FINAL PROGR AM - American Society of Gene & Cell Therapy

AMERICAN SOCIETY OF GENE THERAPY 

8th Annual Meeting 

FINAL PROGRAM 

Jointly sponsored by Stanford University School of Medicine, Stanford, California 

June 1-5, 2005 

America’s Center 

St. Louis, Missouri

AMERICAN SOCIETY OF GENE THERAPY • Final Program 1 

TABLE OF CONTENTS 

Welcome.................................................................................................................................................................... 2 

Committee Meetings and Committee Rosters ...................................................................................................... 3 

Annual Meeting Supporters .................................................................................................................................... 9 

Annual Meeting Exhibitors.................................................................................................................................... 10 

Abstract Reviewers ................................................................................................................................................. 11 

Planning Committee and Group Coordinating Reviewers ................................................................................ 12 

Research Awards and Travel Grant Recipients ..................................................................................................... 13 

Faculty Listing ......................................................................................................................................................... 14 

General Meeting Information ............................................................................................................................... 20 

Program-at-a-Glance ............................................................................................................................................. 24 

Scientific Program Schedule .................................................................................................................................. 25 

America’s Center Floor Plans ................................................................................................................................ 42 

Housing Information and Map of St. Louis ......................................................................................................... 44 

Abstract Directory .................................................................................................................................................. 46 

Abstract Author Index .......................................................................................................................................... 116 

Exhibit Hall Floor Plan ........................................................................................................................................ 146 

Exhibitor Information .......................................................................................................................................... 147 

ASGT EXECUTIVE OFFICE 

555 East Wells Street 

Suite 1100 

Milwaukee, WI 53202-3823 

Phone: (414) 278-1341 

Fax: (414) 276-3349 

E-mail: info@asgt.org 

Internet: www.asgt.org

2 

8th Annual Meeting • June 1 - 5, 2005 • St. Louis 

Dear Colleagues, 

On behalf of the American Society of Gene Therapy, welcome to the 

ASGT 8 th Annual Meeting. We are very pleased to present this year’s 

program featuring the George Stamatoyannopoulos lecture by 

Thomas Tuschl, PhD, Mechanisms of siRNA- and miRNA-guided Gene 

Regulation in Mammals; the Presidential Symposium presentation by 

George Q. Daley, MD, PhD, Combining Gene Therapy with Cell 

Therapy; 18 Education Sessions; 16 Scientific Symposia; 17 Workshops; oral and 

poster sessions chosen from submitted abstracts and Meet-the-Investigator 

lunch sessions. This year, in response to feedback from previous years, the 

Young Investigators’ Symposium has been scheduled as a general session. 

The Society’s committees have done a great deal of hard work in putting 

together this superb program and for that, I thank them. Topics that will be 

covered at this year’s meeting include: Cancer Vaccines, Pre-Clinical Issues in 

Gene Therapy, Stem Cell Gene Therapy, Gene Therapy and Behavior, Skin 

Gene Therapy, Gene Therapy for HIV, Ethical Issues in Clinical Trial Design, 

Nonviral Gene Delivery, and Regenerative Approaches for Skeletal Disorders. 

Many other sessions cover the diverse and exciting developments in the field 

over the past year. 

I would like to thank our exhibitors and industry supporters for their 

participation and generosity, in addition to our exceptional faculty who kindly 

share their knowledge, expertise and time. 

The ASGT Annual Meeting provides an outstanding forum for sharing the 

latest developments in the field of gene therapy and networking with our 

colleagues. I welcome you to St. Louis. 

Sincerely, 

Katherine A. High, MD 

President, ASGT 

ASGT OFFICERS & DIRECTORS 

OFFICERS 

President 


The Children’s Hospital of Philadelphia 

Incoming President 

Mark A. Kay, MD, PhD 

Stanford University School of Medicine 

Vice President 

Theodore Friedmann, MD 

University of California San Diego School of Medicine 

Secretary (06) 

Kenneth Cornetta, MD 

Indiana University 

Treasurer (05) 

Xandra Breakefield, PhD 

Massachusetts General Hospital 

ASGT EXECUTIVE 

OFFICE 

555 East Wells Street 

Suite 1100 

Milwaukee, WI 53202 

Phone: 414-278-1341 

Fax: 414-276-3349 

Email: info@asgt.org 

www.asgt.org 

Executive Director 

Elizabeth Dooley, CAE, CMP 

Director of Meetings & 

Communications 

Lynn Beyer 

Manager, Member Services 

Barbie Brennan 

Program Manager 

Ken Janowski 

Meetings & Exhibits Manager 

Nadine Kinnunen 

Additional On-Site Staff 

John Gardner 

Kelly Hughes 

Paul Rushizky 

Consulting Partner 

Kay Whalen, CAE 

BOARD OF DIRECTORS 

Jean Bennett, MD, PhD (07) 

Cynthia Dunbar, MD (05) 

John F. Engelhardt, PhD (06) 

Helen E. Heslop, MD (05) 

Nicholas Muzyczka, PhD (06) 

Paul D. Robbins, PhD (07) 

Michel Sadelain, MD, PhD (07) 

R. Jude Samulski, PhD (06) 

Jon Wolff, MD (05) 

Joseph C. Glorioso, PhD (05) 

Chair, Advisory Board 

(ex-officio) 

David A. Williams, MD 

Editor-in-Chief, Molecular Therapy 

(ex-officio)


COMMITTEE MEETINGS 

Committee terms technically commence and conclude in June at the Annual Meeting. Incoming 

Chair and committee member terms officially go into effect on June 3, 2005 at the Business 

Meeting. 

WEDNESDAY, JUNE 1 

7:00 pm - 8:15 pm 2006 Planning Committee Meeting 

THURSDAY, JUNE 2 

12:45 pm - 1:45 pm Publications Committee 

12:45 pm - 1:45 pm Ethics Committee 

12:45 pm - 1:45 pm Education 

4:30 pm - 5:30 pm Molecular Therapy Editorial Board 

FRIDAY, JUNE 3 

7:00 am - 8:00 am Industrial Liaison Committee Meeting 

7:00 am - 8:00 am Immunology of Gene Therapy 

7:00 am - 8:00 am Oligonucleotide Based Therapies 

12:30 pm - 1:45 pm Clinical & Regulatory Affairs Committee 

12:30 pm - 1:30 pm Genetic Diseases 

12:30 pm - 1:30 pm Cancer Gene Therapy 

12:30 pm - 1:30 pm Nonviral Gene Transfer Vectors 

12:30 pm - 1:30 pm Hemopoietic Cell Gene Therapy 

12:30 pm - 1:30 pm Embryonic Stem Cells & Tissue Engineering 

12:30 pm - 1:30 pm Neural Disorders Gene Therapy 

12:30 pm - 1:30 pm Infectious Disease 

SATURDAY, JUNE 4 

12:15 pm - 1:30 pm Advisory Board Meeting 

12:30 pm - 1:30 pm Membership Committee 

SOCIETY ANNUAL BUSINESS MEETING 

The ASGT Business Meeting will take place in Room 130 on Friday, June 3, from 4:30 to 5:30 pm. 

All ASGT members in good standing are welcome to attend. Election results will be announced, 

new officers will be installed and the Society’s finances and membership statistics will be reviewed. 

We welcome your input and participation.

