stephen J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 195, 203,. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 336,490, 495, 499, 604, 684Russo, Fabio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 758Ruzo, Albert. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 140Ruzsics, Zsolt . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 385, 712Rwigema, Jean-Claude. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 157, 514Ryals, Renee C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17, 71Ryan, Deborah A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 18Ryan, Martin D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 233Rybalkin, Igor. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 434Ryffel, Bernhard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 552Ryoo, Soo-Ryoon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 895Ryu, Byoung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 529Ryu, Byoung Y.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 90, 226, 343, 909Ryu, Jihoon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 242, 841SSaadatzadeh, Mohammad R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 272Saadatzadeh, Reza. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 269Sabatelli, Patrizia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 666Sabourin, Kara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33Sacchetti, Luciana. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 435Sachar, Einat . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16Sack, Brandon K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 68, 539Sadadcharam, Mira. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 736Sadelain, Michel. . . . . . . . . . . . . . . . . . . . . . . . . 32, 184, 185, 283, 655, 911Saechao, Chai . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 772, 794Saeki, Koichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 303, 634Saenz, Dyana T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 301, 685, 804Safinya, Cyrus R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 393Saga, Kotaro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 187Sagawa, Hiroaki. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 674Saggio, Isabella . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 133Saha, Sunandan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 813Sahenk, Zarife . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149, 155, 290, 581Saighal, Sheena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 625Saini, Massimo. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 337Sainte-Catherine, Vincent . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 110, 113Saito, Nobuhito . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 832Saito, Takahiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 419Saito, Takashi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 56, 565Sakata, Yoichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 421, 544Sakiyama, Yukio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678Sakiyama-Elbert, Shelly E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 589Sakuma, Toshie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 688Sakurai, Fuminori . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 537Sakurai, Kumi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 888Sakurai, Yoshihiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 903Sakurai, Yu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 246Salavert, Ariana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 762Salazar, Felix . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 82Salerno, Angela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 796Sallach, Jessica . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 5Sallberg, Matti . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 111, 289Salles, Gilles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 336Salomon, Daniel R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 408Salomoni, Monica. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276Salsman, Vita. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 179, 470, 471Saltzman, Daniel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 852Saltzman, W. Mark. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 285Salvatore, Francesco . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 435Salwa, Slawomir P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 736Samaranayake, Haritha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 209, 669Sampath, Alapakkam P.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 21Sampath, Prakash . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 182Sampedro, Ana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42Samulski, Jade. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 581Samulski, Jade J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 160Samulski, R. Jude. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 2, 60, 160, 366, 581Sanchez, Ceidy. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 28, 672Sanchez, Massimo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 908Sanchez-Bonilla, Marilyn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 330Sanchez-Salazar, Jorge . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 517Sanders, Niek N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 875Sandhu, Arbans . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 625Sands, Mark S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 22, 768Sanges, Daniela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 624Sangiuolo, Federica. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 908Sankaran, Vijay G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 338Sanmiguel, Julio C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 759Sano, Akiko. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 236Santiago, Virgínia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 570Santilli, Giorgia. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 866Santistevan, Nicholas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 355Sanvito, Francesca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297, 315Sapag, Amalia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 766Sardesai, N. Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 648Sardesai, Niranjan Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 103, 115, 327, 477Sardi, S. P.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 175Abstract Author IndexSargent, R. G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 913Sarkar, Devanand . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 503Sarkar, Rita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 134Sarkaria, Jann N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 499Sarkioja, Merja. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 549Sarwari, Roya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 617Sasaki, Erika . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 899Sather, Blythe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 529Sather, Blythe D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 226, 343Sato, Makoto. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 714, 850Sato, Takeya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 333, 508Sato, Yusuke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 894Sauth<strong>of</strong>f, Harald . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 713Savarala, Sushma. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 757Savilahti, Harri . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 13Savoldo, B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30, 178Savoldo, Barbara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288, 472Savontaus, Eriika . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 145Savontaus, Mikko. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 145Sawall, Stefanie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 833Sawari, Roya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 530Saxena, Romil . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 52Saydam, Okay . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 665, 846Sazani, Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 56Scaife, Matthew D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 509, 771Scallan, Martina F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 705Scaramuzza, Samantha. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225Schöler, Hans. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284Schürmann, Nina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 660, 886Schaffer, David. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 299Schaffer, David V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 8, 442, 802, 805Schaller, Torsten . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 647Schambach, Axel . . . . . . . . . . . . . . . . . . . 284, 302, 304, 318, 528, 619, 869Scharenberg, Andrew M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 90, 909Schatzberg, Scott . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 467Scherman, Daniel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 67, 400, 791Scheulen, Max E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 664, 887Scheurer, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 471Schiedlmeier, Bernhard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 619Schindler, John. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288Schira, Giulia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 337Schlachterman, Alexander . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 308Schlegel, Philipp . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 432Schleifman, Erica B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 285Schlesinger, Pnina. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 719Schlesinger, Sarah J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162Schmidt, Claudia. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162Schmidt, M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223Schmidt, Manfred . . . . . . . . . . . 