4 


COMMITTEE ROSTER (As of April 27, 2005) 

Terms begin and end at the June 8 th Annual Business Meeting (through June 2005) 

STANDING COMMITTEES 

ADVISORY BOARD 

Chair 

Joseph C. Glorioso, PhD (2004 – 2005) 

Members 

W. French Anderson, MD (2004 – 2009) 

David Bodine, PhD (2003 – 2008) 

Malcolm K. Brenner, MD, PhD (2002 – 2007) 

Donald B. Kohn, MD (2004 – 2009) 

Richard C. Mulligan, PhD (2001 – 2006) 

Alan E. Smith, PhD (2002 – 2007) 

George Stamatoyannopoulos, MD (2004 – 2005) 

Inder Verma, PhD (2001 – 2006) 

Savio L.C. Woo, PhD (2000 – 2005) 

INTERNATIONAL 

Chair 

Bernd Gansbacher, PhD (2004 – 2005) 

Members 

Fatima Bosch, PhD (2002 – 2005) 

Odile Cohen-Haguenauer, MD, PhD (2004 – 2005) 

Olivier Danos, PhD (2005 – 2008) 

George Dickson, PhD (2005 – 2008) 

Yoshikatsu Eto, MD, PhD (2005 – 2008) 

David Klatzmann, MD, PhD (2003 – 2006) 

Philippe Moullier, PhD (2004 – 2007) 

Keiya Ozawa, MD, PhD (2003 – 2006) 

Jesus Prieto, MD, PhD (2005 – 2008) 

John Rasko, MD, PhD (2004 - 2007) 

Seppo Yla-Herttuala, MD, PhD (2005 – 2008) 

CLINICAL AND REGULATORY AFFAIRS 

Chair 

Daniel R. Salomon, MD (2004 - 2006) 

Members 

Estuardo Aguilar-Cordova, PhD (1998 - 2005) 

Dale Ando, MD (2002 - 2008) 

Arthur L. Beaudet, MD (2004 - 2007) 

Timothy Billiar, MD (2002 - 2005) 

Barry J. Byrne, MD, PhD (2005 - 2008) 

Joy Cavagnaro, PhD, DABT, RAC (2005 - 2008) 

Kenneth Cornetta, MD (2005 - 2008) 

Linda Couto, PhD (2005 – 2008) 

Terence R. Flotte, MD (2004 - 2005) 

Carl H. June, MD (2004 - 2007) 

Robert W. Martin (2003 - 2006) 

R. Scott McIvor, PhD (2002 - 2005) 

Andra Miller, PhD (2002 - 2008) 

Glenn F. Pierce, MD, PhD (2004 - 2007) 

Greg Podsakoff, MD (2003 - 2006) 

Stephanie Simek, PhD (2003 - 2006) 

Kenneth Weinberg, MD (2005 - 2008) 

EDUCATION 

Chair 

Matthew Weitzman, PhD (2003 - 2005) 

Co-Chairs 

Philip L. Leopold, PhD (2005 - 2006) 


Members 

Estuardo Aguilar-Cordova, PhD (2004 - 2007) 

Michael A. Barry, PhD (2005 - 2008) 

Roberto Cattaneo, PhD (2005 - 2008) 


Olivier Danos, PhD (2004 - 2007) 

Beverly L. Davidson, PhD (2003 - 2006) 

Patrick Kelly, MD (2002 - 2005) 

Donald B. Kohn, MD (2005 - 2008) 

Philip L. Leopold, PhD (2004 - 2007) 

Fulvio Mavilio, PhD, Prof (2004 - 2007) 

Katherine Ponder, MD (2002 - 2005) 

ETHICS 

Chair 

R. Scott McIvor, PhD (2004 – 2006) 

Members 

Jean Bennett, MD, PhD (2002 - 2005) 

Arthur Caplan, PhD (2005 - 2008) 

Lawrence C. Chan, MD (2004 - 2007) 

Cynthia Dunbar, MD (2004 - 2007) 

Theodore Friedmann, MD (2004 - 2005) 

Henry T. Greely, JD (2005 - 2008) 

Dieter Gruenert, PhD (2003 - 2006) 

Jonathan Kimmelman, PhD (2005 - 2008) 

Margaret A. Liu, MD (2002 - 2005) 

Punam Malik, MD (2004 - 2007) 

Seppo Yla-Herttuala, MD, PhD (2002 – 2005) 

For a complete schedule of 

Committee Meetings, see page 3


COMMITTEE ROSTER 

INDUSTRIAL LIAISON 

Chair 

Parris Burd, PhD (2004 – 2005) 

Douglas J. Jolly, PhD (2005 – 2006) 

Members 

Gwen Binder, PhD (2005 - 2008) 

Alan R. Brooks, PhD (2005 - 2008) 

Joy Cavagnaro, PhD, DABT, RAC (2002 - 2005) 

Kenneth Chahine, PhD (2004 - 2007) 

Stephen M.W. Chang, PhD (2004 - 2006) 

Sara M. Cunningham (2005 - 2008) 

Boro Dropulic, PhD (2003 - 2006) 

Mitchell Finer, PhD (2001 - 2007) 

Anthony Green, PhD (2003 - 2006) 

Douglas J. Jolly, PhD (2003 - 2006) 

C. Richter King, PhD (2002 - 2005) 

Edward Lanphier (2004 - 2007) 

Andy Lin, PhD (2005 - 2008) 

Russette M. Lyons, PhD (2002 - 2005) 

Gerard McGarrity, PhD (2005 - 2008) 

Ralph W. Paul, PhD (2004 - 2007) 

Leonard E. Post, PhD (2000 - 2006) 

Alain Rolland, PharmD, PhD (1999 - 2006) 

Samuel C. Wadsworth, PhD (2004 - 2007) 

Peter K. Working, PhD, DABT (2002 - 2005) 

MEMBERSHIP 

Chair 

Paul D. Robbins, PhD (2003 – 2006) 

Members 

C. Anthony Blau, MD (2004 - 2007) 

Seng H. Cheng, PhD (2004 - 2007) 

J. Victor Garcia-Martinez, PhD (2003 - 2006) 

Mark A. Kay, MD, PhD (2002 - 2005) 

Qiang Liu, PhD (2003 - 2006) 

Isabelle Riviere, PhD (2004 - 2007) 

Savio L.C. Woo, PhD (2005 - 2008) 

NOMINATING 

Chair 

George Stamatoyannopoulos, MD (2004 - 2005) 

Members 

Valder Arruda, MD, PhD (2004 - 2005) 

Harry L. Malech, MD (2004 - 2005) 

Punam Malik, MD (2004 - 2005) 

Bruce Sullenger, PhD (2004 - 2005) 

PROGRAM 

Chair 

Arthur W. Nienhuis, MD (2004 - 2005) 

Co-Chairs 


Michel Sadelain, MD, PhD (2004 - 2006) 

Members 

Michele P. Calos, PhD (2003 - 2006) 

Barrie J. Carter, PhD (2001 - 2006) 


Philip R. Johnson, MD (2004 - 2007) 

Luigi Naldini, MD, PhD (2004 - 2007) 

Stephen J. Russell, MD, PhD (2002 - 2005) 


Joseph Zabner, MD (2004 - 2007) 

PUBLICATIONS 

Chair 

George Stamatoyannopoulos, MD (2004 - 2006) 

Members 

W. French Anderson, MD (2003 - 2007) 

David Bodine, PhD (2004 - 2007) 

Barrie J. Carter, PhD (2003 - 2006) 

Jeffrey Chamberlain, PhD (2004 - 2005) 

Joseph C. Glorioso, PhD (2005 - 2008) 

Katherine A. High, MD (2003 - 2006) 


Brendan Lee, MD PhD (2004 - 2007) 

Pedro Lowenstein, MD, PhD (2002 - 2005) 

James M. Wilson, MD, PhD (2005 - 2008) 

SCIENTIFIC COMMITEES 

CANCER GENE THERAPY 

Chair 

Helen E. Heslop, MD (2004 - 2006) 

Members 

Steven M. Albelda, MD (2004 - 2007) 

Laurence JN Cooper, MD, PhD (2004 - 2007) 

James S. Economou, MD, PhD (2003 - 2006) 

David Ennist, PhD (2003 - 2006) 

Paul B. Fisher, MPH, PhD (2005 - 2008) 

Kristen Hege, MD (2003 - 2006) 

Dennis D. Hickstein, MD (2003 - 2006) 

Michael Jensen, MD (2003 - 2006) 

Andre Lieber, MD, PhD (2005 - 2008) 

John C. Morris, MD (2003 - 2006) 

John J. Nemunaitis, MD (2003 - 2006) 

James S. Norris, PhD (2005 - 2008) 

Richard G. Vile, PhD (2004 - 2007) 

Richard J. Whitley, MD (2002 - 2005) 

Masato Yamamoto, MD, PhD (2004 - 2007) 

James Zwiebel, MD (2003 - 2006)

6 



CARDIOVASCULAR GENE THERAPY 

Chair 

Anthony Rosenzweig, MD (2004 - 2006) 

Members 

Andrew H. Baker, PhD (2004 - 2007) 

Timothy Billiar, MD (2002 - 2005) 

Lawrence C. Chan, MD (2004 - 2005) 

Ken Chien, MD, PhD (2005 - 2008) 

David A. Dichek, MD (2004 - 2007) 

J. Kevin Donahue, MD (2004 - 2007) 

Victor Dzau, MD (2003 - 2006) 

Arthur Feldman, MD, PhD (2002 - 2005) 

Roger J. Hajjar, MD (2004 - 2007) 

Ralph Kelly, MD (2003 - 2006) 

Douglas Losordo, MD (2003 - 2006) 

Sonia I. Skarlatos, PhD (2004 - 2007) 

Edith Tzeng, MD (2005 - 2008) 

EMBRYONIC STEM CELLS & TISSUE 

ENGINEERING 

Chair 

Gordon Keller, PhD (2003 - 2005) 

Mervin Yoder, MD (2005 - 2006) 

Members 

Mark E. Davis, PhD (2003 - 2005) 

Ira J. Fox, MD (2005 - 2008) 

Stanton L. Gerson, MD (2003 - 2006) 

Linda G. Griffith, PhD (2005 - 2008) 

Carolyn Lutzko, PhD (2004 - 2007) 

Darwin J. Prockop, MD, PhD (2004 - 2007) 

Mervin Yoder, MD (2003 - 2006) 

GENE THERAPY OF GENETIC DISEASES 

Co-Chairs 

Jean Bennett, MD, PhD (2003 - 2005) 


Members 

Seng H. Cheng, PhD (2004 - 2007) 

Markus Grompe, MD (2003 - 2006) 

Eric Hoffman, PhD (2002 - 2005) 

Philippe Leboulch, PhD (2003 - 2006) 

Brendan Lee, MD, PhD (2004 - 2007) 


Judith St. George, PhD (2002 - 2005) 

Joseph Zabner, MD (2004 - 2007) 

GENETIC VACCINES 

Chair 


Carl H. June, MD (2005 - 2006) 

Members 

K. Reed Clark, PhD (2004 - 2007) 

Stephen L. Eck, MD, PhD (2003 - 2006) 


Carl H. June, MD (2004 - 2007) 

Art Krieg, MD (2003 - 2006) 

David Schowalter, MD, PhD (2005 - 2008) 

Patrick Squiban, MD (2003 - 2006) 

Thierry C. Vandendriessche, PhD (2005 - 2008) 

Britta Elisabeth Wahren, MD, PhD (2003 - 2006) 

David B. Weiner, PhD (2003 - 2006) 

Ning-Sun Yang, PhD (2003 - 2006) 

HEMOPOIETIC CELL GENE THERAPY 

Chair 

Jan Nolta, PhD (2004 – 2006) 

Members 

David Bodine, PhD (2000 - 2006) 

Fabio Candotti, MD (2004 - 2007) 

Gay Crooks, MD (2002 – 2008) 

Mary Dinauer, MD, PhD (2003 - 2006) 

Robert G. Hawley, PhD (2004 - 2007) 

Hans-Peter Kiem, MD (2003 - 2006) 


John J. Rossi, PhD (2005 - 2008) 

Alice F. Tarantal, PhD (2005 - 2008) 

Christof von Kalle, MD (2004 - 2007) 

Christopher Walsh, MD, PhD (2003 - 2006) 

Kenneth Weinberg, MD (2005 - 2008) 

IMMUNOLOGY OF GENE THERAPY 

Chair 

Hildegund CJ Ertl, MD (2004 - 2006) 

Members 

Bruce R. Blazar, MD (2003 - 2006) 

Jonathan S. Bromberg, MD, PhD (2003 - 2006) 

Karin Gaensler, MD (2003 - 2006) 

Roland W. Herzog, PhD (2004 - 2007) 

Michael Jensen, MD (2003 - 2006) 

Karin Jooss, PhD (2003 - 2006) 

Hans-Peter Kiem, MD (2003 - 2006) 

Stanley Riddell, MD (2003 - 2006) 

Isabelle Riviere, PhD (2003 - 2006) 

Maria Grazia Roncarolo, MD (2004 - 2007) 

Michel Sadelain, MD, PhD (2003 - 2006)



INFECTIOUS DISEASE 

Chair 


John A. Zaia, MD (2005 - 2006) 

Members 

Ramesh Akkina, DVM, PhD (2002 - 2005) 


J. Victor Garcia-Martinez, PhD (2004 - 2007) 

David Klatzmann, MD, PhD (2004 - 2007) 

Alexander Kolykhalov, PhD (2005 - 2008) 

Bruce L. Levine, PhD (2004 - 2007) 

Margaret A. Liu, MD (2002 - 2005) 

Roger J. Pomerantz, MD, FACP (1998 - 2005) 

John J. Rossi, PhD (2004 - 2005) 

David Strayer, MD, PhD (2003 - 2006) 

Didier Trono, MD (2004 - 2007) 

John A. Zaia, MD (2002 - 2006) 

MUSCULO-SKELETAL DISORDER GENE 

THERAPY 

Chair 

Jeffrey Chamberlain, PhD (2003 - 2006) 

Members 

Olivier Danos, PhD (2001 - 2005) 

George Dickson, PhD (2004 - 2007) 

Dongsheng Duan, PhD (2004 - 2007) 

Dan Gazit, DMD, PhD (2004 - 2007) 

Steven C. Ghivizzani, PhD (2004 - 2007) 

Johnny Huard, PhD (2004 - 2007) 

Jay R. Lieberman, MD (2003 - 2006) 

Terence A. Partridge, PhD (2004 - 2007) 

Paul D. Robbins, PhD (2003 - 2005) 

Michael Themis, PhD (2004 - 2007) 

Jon Wolff, MD (2001 - 2007) 

Xiao Xiao, PhD (2005 - 2008) 

NEURAL DISORDERS GENE THERAPY 

Chair 

Beverly L. Davidson, PhD (2005 - 2006) 

Pedro Lowenstein, MD, PhD (2003 - 2005) 

Members 

Krys Bankiewicz, MD, PhD (2004 - 2007) 

Xandra Breakefield, PhD (2004 - 2007) 

E. Antonio Chiocca, MD, PhD (2003 - 2006) 

Nicole Deglon, PhD (2004 - 2007) 

David J. Fink, MD (2002 - 2005) 

John Forsayeth, PhD (2004 - 2007) 

Sanjiv Gambhir, MD, PhD (2003 - 2006) 

Michael Kaplitt, MD, PhD (2003 - 2006) 

George Karpati, MD (2003 - 2006) 

Ronald Mandel, PhD (2004 - 2007) 

Daniel A. Muruve, MD (2003 - 2006) 

Edward Schuchman, PhD (2003 - 2006) 

H. David Shine, PhD (2003 - 2006) 

James B. Uney, PhD (2003 - 2006) 

John H. Wolfe, VMD, PhD (2004 - 2007) 

David C. Yeomans, PhD (2005 - 2008) 

NONVIRAL GENE TRANSFER VECTORS 

Chair 

Dexi Liu, PhD (2004 - 2006) 

Members 

Michael A. Barry, PhD (2004 - 2007) 

Michele P. Calos, PhD (2004 - 2007) 

Esther H. Chang, PhD (2003 - 2006) 

Mark E. Davis, PhD (2005 - 2008) 

Pamela Davis, MD, PhD (2004 - 2007) 