138, 286, 297, 298, 305, 345, 346, 347, 612Schmidt, Manuel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 189, 664, 822, 887Schneider, Kellie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36Schroeder, Mark A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 499Schroeder, Timm. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284Schr<strong>of</strong>f, Matthias . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 189, 822Schuldt, Nathan J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 651Schuldt, Nathaniel J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 638Scott, Jeannine. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 638Scott, Stephanie. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 487Scotton, Chiara. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258Scruggs, Brittni. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314, 411Seethammagari, Mamatha. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 474Seger, Reinhard A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 233, 869Segura, Maria Mercedes . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 573Seidler, Daniela G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 198Seinstra, Beatrijs. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 815Selby, Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 606, 653Semaan, Assaad. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250Semenova, Katerina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 158Senac, Julien S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 701Sena-Esteves, Miguel. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 57, 450Sengupta, Sadhak . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 718Senol, Ozlem . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 665Senyukov, Vladimir . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479Senzer, Neil . . . . . . . . . . . . . . . . . . 148, 196, 213, 245, 247, 248, 252, 292,. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 335, 487, 532, 536, 564, 586, 892Seo, Hae-Hyun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 384Seregin, Sergey S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 638, 651Sergi Sergi, Lucia . . . . . . . . . . . . . . . . . . . . . . . . . . . 297, 313, 316, 545, 613Serguei, B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26Servais, Elliot . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 184Serve, Kinta. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 330Sesenoglu-Laird, Ozge . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445, 622Sest, Maike . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 239Sethupathy, Praveen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 54Sevin, Caroline . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19Seward, Shelly M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250Seymour, Len. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 602Seymour, Len W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 732Shabashvili, Arseniy E.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 518, 854Shafer, Jessica A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 473Shaffer, Donald . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 471Shah, Ami . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296Shah, Rachel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 308Shaheen, Sharif M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 401Shahrokhi, Somayeh. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 871Shammas, Masood A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250Shan, Changguang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 432Shanker, Manish. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 243


176<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexShao, Hongwei. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 603Sharif, Faisal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 439Sharifi, Zohre . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 871Sharma, Nynne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 726Shashkova, Elena V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 489Shaw, Georgina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 270Shaw, Kit L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296Shayakhmetov, Dmitry M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 541Shchelochkov, Oleg A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 677Shea, Lonnie D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 121, 764, 770Sheehan, Andrea M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 293Shen, Hui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 709Shen, Shen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 62Shen, Xuefei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 103, 115, 327, 477Shen, Ying. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 713Shenker, Larissa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 185Shenker, Larrisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 184Sher, Yu-Ping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 844Shevchenko, Eugeniy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 434Shevelev, Alexander . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 434Shi, Qin. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 756Shida, Hisatoshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 34, 840Shihabuddin, Lamya S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55, 557Shiku, Hiroshi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 652Shilling, Christopher . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 581Shilling, Christopher J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155, 290Shim, Kyu-Woong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818Shima, Midori . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 903Shimabukuro-Vornhagen, Alexander. . . . . . . . . . . . . . . . . . . . . . . . . . . 664, 887Shimada, Hideaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 851Shimada, Takashi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 410, 510, 680Shimamura, Munehisa. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 774Shimizu, Hir<strong>of</strong>umi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 419Shimizu, Hiroyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 192Shimizu, Keiji. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 513Shimizu, Masaki. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 227Shimoda, Shinya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 899Shin, Jin-Hong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 151, 587, 601Shin, Qiaoyun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 731Shin, Seong-Phil . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 384Shin, Young Eun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 128Shirakawa, Toshiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 167Shirazi, Rahau S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 393Shirley, Shawna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 823Shi-Suo, Du . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 73Shitara, Shingo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 236Shivakumar, Rama . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 675Shontz, Kimberly . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149Shoshani, Ehud . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616Shovlin, Lindsay . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 41Shu, Yuelong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 172Shuai, Lifang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 171, 807, 808Shui, Hongshu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 620Shukla, Ravi S.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 567Shukla, Vasundhara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 132Shultz, Leonard D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 285Siddiq, Ishita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 783Sidow, Arend . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 14Siegal, <strong>Gene</strong> P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36Silbergeld, Daniel L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 294Siler, Ulrich . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 233, 869Silka, Kimberly A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 21Silva, Ana Carina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 579Silva, Flavia H.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 440Simão, Daniel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 579Simioni, Paolo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 615Simonelli, Francesca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59Simonsson, Bengt. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 83, 820Simula, Pekka . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 177Singh, Anand Kamal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 132Singh, Harjeet . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479Singh, Rakesh K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 593Sirrka, Liisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 538Sitaraman, Varsha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 372Sivakumar, Kumaraswamy. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155Skarica, Mario . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 859Skiena, Steven. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 372Skinner, Amy M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 692Skog, Johan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 57, 846Skripchenko, Yelena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 274Skvorak, K. J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139Slawin, Kevin M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 474Sleep, Susan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 668Sleeper, Meg M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 433, 598Sleer, Leanne. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 536Sloan, Jennifer . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 135Slobodkin, Gregory . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893Sloniecka, Marta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204Slovin, Susan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 184Smerdou, Cristian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42Smith, Bruce F.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 379, 587Smith, Carol. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162Smith, Edvard C. I. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 556Smith, Jeffrey S.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 542Smith, Laura J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 3Smogorzewska, Monika . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296Snead, Nicholas M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 888Snyder, Brooke R.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 778, 782EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 177Abstract Author IndexSnyder, Jenni A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 310Snyder, Richard O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 305, 365Soballe, Kjeld. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 787Sobczak, Krzyszt<strong>of</strong> . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 49Sobolewki, Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 425Soden, Declan M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 736Soedling, Helen M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 716Sokolic, Robert. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296Soldati, Corrado . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295Söllü, Cem. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 95Soma, Emilienne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 110, 113Somanathan, Suryanarayan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 543Somia, Nikunj V.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 221, 232Sommerfield, Marla . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 488Somoto, Vivian. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 440Sondhi, Dolan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19Song, Cunxian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 753Song, Hyung Geun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 406Song, Jae J.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 257Song, Min-sun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 888Song, Shuxian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 326Song, Sihong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 375, 775Song, Xiao-Tong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 656Song, Xiatong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 816Sørensen, Charlotte Brandt. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 91Sorenson, Brent . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 852Sorrentino, Brian P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 222, 354, 860, 861Sosale, Nisha. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 357Sousa, Marcos F. Q. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 579Soustek, Meghan S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 596Sowers, A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 74Sowers, Anastasia. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 77Spanggaard, Iben . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 106Sparks, Jeff . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893Spence, Alexander . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 294Spencer, David M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288, 474Spencer, H. Trent . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 527, 814Spencer, H. Trent B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 230Spencer, Jacqueline F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 489Spitalieri, Paola . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 908Spratt, S. Kaye. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 783, 798Sprengel, Rolf . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 241Spriggs, David R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 183Springer, Joseph . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 44, 224, 373Springfeld, Christoph . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 833Spyridonidis, Alexandros . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 340, 729Squillace, Karen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 633Sreenath Nagamani, Sandesh C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 677Srinivasan, Rahul . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 781Srivastava, Arun . . . . . . . . . . . . . 61, 363, 369, 370, 540, 587, 700, 704, 706Stabenow, Dirk. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312Stalvey, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 444Stamatoyannopoulos, George. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137Stanek, Lisa M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55, 557Staunstrup, Nicklas H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 726Staunstrup, Nicklas Heine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 91Stavrou, Eleana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 340, 729Stayton, Patrick S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 558Stedman, Hansell . . . . . . . . . . . . . . . . . . . . . . . 156, 374, 454, 458, 467, 785Stedman, Hansell H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 600Steel, Jason C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 483Steet, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 759Stefanska, Anna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159Stefanski, Jolanta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 32, 184Steffes, Christopher. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250Stein, David. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 646Stemmer-Rachamimov, Anat O.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 665Stenderup, Karin. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 695Stephenson, Joe. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33, 35Sterling, Brigid V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 226Stern, Baruch S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616Stetler-Stevenson, M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31Stevenson, Mark . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 732Stewart, Duncan J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 907Stirling, Brigid . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 529Stirling, Brigid V.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 343Stocca, Alessia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 427Stockdale, Katie R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 662, 663Stoddard, Barry L.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 89Stok, Merel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 315Stone, Brandon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 514Stone, Daniel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 8Storb, Rainer . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 553, 623Straath<strong>of</strong>, Karin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288Strauss, Robert. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 265Strom, S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139Stroup, Katie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 689Studer, Lorenz . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 283Su, Leonard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 454Su, Qin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58, 362, 450, 671Suda, Takeshi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 322Sudol, Kelly L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 18Sudres, Muriel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 552Suehara, Tetsuya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 670Suehiro, Youko. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263Suel-Petat, Laurence . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 793Suga, Tomohiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 468Sugiyama, Daisuke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263


178<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexSugiyama, O.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 687Sukegawa, Jun. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 508Sule, Gautam. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 46Sullenger, Bruce A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 561, 667Sullivan, Millicent O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 590Sullivan, Timothy A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 441Sumaroka, Marina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 600Sumimoto, Hidetoshi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 480Sumiyoshi, Teiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 522Sumner-Jones, Stephanie G.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 880Sumners, Colin. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 518, 854Sun, Baodong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 47, 136Sun, Bo K.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 257Sun, Lan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 433Sun, Q. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139Sun, Samuel S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 326Sun, Wenchao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 746Sun, Xingshen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 620Sundaram, Ramakrishnan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 852Surace, Enrico M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59, 624, 702Surosky, Richard. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 783Sürth, Julia D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 304Suwanmanee, Thipparat . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 534Suzuki, Kei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 184Suzuki, Masataka. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 46, 224, 309, 642Suzuki, Ryo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 102Suzuki, Ryosuke. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 748Suzuki, Takayuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 537Suzuki, Toru. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 216Suzuki, Yumi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 585Swaim, William D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 77Swain, JaBaris D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 600Swaney, William P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 280Swanson, Debra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 20, 221Swanson, Debra L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 232Sweeney, Colin L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 220Sweeney, H. Lee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 433, 625Swiderski, Piotr . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 287Symens, Nathalie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 742Sysol, Justin R.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 135Sze, Daniel Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 214Szpirer, Cedric Y.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 740TTabata, Toshiaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 303, 634Tada, Yuji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 851Tadlock, Matthew V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 408Tafuro, Sabrina. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 140Tagalakis, Aristides . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 747Tagawa, Masatoshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 851Tahan, V.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139Tahara, Hideaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 34, 840Tai, April . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 689Tai, Ching-Lung. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 583Tai, Khalid . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 350Tak, Paul P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 231, 548, 870Tak, Paul-Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 66Takahashi, Atsushi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263, 899Takahashi, Masamichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 832Takahashi, Rei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 862Takahashi, Yoko E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 102Takahashi, Yuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 670, 862Takakura, Yoshinobu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 670, 862Takeda, Kiyoshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 480Takeda, Shin’ichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . 56, 151, 565, 582, 585Takeya, Yasushi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 884Takiya, Christina M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 440Talbot, Gill . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 158Tamai, Katsuto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 187, 628Tampier, Lutske . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 766Tamura, Masakazu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 513Tan, L. G.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 697Tan, M. H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 697Tan, Shuping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 376Tan, Wenjie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 78, 114, 172, 238Tang, Qiushi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 51, 53Tang, Ruhang. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 420Tang, Terence. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 218Tang, Yaoping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 677Tang, Ying . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 635Tang, Yucheng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 85Tangney, Mark . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 398, 705, 812, 848Tanguay, Robert M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 242Tani, Kenzaburo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 192, 263, 899Tani, Tomomi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 591Tannous, Bakhos A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 57Tansey, Malu G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 773Tapscott, Stephen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 553Tapscott, Stephen J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 623Tarantal, Alice F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 522Tarragona, Tony . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162Tateno, Chise . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 141Tatsumi, Kohei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 141, 903Tatsuzawa, Osamu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678Taube, Ran . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 645Tavernier, Geertrui. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 261Tayler, Matthew A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 191Taylor, Catherine A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 218EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 179Abstract Author IndexTaylor, Clare. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 32, 911Taylor, Justin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 659Telen, Marilyn J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 667Tellez, Joe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 857Tellez, Joseph. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 551Temme, Achim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 833Templeton, Nancy S. . . . . . . . . . . . . . . . . . . . . . . . . . 247, 564, 574, 586, 731ten Bloemendaal, Lysbeth . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 138Tenenbaum, Lilianne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 708Tennant, Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 880Terada, Nobuyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 500Terakura, Seitaro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 618Terao, Shuji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 216Tesarfreund, Matthew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 475Testa, Francesco . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59Teter, Mary A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 847Tey, Siok K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288Thaci, Bart. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 835Thakur, Garima. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 132Thatava, Tayaramma. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 633Theobald, Narda . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 354Thibault, Marc . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 396Thibodeau-Beganny, Stacey . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 95Thiel, Kristina W.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 662, 663Thiel, William H.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 662, 663Thomas, Darby L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 377, 571Thompson, Colin J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 254Thompson, Jill . . . . . . . . . . . . . . . . . 175, 481, 496, 606, 607, 610, 611, 653Thompson, John E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 218Thompson, Patrick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 767Thornton, Charles A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 49Thrasher, Adrian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 158, 617, 647Thrasher, Adrian J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 866, 229Throm, Robert E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 354, 861Thung, Swan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 38Thurlings, Rogier. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 548Tian, Jie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 542Tian, Jun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 358Tian, Runxia. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 603Tian, Wenhong. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 238, 376Tiboni, Francesca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 319, 523Tiemann, Katrin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 555Tikoo, Suresk K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 724Tilstra, Jeremy S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 629, 635Timares, Laura . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 379Tisdale, John F.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 352Tkachuk, Vsevolod A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 434Tobita, Kimimasa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 703Todo, Tomoki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 832, 839Todorow, Carlyn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 344, 676Toietta, Gabriele . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 898Toita, Nariaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678Tojyo, Masahiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 825Tol, Nikki. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 40, 502Toma, Shoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 826Tomasulo, Catherine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 600Tomizuka, Kazuma . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 236Tong, Alex W.. . . . . . . . . . . . . . . . . . 148, 196, 245, 248, 252, 292, 335, 564Tonne, Jason M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 599Torbett, Bruce E.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 348, 650Torrente, Yvan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 460, 464, 795Toscano, Miguel G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 859Toth, Karoly . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 489, 837Totterman, Thomas H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 176Toumadje, Araz . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 905Toutouna, Louiza . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 729Towers, Greg . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 647Townsend, Dewayne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 584Toyota, Hiroyasu. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 862Tran, Kaylie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 64Tran, Kaylie N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 169Tran, Rosa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 361Trani, Marianna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 430Transfiguracion, Julia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 361Tremblay, Jacques P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 462, 769, 784, 790Trevino, Isaac . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 773Tripic, Tamara. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 473, 799Trobridge, Grant D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 909Trochon-Joseph, Veronique . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 110, 113, 853Trueck, Christina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 693Trujillo, Miguel A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 80, 505Truong, Lynn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 798Trygg, Cynthia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314Tsai, Richard K.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 357Tschaika, Marina. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 189, 664, 822, 887Tschernia, Nick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 551Tschulena, Ulrich. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 339Tsokolaeva, Zoya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 434Tsokos, Maria. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 135Tsukahara, Tomonori . . . . . . . . . . . . . . . . . . . . . . . . . 207, 421, 428, 544, 554Tsuno, Takaya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 482Tsuzuki, Teruhisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 97Tuppurainen, Venla . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 212Turchetto, Lucia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276Turnis, Meghan E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 816Turrell, Susan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 215Tuschong, Laura M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 234, 524, 858Tuunanen, Pasi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159


180<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexTuve, Sebastian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 180Tyr, Elena. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445Tyr, Olena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 622Tysome, James. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 605Tzekov, Radouil . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 444UUchibori, Ryosuke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 207Uchida, Hiroaki. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 640Uchida, Naoya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 352Uchida, Yuji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 468Uchil, Pradeep . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 285Uchino, Makoto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 468, 788Uchio, Katsuhisa. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 468Uchiyama, Masayuki. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 97Uchiyama, Toru . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 227Ueda, Yasuji. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 824Uesugi, Motonari . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 670Uhrig, Silke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 5Uitto, Jouni . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 320Ukawa, Masami . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 743Ulasov, Ilya V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 191, 718, 835Ulkoski, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893Ulrich-Vinther, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 787Uludag, Hasan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 129, 562, 568, 755Umino, Yumiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17Ungari, Silvia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 313, 316Ungaro, Daniela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 313, 315Ungerechts, Guy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 833United Dystrophinopathy Project Consortium . . . . . . . . . . . . . . . . . . . . . . . . . 152Uno, Yoshitaka. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 259Unzu, Carmen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42Urabe, Masashi . . . . . . . . . . . . . . . . . . . . . . . . . . . . 207, 421, 428, 544, 554Urasawa, Nobuyuki. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 56Urata, Yasuo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 192Urban, Jon. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 402Urban, Nicole . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 265Urness, Myra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 325Urtti, Arto O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 750Usachenko, Yelena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 815Utoh, Rie. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 141, 419VVähäkangas, Elisa. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 765Völkel, Christine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284Vago, Luca. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295Vaha-Koskela, Markus . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39Valdmanis, Paul N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 54Valero, Valderez B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 440Vallanti, Giuliana. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225, 276, 613Valles-Ayoub, Yadira . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 772, 794Valley, Robert. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 328Valli, Emanuele . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258Valouev, Anton . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 14van Beusechem, Victor W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 40, 502van de Loo, Fons A. J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 559, 627van den Berg, Fiona . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 563van den Berg, Wim B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 559, 627van der Loo, Johannes C. M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 280van der Meulen-Muileman, Ida . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 40van Deutekom, Judith C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 153van Deventer, Sander . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 255Van Dusen, Nate. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 353van Logtenstein, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 249van Ommen, Gert-Jan B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 153Van Pijkeren, Jan P.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 398Van Poucke, Mario . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 875van Praag, Henriette . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 355van Rensburg, Ruan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 93van Rooijen, Nico . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 380, 723van Sinderen, Douwe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 848van Til, Niek P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 530, 617Van Vliet, Kim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58, 65Van Zwieten, Ruthger . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 533Vandenberghe, Luk H. . . . . . . . . . . . . . . . . . . . . . . . . . . . 1, 65, 368, 412, 759Vanier, Marie-Thérèse . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19Vanrell, Lucia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 485, 708Varghese, Steve . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 773Varki, Ajit . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 546Varney, Michelle L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 593Vasan, Sandhya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162Vasseur, Virginie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 552Vaughan, Andrew E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 623Vaughan, Erin E.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 424, 588Veenbergen, Sharon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 559Velazquez, Omaida C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 603Venditti, Charles P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 135, 679, 701Vera, Juan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 469Vera, Juan F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 28, 29, 672, 799Verhoeyen, Els . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 336Vermeij, Eline A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 627Vermeulen, Christie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 40, 502Verstegen, Monique M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 617Vervoordeldonk, Margriet J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 66, 231, 548Vetrini, Franesco. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 388Viale, Diego L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 609, 834Viapiano, Mariano . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 199Viarengo, G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 360EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 181Abstract Author IndexVickers, Selwyn M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 391, 497Viita, Helena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 512Vile, Richard. . . . . . . . . . . . . . . . . . . 