Richard Heller, PhD (2002 - 2005) 

Leaf Huang, PhD (2002 - 2005) 

Alexander V. Kabanov, PhD (2003 - 2006) 

Sung Wan Kim, PhD (2002 - 2005) 

Kam W. Leong, PhD (2004 - 2007) 

Ian MacLachlan, PhD (2004 - 2007) 

Lluis M. Mir, DSc (2003 - 2006) 

Daniel Scherman, PhD (2004 - 2007) 

Francis C. Szoka, Jr., PhD (2004 - 2007)

8 



RESPIRATORY TRACT GENE THERAPY 

Chair 

Paul B. McCray, Jr., MD (2005 - 2006) 

Daniel Weiss, MD, PhD (2003 - 2005) 

Members 

Eric W.F.W. Alton, MD, FRCP (2005 - 2008) 

Raj Batra, MD (2001 - 2006) 

Bruce Bunnell, PhD (2004 - 2007) 

Pamela Davis, MD, PhD (2005 - 2008) 

David A. Dean, PhD (2004 - 2007) 

John F. Engelhardt, PhD (2005 - 2008) 

Jim Hu, PhD (2004 - 2007) 

Stephen Hyde, PhD (2004 - 2007) 

Larry G. Johnson, MD (2005 - 2008) 

Robert Kaner, MD (2001 - 2005) 

Jay K. Kolls, MD (2001 - 2006) 

Paul B. McCray, Jr., MD (2003 - 2006) 

Alistair Ramsay, PhD (2003 - 2006) 

Guoshun Wang, DVM, PhD (2004 - 2007) 

Daniel Weiss, MD, PhD (2001 - 2006) 

Pamela L. Zeitlin, MD, PhD (2003 - 2006) 

VIRAL GENE TRANSFER VECTORS 

Chair 


Brian P. Sorrentino, MD (2005 - 2006) 

Members 

Douglas Brough, PhD (2002 - 2005) 

Barry J. Byrne, MD, PhD (2004 - 2007) 

Jay A. Chiorini, MD (2003 - 2006) 

Peter Colosi, PhD (2004 - 2007) 


Francois-Loic Cosset (2005 - 2008) 

Jeffrey A. Engler, PhD (2005 - 2008) 

Howard J. Federoff, MD, PhD (2004 - 2007) 

Michael J. Imperiale, PhD (2004 - 2005) 

Noriyuki Kasahara, MD, PhD (2003 - 2006) 

Stefan Kochanek, MD (2002 - 2005) 

Michael R. Linden, PhD (2005 - 2008) 

Hiroyuki Mizuguchi, PhD (2005 - 2008) 

Hiroyuki Nakai, MD, PhD (2005 - 2008) 

Philip Ng, PhD (2004 - 2007) 

Robin J. Parks, PhD (2003 - 2006) 

Samuel D. Rabkin, PhD (2002 - 2005) 

Brian P. Sorrentino, MD (2004 - 2007) 

Arun Srivastava, PhD (2005 - 2008) 

David Strayer, MD, PhD (2002 - 2005) 


James M. Wilson, MD, PhD (2003 - 2006)


ASGT 8TH ANNUAL MEETING SUPPORTERS 

PARTNER ($26,000+) 

Abstracts-on-Disc 

General Meeting Support 

Supported in part by March of Dimes Birth Defects Foundation Grant 

No. 4-FY04-159 

Three Excellence in Research Awards 

Three Excellence in Research Awards 

Four Travel Grants 

Printing of Abstract Supplement Registration Bags 

CONTRIBUTOR ($10,000 - $25,000) 

PATRON ($500 - $9,000) 

Water Bottles Annual Reviews 

Two Excellence in Research Awards 

Three Travel Grants 

Excellence in Research Award 

Conference Notebooks 




National Heart, Lung and Blood Institute 


Lanyards

10 


ASGT 8 TH ANNUAL MEETING EXHIBITORS (As of April 27, 2005) 

Aldevron 

AllCells, LLC 

Alzet® Osmotic Pumps, Durect Corporation 

amaxa GmbH 

American Society of Gene Therapy 

Arkios BioDevelopment International 

Avanti Polar Lipids, Inc. 

Baltimore Area Convention & Visitors Association 

BioProcess International 

BioSpherix, Ltd. 

Cellgenix, GMBH 

Corning Incorporated 

DNAVEC Corporation 

Elsevier 

GE Healthcare 

GENEART GmbH 

Invitrogen Corporation 

InvivoGen 

Kemp Biotechnologies 

Lark Technologies 

Mary Ann Liebert, Inc. 

MDS Pharma Services 

Mirus Bio Corporation 

EXHIBIT HALL HOURS 

Thursday, June 2 2:00 pm – 7:30 pm 

(Welcome Reception begins at 4:00 pm) 

Friday, June 3 2:00 pm – 7:30 pm 

(Networking Reception begins at 4:00 pm) 

Saturday, June 4 3:00 pm – 7:00 pm 


Molecular Medicine BioServices 

National Gene Vector Laboratories 

Nature Publishing Group 

Nature Technology Corporation 

Office of Biotechnology Activities 

Olympus America Inc. 

Open Biosystems 

Pall Life Sciences 

PolyPlus Transfection 

Puresyn, Inc. 

RheoGene, Inc. 

Sangamo BioSciences, Inc. 

SeqWright, Inc. 

Sigma-Aldrich 

Southern Research Institute 

St. Jude Children’s Research Hospital 

Virapur, LLP 

VisualSonics, Inc. 

Vivascience, Inc. 