175, 481, 496, 606, 607, 610, 611, 653Vile, Richard G.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 208Villa, Anna. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225, 617Villani, Edoardo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59Vincent, Justin M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 572, 575, 738Vinogradov, Serguei V.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 593Vinti, Maria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777Viola, Francesco . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59Viollet, Laurence. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155Virk, Mandeep S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 687Virtanen, Siru. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 145Visel, Meike. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 442Visigalli, Ilaria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 313, 315, 316Visser, Trudi P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 530, 617Vitetta, Ellen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288Vlasik, Tatyana. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 434Vleck, Jan P.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 856Vo, Thuy D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 98Vogel, Peter. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 525Vogler, Carole. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 768Voigtlander, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 385Voit, Thomas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 154Volpin, Monica . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 298Volz, Baerbel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 189, 822von Bergwelt-Baildon, Michael S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . 664, 887von Kalle, Christ<strong>of</strong>. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 286, 305Von Kalle, Christ<strong>of</strong>. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 345von Kalle, Christ<strong>of</strong>. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 347, 612, 833von Messling, Veronika . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 169VonKalle, Christ<strong>of</strong> . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297, 298Vorhies, John . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 564Voss, Tiffini . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 779Vosters, Jelle L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 870Voutetakis, Antonis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 143Vrettou, Christina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 880WWada, Jun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 167Waddington, S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223, 697Wade-Martins, Richard. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 602, 732Wadhwa, Renu. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 242, 841Wagemaker, Gerard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 530, 617Waisbren, Emily . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 237Waite, Katherine. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39Wakimoto, Hiroaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 608, 829Walker, Christopher M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149, 155,. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 290, 581Walker, William . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 685Wall, Cheryl . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155Wallraven, Gladice . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 292, 520Wan, Wen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36Wancewicz, Edward . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 557Wanders, Alkwin. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 176Wandless, Tom J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 350Wang, Adele Y.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 79Wang, Anqi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 171, 807, 808, 854Wang, Bing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 457, 635Wang, Cathy X. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 348Wang, Danher . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 504, 643Wang, Gang. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 238, 376Wang, Gary P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 13Wang, Hao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 453Wang, Hong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 515Wang, Hongjie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 415, 657, 843Wang, Hongxun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 195, 495Wang, Huan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 416Wang, Hui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 200Wang, James . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 341Wang, Jenny . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39Wang, Jianbin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11, 801Wang, Jun. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 422Wang, Li . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 433Wang, Lijun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 377, 571Wang, Lili . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 45, 416Wang, Minghui. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36, 503, 834Wang, Mingxing. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 890Wang, Nanya. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 510Wang, Pengju . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 605Wang, Pin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 686, 689Wang, Pin-Yi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 498Wang, Qiang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58Wang, Shengdian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 605Wang, Wei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 347Wang, Weijun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 200Wang, Wen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 172Wang, Wenjing. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 171, 806, 808Wang, Xinzhong. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 517Wang, Xiuyan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 32, 184, 911Wang, Yaohe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 605Wang, Yifeng. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 186Wang, Yong. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 753Wang, Yuan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 266Wang, Yue. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 694Wang, Yuhua . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 329Wang, Yun. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 709Wang, Zejing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 553, 623


182<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexWang, Zhaohui. . . . . . . . . . . . . . . . . . . . . . . . . . . . . 245, 247, 532, 564, 892Wang, Zhongya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 306, 681, 682Wanisch, Klaus. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 451Ward, Charles. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 372Wargo, Jennifer A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 190Warlich, Eva. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284Washington, Kareem N.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 352Watanabe, Yoshihiko. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 862Watcharanurak, Kanitta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 862Watts, Ashlee A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 912Watts, Korashon L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 631Watts, Victoria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155Weaver, Donald W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250Webb, Craig. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 487Weber, Christian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 305Weber, Christophe. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 432Weihrauch, Martin R.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 664, 887Weiland, Ola . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 289Weinberg, Marc S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 563Weiner, D. B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 648Weiner, David B.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 103, 115, 327, 477Weinfeld, Michael. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 568Weiss, Robert B.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 152Wenzel, Daniela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 693Werfel, Stanislas M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 241Wesa, Amy K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 271Westenberg, Marcel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 716Wheeler, Thurman M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 49White, Edward A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 394Whitehouse, Adrian. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 215Whitley, Chester B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 20Wibowo, Mia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 181Wicke, Daniel C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 318Wielgosz, Matthew M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 733Wilber, Andrew. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 338, 339Wilen, Craig B.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 801Wilkinson, Leslie. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893Willenbring, Holger . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 910Willerth, Stephanie M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 802Williams, Bradley A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 796Williams, James A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 166, 572, 575, 738Williams, Jon P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 201Williams, Kyha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 142Willmon, Candice L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 611Wilson, Aisha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 412Wilson, Carolyn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 649Wilson, James M. . . . . . . . . . . . 1, 45, 65, 164, 368, 412, 416, 543, 625, 759Wilson, John . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 672Wilson, Kyle A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 273Wilson, Matthew H. . . . . . . . . . . . . . . . . . . . . . . . . . . . 12, 15, 179, 472, 813Wilson, W. H.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31Wilton, Steve D.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 789Wimley, William . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314, 411Wimmer, Eckard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 372Windebank, Anthony J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 780Winnik, Francoise M.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 756Winterton, Matthew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 659Wipf, Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 157Wirth, Thomas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 212, 512Wittig, Burghardt . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 189, 664, 822, 887Witting, Scott R.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 52, 675Wohlgensinger, Vital . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 233, 869Wolchok, Jedd D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 817Wold, William . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 196, 496Wold, William S. M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 489, 837Wolf, Daniel A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 20Wolf, Gernot . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 726Wolfe, John H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 317, 901Wolff, Jon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 160Wolff, Thomas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 430, 597Wong, Fay E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 645Wong, Kamehameha K.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 3Wonganan, Piyanuch. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 435Wongsrikeao, Pimprapar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 804Wongthida, Phonphimon . . . . . . . . . . . . . . . . . . . . . . . . . . 175, 481, 607, 610Woo, Savio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 38, 277Wood, Josh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 857Woodward, Josha A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 692Worgall, Stefan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 161, 452, 752Worgall, Stefen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 163Worley, Kim C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 574Wozniak, David F.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 768Wright, J. F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 308Wright, J. Fraser. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 614Writer, Michele J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 394Wu, Bo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 890Wu, Chih-Hsien. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 882Wu, Hao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 84Wu, Lily. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 714, 850Wu, Ping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 390Wu, Robert A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 864Wu, Shourong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 891Wu, Susan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 598Wu, Xiaobing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 78, 238, 376Wu, Yihan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 839Wunder, Stephanie L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 757Wurdinger, Thomas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 665EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 183Abstract Author IndexXXagorari, Angeliki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137Xia, Danny. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 98Xia, Xuexing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 737Xiao, Bin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 683Xiao, Ping-Jie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 60Xiao, Ru . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 1, 65, 368Xiao, Weidong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 310Xiao, Xiao . . . . . . . . . . . . . . . . . . . . . . . . . . 6, 160, 420, 457, 581, 683, 790Xiao, Yan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 332Xie, Boxun. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 38Xie, Jun. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58, 362, 450, 671Xie, Qing. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58, 362, 450Xiong, Weidong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26Xiong, Zhengming. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 826Xiuying, Liu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 663Xu, Acong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 467Xu, Xiabin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 423Xu, Yaqin. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 163Xu, Zhili . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 542Xu, Zuoshang. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 450Xue, Shao-An . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 349YYáñez-Muñoz, Rafael J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 451Yadav, Swati . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 160Yagi, Hiroya. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 421, 544, 554Yagiz, Kader . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26, 181, 475Yaguchi, Tomoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 841Yamada, Masafumi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678Yamada, Meiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 192Yamada, Yuma. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 748Yamaguchi, Saori . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 899Yamaguchi, Tomoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 537Yamamoto, Masato. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 80, 391, 497Yamamoto, Motoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 410Yamamoto, Sakura . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 167Yamamoto, Seiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 680Yamaoka, Norie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 500Yamasaki, Satoshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 391Yamashita, Atsushi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 401Yamato, Masayuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 419Yamazaki, Takehiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 187, 628Yamazoe, Sayumi. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 670Yamoshita, Satoshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 468Yan, J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 648Yan, Jian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 115, 327, 477Yan, Ziying . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 4, 620Yanagisawa, Teruyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 508Yang, Bin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 450Yang, James C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 25Yang, Lin. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 6Yang, Ningning. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 741Yang, Shan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 851Yang, Sherry W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 507Yang, Shicheng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 476, 478Yang, Wendy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 557Yang, Xiao. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 69, 885Yang, Xiao Yi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 47Yang, Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 913Yang, Ye . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 130Yanik, Tulin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 143Yannaki, Evangelia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137Yao, Feng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 237Yaouanc, Jean Jacques . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 745Yardeni, Tal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 144Yarnall, Charles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156Yasukawa, Masaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 652Yasumura, Eduardo G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 440Yawata, Toshio. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 513Ye, Bin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 218Ye, Delia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 165Ye, Guo-jie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 377, 571Ye, L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 913Ye, Peiqing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 535, 550Ye, Yihong. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 876Yeagy, Brian A.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 408Yeh, Raymond . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 32, 815Yehling, Eric. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 23Yen, Benjamin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 535Yen, Tzu-Chen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 583Yeung, Jonathan C.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 872Yi, Yaling. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 620Yin, Xiao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 78, 114Yip, Ping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 451Ylä-Herttuala, Seppo . . . . . . . . . . . . . . . . . . . . . 159, 209, 212, 512, 669, 765Ylaya, Kris . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 659Yockman, James W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 86, 126Yokota, Takanori. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 259Yokota, Toshifumi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 56, 565Yonemitsu, Yoshikazu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 824Yong, Liu. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 819Yongabi, Felicitas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159Yoo, Ji Young . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 198, 811, 841Yoo, Ji-Young . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 836Yoon, A-Rum . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 84, 198, 720Yoon, DoHeum. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 260Yoon, Sang Soon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 406