Waisman Clinical Biomanufacturing Facility 

Wiley 

Williamsburg BioProcessing


ABSTRACT REVIEWERS 

Committee Chair 


RNA Virus Vectors 

Coordinating Reviewer 

Michel Sadelain, MD, PhD 

Abstract Reviewers 

Lung-Ji Chang, PhD 

Tal Kafri, PhD 

Stephen J. Russell, MD, PhD 

John Tisdale, MD 

AAV Vectors 


Arun Srivastava, PhD 


Saswati Chatterjee, PhD 

John F. Engelhardt, PhD 

Roland W. Herzog, PhD 

R. Jude Samulski, PhD 

Adenovirus Vectors 


Michael J. Imperiale, PhD 


Joseph T. Bruder, PhD 

Maria A. Croyle, PhD 

Erik S. Falck-Pedersen, PhD 

Daniel A. Muruve, MD 

Other DNA Viruses 




William J. Bowers, PhD 

Istvan Fodor, PhD 

David Krisky, MD, PhD 

Yoshinaga Saeki, MD, PhD 

Naked DNA Transfer 


Jon Wolff, MD 


Richard Heller, PhD 

Carol H. Miao, PhD 

Blake Roessler, MD 

Ronald K. Scheule, PhD 

Lipid Mediated Gene Transfer 


Ralph W. Paul, PhD 


Todd Giorgio, PhD 

James E. Hagstrom, PhD 

Ram I. Mahato, PhD 

Volkmar Weissig, PhD 

Molecular Conjugates 


David V. Schaffer, PhD 


Kam W. Leong, PhD 

Suzie H. Pun, PhD 

Theresa M. Reineke, PhD 

Lonnie D. Shea, PhD 

Inborn Errors of Metabolism 


Brendan Lee, MD, PhD 


Barry J. Byrne, MD PhD 

Alessandra D’Azzo, PhD 

Mark S. Sands, PhD 

Edward Schuchman, PhD 

Cardiovascular 


J. Kevin Donahue, MD 


Frank J. Giordano, MD 

Walter J. Koch, PhD 

Marc Penn, MD, PhD 

Robert Simari, MD 

Neurologic 


Beverly L. Davidson, PhD 


Martha C. Bohn, PhD 

Steven Goldman, MD, PhD 

Pedro Lowenstein, MD, PhD 

Ronald Mandel, PhD 

Muscle and Connective Tissue 


Paul D. Robbins, PhD 


Jeffrey Chamberlain, PhD 

Olivier Danos, PhD 

Steven C. Ghivizzani, PhD 

Johnny Huard, PhD 

Infectious Diseases and Vaccines 


John A. Zaia, MD 


Ramesh Akkina, DVM, PhD 

Boro Dropulic, PhD 

Roger J. Pomerantz, MD, FACP 

Bruce E. Torbett, PhD, MSPH 

Cancer Immunotherapy 


Michael Jensen, MD 


G. Yancey Gillespie, PhD 

Michael Kalos, PhD 

Martin Pule, MD 

K.K. Wong, MD 

Cancer Apoptosis and Suicide 


Timothy Thompson, PhD 


Sunil Chada, PhD 

Svend O. Freytag, PhD 

Jack A. Roth, MD 

Patricia Yotnda, PhD 

Cancer Targeted Gene Therapy 


Albert B. Deisseroth, MD, PhD 


Estuardo Aguilar-Cordova, PhD 

William F. Benedict, MD 

Ronald G. Crystal, MD 

Ralph R. Weichselbaum, MD, PhD 

Hematologic 


Jan Nolta, PhD 


David W. Emery, PhD 

Craig T. Jordan, PhD 

Karen E. Pollok, PhD 

Christof von Kalle, MD 

Lung and Respiratory Disease 


Terence R. Flotte, MD 


Thomas W. Ferkol, MD 

William B. Guggino, PhD 

Raymond J. Pickles, PhD 

Joseph Zabner, MD 

Gene Regulation 


C. Anthony Blau, MD 


Martin Fussenegger, PhD 

Punam Malik, MD 

Brian P. Sorrentino, MD 

David Spencer, PhD 

James M. Wilson, MD, PhD

12 


PLANNING COMMITTEE & GROUP COORDINATING REVIEWERS 


University of Washington 

1959 NE Pacific Street 

Box 357710, K260 HSB 

Seattle, WA 98195 



975 W Walnut Street, Room 130 

Indianapolis, IN 46202 


The Children’s Hospital of Philadelphia 

Howard Hughes Medical Institute 

302 Abramson Research Center 

3615 Civic Center Boulevard 

Philadelphia, PA 19104-4399 


University of Michigan Medical School 

1500 E Medical Center Drive 

6304 Cancer Center 

Ann Arbor, MI 48109-0942 


Stanford University School of Medicine 

Departments of Pediatrics and Genetics 

300 Pasteur Drive, Room G-305A 

Stanford, CA 94305-5208 

Arthur Nienhuis, MD 


1011 Island Park Drive 

Memphis, TN 38103 


Washington University School of Medicine 

Division of Oncology 

Section of Stem Cell Biology 

660 South Euclid, Box 8007 

St. Louis, MO 63110 


Memorial Sloan-Kettering Cancer Center 

1275 York Avenue, Room S1014 

New York, NY 10021 

Jon Wolff, MD 

University of Wisconsin Madison 

Department of Pediatrics & Medical Genetics 

Waisman Center 

1500 Highland Ave 

Madison, WI 53705-2280 

Thank You 

Planning Committee 

& Reviewers!


RESEARCH AWARDS AND TRAVEL GRANT RECIPIENTS 

EXCELLENCE IN RESEARCH AWARDS 

For Students and Postdoctoral Fellows 

Alessandra Biffi – Postdoctoral Fellow 

Cure of Established Disease and Direct Evidence of Cellular 

Cross-Correction in the Nervous System of Metachromatic 

Leukodystrophy Mice after HSC-Based Gene Therapy 

Katherine Bowman – Student 

Oral Delivery of Non-Viral DNA Nanoparticles for 

Hemophilia A 

*Li Chen – Postdoctoral Fellow 

Complete and Persistent Phenotypic Correction of Phenylketonuria 

in Mice by Site-Specific Genome Integration of 

Phenylalanine Hydorxylase cDNA 

Aurelie Goyenvalle – Student 

Highly Efficient Exon-Skipping and Sustained Correction of 

Muscular Dystrophy Using an Adeno-Associated Viral Vector 

*Fanconi Anemia Research Fund Recipient 

TRAVEL GRANT RECIPIENTS 

For Students and Postdoctoral Fellows 

George Aslanidi 

Alessandra Biffi 

Martina Blumenthal 

Katherine Bowman 

Jian Chen 

Li Chen 

Hu Dai 

Mo Dao 

Michele De Palma 

Hongzhe Duan 

Anja Ehrhardt 

Herena Eixarch 

Anuj Gaggar 

Sabine Gerull 

Debadyuti R. Ghosh 

Aurelie Goyenvalle 

Jaeseok Han 

Scott Q. Harper 

Anne Kiang 

Changlong Li 

Jaeseok Han – Student 

Glucose-Dependent Insulin Production by Liver-Specific, 

Glucose-Regulatable Synthetic Promoters Results in the Cure 

of Diabetes 

Anne Kiang – Student 

Complement Blockade Prevents Several Innate Toxicities 

Rapidly Induced after Intravenous Adenovirus (Ad) Vector 

Administration 

Ruth Seggewiss – Postdoctoral Fellow 

Myeloid Sarcoma Associated with Gene Marking of 

Hematopoietic Stem Cells in a Rhesus Macaque 

Yan Shou – Postdoctoral Fellow 

Mouse Models for the Analysis of Genetic Risk Factors for 

Hematopoietic Transformation in XSCID Gene Therapy 

Angelo Leone Lombardo 

Francesco Marangoni 

Robert A. Marr 

Joel Montane 

John R. Ohlfest 

Cedric Raoul 

Luis Sanchez-Perez 

Manfred Schmidt 

Kerstin Schwarzwaelder 

Ruth Seggewiss 

Yan Shou 

Yue Si 

Matthias Stephan 

Konrad L. Streetz 

Yuki Takahashi 

Uma Thanarajasingam 

Zhijian Wu 

Tom Wurdinger 

Fang Xu 

Xiao-Bing Zhang 

The Excellence in Research Awards and Travel Grants were made possible 

through grant support from

14 


FACULTY LIST (as of April 15, 2005) 

Estuardo Aguilar-Cordova, PhD 

Advantagene 

Waban, Massachusetts 

Alessandro Aiuti, MD, PhD 

Fondazione Centro San Raffaele 

Del Monte Tabor 

Milano, Italy 


Colorado State University 

Fort Collins, Colorado 

Steven M. Albelda, MD 

University of Pennsylvania 

Philadelphia, Pennsylvania 

Andrea Amalfitano, DO, PhD 

Duke University Medical Center 

Durham, North Carolina 

Dale Ando, MD 

Sangamo BioSciences, Incorporated 

Richmond, California 

Valder Arruda, MD, PhD 


School of Medicine 


Andrew H. Baker, PhD 

University of Glasgow 

Glasgow, Scotland 

Jacques Banchereau, PhD 

Baylor Institute for Immunology 

Research 

Dallas, Texas 

Glen Barber, PhD 

University of Miami School of 

Medicine 

Miami, Florida 

Michael A. Barry, PhD 

Baylor College of Medicine and 

Rice University 

Houston, Texas 

David L. Bartlett, MD 

University of Pittsburgh 

Medical Center Cancer Pavilion 

Pittsburgh, Pennsylvania 

Christopher Baum, MD 

Hannover Medical School 

Hannover, Germany 

John C. Bell, PhD 

Ottawa Regional Cancer Centre 

Research Labs 

Ottawa, Canada 


MD Anderson Cancer Center 




Seattle, Washington 

David Bodine, PhD 

National Human Genome 

Research Institute 

Bethesda, Maryland 


Northwestern University 

Chicago, Illinois 

Chiara Bonini, MD 

Hospital San Raffaele 

Milan, Italy 

William J. Bowers, PhD 

University of Rochester 

School of Medicine and Dentistry 

Rochester, New York 



Charlestown, Massachusetts 

Malcolm K. Brenner, MD, PhD 

Baylor College of Medicine 


Joseph T. Bruder, PhD 

Genvec Incorporated 

Gaithersburg, Maryland 

Mary Bartlett Bunge, PhD 

Miami Project to Cure Paralysis 

Miami, Florida 

Bruce Bunnell, PhD 

Tulane University 

New Orleans, Louisiana 

Barry J. Byrne, MD, PhD 

University of Florida 


Gainesville, Florida 

Andrew Byrnes, PhD 

Food and Drug Administration 

Rockville, Maryland 

Michele P. Calos, PhD 

Stanford University 


Stanford, California 

Fabio Candotti, MD 

NIH/NHGRI 


Maria Castro, PhD 

Gene Therapeutics 


Los Angeles, California 

Joy Cavagnaro, PhD, DABT, RAC 

Access BIO 

Leesburg, Virginia 

Marina Cavazzana-Calvo, MD, PhD 

Hopital Necker Enfants-Malades 

Paris, France 

Sunil Chada, PhD 

Introgen Therapeutics Incorporated 


Jeffrey Chamberlain, PhD 




Lawrence C. Chan, MD 



Esther H. Chang, PhD 

Georgetown University 

Medical Center 

Washington, DC 

Saswati Chatterjee, PhD 

City of Hope National 


Duarte, California 

Seng H. Cheng, PhD 

Genzyme Corporation 

Framingham, Massachusetts 

E. Antonio Chiocca, MD, PhD 

The Ohio State University 


Columbus, Ohio 

Sally Choe, PhD 

Pfizer Incorporated 

Ann Arbor, Michigan 

Kyunghee Choi, PhD 

Washington University 


Saint Louis, Missouri 

K. Reed Clark, PhD 

Childrens Research Institute 


Paula Clemens, MD 


Pittsburgh, Pennsylvania


FACULTY LIST 

Laurence JN Cooper, MD, PhD 






Indianapolis, Indiana 

Robert H. Costa, PhD 

University of Illinois at Chicago 


Maria A. Croyle, PhD 

The University of Texas at Austin 

College of Pharmacy 

Austin, Texas 

Ronald G. Crystal, MD 

Weill Medical College 

of Cornell University 

New York, New York 

David T. Curiel, MD, PhD 

University of Alabama at 

Birmingham Gene Therapy Center 

Birmingham, Alabama 

George Q. Daley, MD, PhD 

Children’s Hospital 

Boston, Massachusetts 

Olivier Danos, PhD 

Genethon 

Evry, France 

Beverly L. Davidson, PhD 

University of Iowa 

Iowa City, Iowa 

Mark E. Davis, PhD 

California Institute of Technology 

Pasadena, California 


Sidney Kimmel Cancer Center 

San Diego, California 

Federica Del Monte, MD, PhD 



Boro Dropulic, PhD 

Lentigen Corporation 

Clarksville, Maryland 

Cynthia Dunbar, MD 

NIH/NHLBI Hematology Branch 


Andrew Ellington, PhD 

The University of Texas Austin 

Austin, Texas 

David W. Emery, MD 





College of Medicine 


Hildegund CJ Ertl, MD 


Wistar Institute 


Christopher Evans, PhD 

Harvard Medical School 


Howard J. Federoff, MD, PhD 



Thomas W. Ferkol, MD 



Saint Louis, Missouri 

Giuliana Ferrari, PhD 

H.S. Raffaele - Telethon Institute 

for Gene Therapy 

Milan, Italy 

Mitchell Finer, PhD 

Genteric 

Redwood City, California 

David J. Fink, MD 

University of Michigan 





Dimitry Gabrilovich, MD, PhD 

H. Lee Moffitt Cancer Center 

Tampa, Florida 

Guangping Gao, PhD 



Dan Gazit, DMD, PhD 

Hebrew University of Jerusalem 

Jerusalem, Israel 

Stanton L. Gerson, MD 

Case Western Reserve University 

Cleveland, Ohio 




Peter Glazer, MD, PhD 

Yale University School of Medicine 

New Haven, Connecticut 

Joseph C. Glorioso, PhD 




Manuel Grez, PhD 

Georg-Speyer-Haus 

Frankfurt, Germany 

Uta Griesenbach, PhD 

Imperial College Faculty of Medicine 

London, United Kingdom 

Linda G. Griffith, PhD 

Massachusetts Institute 

of Technology 

Cambridge, Massachusetts 

Markus Grompe, MD 

Oregon Health Science University 

Portland, Oregon 

Dieter Gruenert, PhD 

California Pacific Medical Center 


San Francisco, California 

Yongsong Guan, MD 

West China Hospital of Sichuan 

University 

Chengdu, China 

Emanuela Gussoni, PhD 

Harvard Medical School/ 

Children’s Hospital 


Neil R. Hackett, PhD 

Weill Medical College of 

Cornell University 


Christine L. Halbert, PhD 

Fred Hutchinson 

Cancer Research Center 



Mirus Corporation 

Madison, Wisconsin 

Roger J. Hajjar, MD 



H. Kirk Hammond, MD 

University of California 

San Diego School of Medicine 

La Jolla, California

16 


FACULTY LIST 

Cary Harding, MD 

Oregon Health Sciences University 

Portland, Oregon 

Stephen Hart, PhD 

Institute of Child Health 


Stephen D. Hauschka, PhD 



Kristen Hege, MD 

Cell Genesys Incorporated 

South San Francisco, California 

Adriana Heguy, PhD 

Weill Medical College of 

Cornell University 


Richard Heller, PhD 

University of South Florida 



Ulrich R. Hengge, MD 

University of Duesseldorf 

Duesseldorf, Germany 


The Children’s Hospital of 

Philadelphia 


Helen E. Heslop, MD 





Philadelphia 



Thomas J. Hope, PhD 

University of Illinois at Chicago 



Jim Hu, PhD 

Hospital for Sick Children 

Toronto, Canada 

Leaf Huang, PhD 


Center for Pharmacogenetics 


Johnny Huard, PhD 

Children’s Hospital of Pittsburgh 


R. Keith Humphries, MD, PhD 

Terry Fox Lab 

Vancouver, Canada 

Stephen Hyde, PhD 

University of Oxford 

Oxford, United Kingdom 

John Iacomini, PhD 

Brigham and Women’s Hospital 



University of Michigan 

Medical School 


Michael Jensen, MD 




Larry G. Johnson, MD 

University of North Carolina 

at Chapel Hill 

Chapel Hill, North Carolina 

Douglas J. Jolly, PhD 

Advantagene, Incorporated 

Encinitas, California 

Karin Jooss, PhD 

Cell Genesys, Incorporated 


Carl H. June, MD 



Alexander V. Kabanov, PhD 

University of Nebraska 


Omaha, Nebraska 




Michael Kaplitt, MD, PhD 

Weill Medical College of Cornell 

University 


Noriyuki Kasahara, MD, PhD 

University of California Los Angeles 

Geffen School of Medicine 






David Kaye, FRACP, PhD, FACC 

Baker Heart Research Institute 

Melbourne, Australia 