184<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract Author IndexYoshida, Kie. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 259Yoshihara, Chieko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 825Yoshikawa, Hiroyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 554Yoshimura, Toshiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 585Yoshizato, Katsutoshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 141You, Chang Xuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 819You, Chia Chin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 173Younes, Anas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 473Yu, Di . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 501, 828Yu, Gang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 105Yu, Hongwei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 412, 759Yu, Jimin. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 411Yu, Julie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 299Yu, Seungshin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818Yu, William . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 713Yu, Yang . . . . . . . . . . . . . . . . . . . . . . . . . 248, 275, 292, 335, 487, 532, 564Yu, Zhiya. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 190, 478Yuan, C. M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31Yuan, Jinyun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 650Yuan, Zhenhua. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 6, 683Yue, Yongping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 150, 466, 587, 601Yuen, Carrie. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 809Yu-Feng, Lin. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288Yugeta, Naoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 582Yui, Nobuhiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 401Yumul, Roma . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 180, 264, 843, 849, 863Yun, Chae-Ok . . . . . . . . . . . . 84, 86, 188, 198, 202, 242, 516, 720, 811, 841Yun-shan, Tan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 73Yurek, David M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 776Yusa, Kosuke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 15Yvon, Eric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288ZZacchigna, Serena. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 423, 431Zahid, Maliha. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 7Zaitseva, Marina A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 735Zamore, Phillip D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 362Zaoui, Karim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 305Zavaljevski, Maja . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 730Zeigerman, Haim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 719Zenadi, Rahima . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 667Zeng, Jinfeng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 807Zeng, Lepeng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 402Zeng, Yué . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 463Zeng, Zhao-Chong. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 211Zentilin, Lorena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 311, 423, 431Zerah, Michel. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19Zerfas, Patricia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 135Zervou, Fani. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137Zhan, Jun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 733Zhang, Dazhi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 819Zhang, Fang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 617Zhang, Feijie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 306Zhang, Guisheng. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 322Zhang, Hongwei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 362, 450, 593Zhang, Jane. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 287, 661Zhang, L.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 178Zhang, Lin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 775Zhang, Ling . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 219, 476, 478, 808, 854Zhang, Ping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 47Zhang, Shuyuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 274, 803Zhang, Song-Nan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 202, 811Zhang, Ting . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 493Zhang, Wei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 574, 876Zhang, Wenli . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 92, 636Zhang, Xiaoling . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 756Zhang, Xiao-Xiang. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 392Zhang, Xichen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 515Zhang, Xiongwen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 217Zhang, Yadong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 876Zhang, Yanling . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 219Zhang, Yonggong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 251Zhang, Yonghui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 694Zhang, Zheng-Yu. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 211Zhao, Hui Fen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 867Zhao, Huifen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 524, 525Zhao, Ling . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 789Zhao, Lu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 647Zhao, Tongmao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 482Zhao-Chong, Zeng. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 73Zheng, Changyu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 77, 143, 761Zheng, Heng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 580Zheng, Xin. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 808Zheng, Zhili . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 476, 478Zheng-Yu, Zhang. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 73Zhong, Li. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58, 61, 363, 369, 370, 700Zhong-shan, Liu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 73Zhou, Dongming. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 389Zhou, Jiehua . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 287, 555Zhou, Jingmin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 69, 673Zhou, Liqin. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 15Zhou, Shangzhen . . . . . . . . . . . . . . . . . . . . . . . . . . . . 43, 546, 615, 630, 885Zhou, Sheng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 222, 860Zhou, Tian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 108Zhou, Xiangyang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 389Zhou, Xianzheng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 868Zhou, Xiaoou . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 656Zhu, Liwei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 186, 566EXHIBITOR PROSPECTUSfinal program


13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 185Abstract Author IndexZhu, Quan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 645Zhu, Wen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 163Zhu, Zeng B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 834Zhuge, Ying . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 603Zidovska, Alexandra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 393Ziliotto, Raul . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276Zimmermann, Katrin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 693Zinn, Kurt . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36Zolopa, Andrew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 646Zolotukhin, Irene . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 763Zolotukhin, Sergei. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 61, 371, 378Zoltick, Philip W.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 320, 344, 357, 676Zoon, Kathryn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 482Zou, Jie. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 130Zou, Jizhong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 220Zoumbos, Nikolas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 340, 729Zourelias, Lee. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 104Zubair, Adnan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 689Zuhaili, Baraa. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 237Zurita, Juan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 185Zweier-Renn, Lynnsey A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 342, 690


186<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>NotesEXHIBITOR PROSPECTUSfinal program


ASGCT-1209-226Mark Your Calendars!<strong>American</strong> Socie t y <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>14 th Annual MeetingS e at t l e , WA | May 18-22, 2 011Exciting scientific session topicswill include:- Stem <strong>Cell</strong> Therapeutics- Oligonucleotide Therapies- Novel Vector Development- Mechanisms <strong>of</strong> Vector Transduction- Host-Vector Interactions- Clinical Trial Advancements

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