Haig H. Kazazian, Jr., MD 




Hans-Peter Kiem, MD 

Fred Hutchinson Cancer 

Research Center 


Nancy M.P. King, JD 


at Chapel Hill 


Richard A. Knazek, MD 

National Center for 

Research Resources 


Donald B. Kohn, MD 

Childrens Hospital Los Angeles 


Victor Krasnykh, PhD 



David Krisky, MD, PhD 



Akihiro Kume MD, PhD 

Jichi Medical School 

Tochigi, Japan 





Paola Leone, PhD 

University of Medicine & Dentistry 

of New Jersey 

Camden, New Jersey 

Philip L. Leopold, PhD 

Weill Medical College of Cornell 

University 


Bruce L. Levine, PhD 




Andre Lieber, MD, PhD 


Seattle, Washington


FACULTY LIST 

Michael R. Linden, PhD 

Mount Sinai School of Medicine 


Hans J. Lipps, PhD 

University of Witten 

Witten, Germany 

Dexi Liu, PhD 



Margaret A. Liu, MD 

Transgene 

Lafayette, California 

Barry London, MD, PhD 




Douglas Losordo, MD 

Caritas St. Elizabeth’s 



Pedro Lowenstein, MD, PhD 

Gene Therapeutics 



Carolyn Lutzko, PhD 



Ian MacLachlan, PhD 

Protiva Biotherapeutics Incorporated 

Burnaby, Canada 

Harry L. Malech, MD 

NIH/NIAID Laboratory of 

Host Defenses 


Punam Malik, MD 



Ronald Mandel, PhD 

University of Florida College of 

Medicine 


Francesco M. Marincola, MD 

National Institutes of Health 


R. Scott McIvor, PhD 

University of Minnesota 

Minneapolis, Minnesota 

Gerry McLachlan, PhD 

Wellcome Trust Centre 

Edinburgh, United Kingdom 

Michael McManus, PhD 

University of California San 

Francisco Diabetes Center 

San Francisco, California 


University of Washington and 

Children’s Hospital and 

Regional Medical Center 


Lluis M. Mir, DSc 

Centre National de la Recherche 

Scientifique - Institute 

Gustave-Roussy 

Villejuif, France 

Kohnosuke Mitani, PhD 

Saitama Medical School 

Hidaka, Japan 

John C. Morris, MD 

National Cancer Institute 


Nicholas Muzyczka, PhD 



Muna Naash, PhD 

University of Oklahoma Health 

Sciences Center 

Oklahoma City, Oklahoma 

Hiroyuki Nakai, MD, PhD 




Luigi Naldini, MD, PhD 

Fondazione Centro S. Raffaele Del 

Monte Tabor 

Milan, Italy 

Robert M. Nelson, MD, PhD 


Philadelphia 


Glen R. Nemerow, PhD 

The Scripps Research Institute 

La Jolla, California 

John J. Nemunaitis, MD 

Mary Crowley Medical 


Dallas, Texas 

Arthur W. Nienhuis, MD 

St. Jude Children’s 

Research Hospital 

Memphis, Tennesee 




St. Louis, Missouri 

James S. Norris, PhD 

Medical University of 

South Carolina 

Charleston, South Carolina 

Dennis Panicali, PhD 

Therion Biologics Corporated 

Cambridge, Massachusetts 

Drew Pardoll, MD, PhD 

John Hopkins University 


Baltimore, Maryland 

William M. Pardridge, MD 

University of California Los Angeles 



Robin J. Parks, PhD 

Ottawa Health Research Institute 

Ottawa, Canada 

Ralph W. Paul, PhD 

Targeted Genetics Corporation 



Herman B. Wells Center for 

Pediatric Research 


Suzie H. Pun, PhD 



Joseph Rabinowitz, PhD 

Thomas Jefferson University 


Samuel D. Rabkin, PhD 



Jeffrey Rade, MD 

Johns Hopkins School of Medicine 

Baltimore, Maryland 

Thomas A. Rando, MD, PhD 




The University of Cincinnati 

Cincinnati, Ohio 

Jakob Reiser, PhD 

LSU Helath Sciences Center 

New Orleans, Louisiana

18 


FACULTY LIST 

Morayma Reyes, MD, PhD 


Sammamish, Washington 

Stanley Riddell, MD 

Fred Hutchinson Cancer 



Isabelle Riviere, PhD 

Memorial Sloan Kettering 

Cancer Center 


Paul D. Robbins, PhD 



Maria Grazia Roncarolo, MD 

San Raffaele Institute for 

Gene Therapy 

Milano, Italy 

Daniel Rosenblum, MD 



Anthony Rosenzweig, MD 



John J. Rossi, PhD 

Beckman Research Institute 

City of Hope 


Jack A. Roth, MD 

University of Texas 



Jonathan B. Rothbard, PhD 


Menlo Park, California 

Jayanta Roy-Chowdhury, MD 

Albert Einstein College of Medicine 

Bronx, New York 

David W. Russell, MD, PhD 



Stephen J. Russell, MD, PhD 

Mayo Clinic 

Rochester, Minnesota 


Memorial Sloan-Kettering 

Cancer Center 



The Ohio State University 

Medical Institute 


R. Jude Samulski, PhD 

University of North Carolina at 

Chapel Hill 





St Louis, Missouri 


University of California Berkeley 

Berkeley, California 

Daniel Scherman, PhD 

Institut National de la Santé et de la 

Recherche Médicale 

Paris, France 

Ronald K. Scheule, PhD 

Genzyme Corporation 

Framingham, Massachusetts 

Edward M. Schwarz, PhD 



Mercedes Serabian, MS, DABT 



Lonnie D. Shea, PhD 

Northwestern University 

Evanston, Illinois 

Rebecca Sheets, PhD 



Stephanie Simek, PhD 



Patrick L. Sinn, PhD 



Albert J. Sinusas, MD 

Yale University School of Medicine 

New Haven, Connecticut 

Sonia I. Skarlatos, PhD 



Nay Wei Soong, PhD 

Maxygen Incorporated 

Redwood City, California 


St Jude Children’s Research Hospital 

Memphis, Tennesee 

David Spencer, PhD 



Arun Srivastava, PhD 



Alachua, Florida 

Daniel H. Sterman, MD 




Bruce Sullenger, PhD 

Duke University Medical Center 

Durham, North Carolina 

Lorne Taichman, MD, MSc, PhD 

State University of New York at 

Stony Brook 

Stony Brook, New York 

Shin’ichi Takeda, MD, PhD 

National Institute Neuroscience 

Kodaira, Japan 

Alice F. Tarantal, PhD 

University of California Davis 

Primate Center 

Davis, California 

Adrian J. Thrasher, MD, PhD 

Institute of Child Health 






The Scripps Research Institute 

La Jolla, California 

Thomas Tuschl, PhD 

Rockefeller University 


Jeffrey Ulmer, PhD 

Chiron Vaccines 

Emeryville, California 

James B. Uney, PhD 

University of Bristol 

Bristol, United Kingdom 

Thierry C. Vandendriessche, PhD 

University of Leuven 

Leuven, Belgium


FACULTY LIST 


Cincinnati Children’s Hospital 

Research Foundation 

Cincinnati, Ohio 

Dorothee Von Laer, MD 

Georg Speyer Haus 

Frankfurt, Germany 

Ernst Wagner, PhD 

University of Munich 

Munich, Germany 

Britta Elisabeth Wahren, MD, PhD 

Swedish Institute for Infectious 

Disease Control 

Stockholm, Sweden 

David B. Weiner, PhD 



Daniel Weiss, MD, PhD 

University of Vermont 


Burlington, Vermont 

Bryan Williams, PhD 

The Cleveland Clinic Foundation 

Cleveland, Ohio 

Carolyn Wilson, PhD 



James M. Wilson, MD, PhD 




William S. M. Wold, PhD 

St. Louis University 


St. Louis, Missouri 

John H. Wolfe, VMD, PhD 

The Children’s Hospital 

of Philadelphia 


Jon Wolff, MD 

University of Wisconsin Madison 

Madison, Wisconsin 

Savio L.C. Woo, PhD 

Mount Sinai School of Medicine 


Peter K. Working, PhD, DABT 

Cell Genesys, Incorporated 


Xiao Xiao, PhD 



Ning-Sun Yang, PhD 

Inst of BioAgricultural Sciences 

Academia Sinica 

Taipei, Taiwan 

Mervin Yoder, MD 

Cancer Research Institute 





Hua Yu, PhD 

H. Lee Moffitt Cancer Center 






Steven J. Zullo, PhD 


Bethesda, Maryland

20 


GENERAL MEETING INFORMATION 

AMERICAN SOCIETY OF GENE 

THERAPY 

MISSION AND GOALS 

The American Society of Gene Therapy (ASGT) is a 

professional non-profit medical and scientific 

organization dedicated to the understanding, 

development and application of gene and related cell and 

nucleic acid therapies and the promotion of professional 

and public education in the field. 

ASGT is the largest medical professional organization 

representing researchers and scientists dedicated to 

discovering new gene therapies. ASGT was established in 

1996, and has grown to close to 3,000 members from 

around the world. ASGT is committed to: 

· Promoting Research, Development and Application 

· Exchanging Information and Promote Education 

Among Professionals and the Public 

· Promoting Development of Clinical Translations 

The ASGT 8th Annual Meeting will serve as a 

comprehensive forum to achieve these goals. 

NEEDS 

Clinical gene transfer has become more and more 

complex due to ongoing developments in the fields of 

gene therapy itself, together with bioethics, research 

integrity, and financial conflicts, as well as new federal 

mandates, regulations and guidelines. Oligonucleotide 

Based Therapies, RNAi and Vaccine Therapies will also 

be discussed as well as many other topics. This meeting 

will provide an educational forum for scientists and 

clinicians to expand their knowledge about the broad 

developments in these fields. 

EDUCATIONAL OBJECTIVES 

At the conclusion of the activity, the participant should be 

able to: 

· Discuss key aspects of the current laboratory and/or 

clinical research in gene therapy. 

· Demonstrate knowledge of methods being used by 

gene therapy researchers and practitioners. 

· Describe one or more stimulating new areas of 

inquiry in the area of gene therapy. 

· Demonstrate familiarity with federal regulations and 

guidelines for clinical gene transfer studies. 

EDUCATIONAL METHODS AND 

MATERIALS 

Lectures, Case Presentations, Panel Discussions, 

Question and Answer Sessions, Workshops, Audio/Video 

Presentations, Abstracts, Posters, Executive Summary. 

TARGET AUDIENCE 

The target audience includes basic science and 

translational researchers, clinical investigators, physicians, 

industry executives and scientists, postdoctoral fellows, 

graduate students, employees of federal government and 

regulatory agencies, and other healthcare professionals 

with an interest in the latest advancements in the field of 

gene therapy. 

EVALUATION METHOD 

Evaluation by questionnaire will address program 

content, presentation, and possible bias. Please take time 

to complete the evaluation forms distributed at each 

session you attend. Your input and comments are 

essential in planning future educational sessions. When 

completed, evaluations can be returned to the ushers 

assigned to your session or dropped in the evaluation 

collection boxes located throughout America’s Center. 

ACCREDITATION/CREDIT 

DESIGNATION 

This activity has been planned and implemented in 

accordance with the Essential Areas and policies of the 

Accreditation Council for Continuing Medical Education 

through the joint sponsorship of Stanford University 

School of Medicine and the American Society of Gene 

Therapy. Stanford University School of Medicine is 

accredited by the ACCME to provide continuing medical 

education for physicians. 

Stanford University School of Medicine designates this 

educational activity for a maximum of 31 category 1 

credits toward the AMA Physician’s Recognition Award. 

Each physician should claim only those hours of credit 

that he/she actually spent in the activity. 

DISCLOSURE 

It is the policy of Stanford University that the provider 

and faculty/presenters disclose any significant financial or 

other relationships with commercial companies whose 

products may be discussed in the activity, or the 

commercial supporters, if any. Stanford University also 

requires that faculty disclose any unlabeled use or 

investigational use (not yet approved for any purpose) of 

pharmaceutical and medical device products. Specific 

disclosure will be made to the participants prior to the 

educational activity. 

Faculty, topics, program schedule, and credit are subject 

to change. 

CME CERTIFICATES 

CME Certificates will be available at the CME desk for 

participants requiring CME credit for this meeting.



CME SIGN IN 

All participants requiring CME credit must sign in each 

day they are in attendance at the ASGT 8 th Annual 

Meeting. The sign-in area is adjacent to the registration 

desk, outside of Hall 1 (Washington Avenue Lobby). A 

CME Certificate will not be available unless you sign in 

each day at the meeting. 

ABSTRACT VOLUME 

All abstracts accepted for presentation at the ASGT 8 th 

Annual Meeting have been published in the May 

supplement of Molecular Therapy. Each attendee will 

receive one copy of the supplement along with other 

registration materials. 

ADMISSION TO SESSIONS 

Official name badges will be required for admission to all 

ASGT sessions. All Annual Meeting attendees receive a 

name badge with their registration bag. Name badges 

should be worn at all times inside the convention center, 

as badges will be used to control access to sessions and 

activities. Attendees are cautioned against wearing their 

name badges while away from the convention center as 

badges may draw unwanted attention to your status as 

visitors to St. Louis. 

ADMISSION BY TICKET ONLY 

The Meet-the-Investigator sessions require tickets for 

admission. Tickets for these sessions are not included in 

the meeting’s regular registration fees. Tickets will be 

collected at the door by ushers. There are still a limited 

number of tickets available which may be obtained at the 

Registration Desk. Tickets are $20 and include a box 

lunch. 

BUSINESS CENTERS 

Business centers are available for attendees at America’s 

Center and the Renaissance Grand. Please stop by either 

of the centers for a complete list of fees, services and 

hours. 

BUSINESS MEETING 

Friday, June 3, 2005 

4:30 p.m. - 5:30 p.m. 

Room 130 

All members welcome! 

See page 3 for more details. 

COMMITTEE MEETINGS 

See page 3 for Committee Meeting information. 

GUEST ATTENDANCE 

ASGT asks registered attendees to refrain from taking 

children, spouses, or guests to any educational session or 

functions offered at the 8 th Annual Meeting. 

CELL PHONES 

Please insure that cell phone ringers and pagers are 

turned off during all sessions. 

NO SMOKING 

Smoking is prohibited at all 8 th Annual Meeting sessions 

and events. 

JOB BANK 

All job openings posted as of May 20, 2005 on the ASGT 

Job Bank have been included in the Jobs Available 

Booklet found in the registration bags. Contact 

information is included in the booklet. 

Job postings for open positions and resumes may be 

posted on the Job Posting Area boards near the 

Registration desk outside of Hall 1 (Washington Avenue 

Lobby). You are responsible for making copies of items 

you wish to post. Please do not post job openings or 

resumes on the ASGT Message Board. 

INTERNET KIOSK POLICY 

Internet access will be available to meeting attendees in 

two locations. One kiosk is located on Level One in the 

Plaza Lobby and the second kiosk is in the Second Floor 

Atrium on Level Two. Please limit your Internet use to 15 

minutes at a time so that other attendees can access the 

Internet as well. 

SHUTTLE BUS 

ASGT will not be providing shuttle transportation for the 

8 th Annual Meeting in St. Louis. America’s Center is 

within a one block radius of the ASGT Annual Meeting 

hotels. 

SPECIAL ACCESSIBILITY NEEDS 

We encourage participation by all individuals. If you 

require special accommodations and did not previously 

contact the ASGT Executive Office, please go to the 

Registration Desk. The ASGT staff will be happy to assist 

you with your specific needs.

22 



EXHIBITS 

Exhibits will be located in Hall 1 (Level One) of 

America’s Center. Please allow adequate time in your 

daily schedule to visit the exhibits. Please take the time to 

speak with representatives of companies that provide 

services or market products directly related to your 

professional needs. Per ASGT policy, promotional 

materials will not be permitted in the meeting halls or 

registration area or in any location of America’s Center 

outside the rented exhibit space. 

A Welcome Reception will be held in the Exhibit Hall on 

Thursday, June 2, and Networking Receptions in the 

Exhibit Hall on Friday, June 3, and Saturday, June 4. 

Receptions will begin at 4:00 pm daily. 





LEAD RETRIEVAL 

A lead retrieval system has been made available to all 

exhibitors of the ASGT 8 th Annual Meeting. Exhibitors 

may ask to scan attendee name badges with a hand held 

scanner in order to obtain attendee contact information. 

POSTERS 

Abstract Posters will be on display in Hall 2. 


ABSTRACT POSTER SESSION I 

10:00 am – Noon Poster Setup by Authors 

Noon – 7:30 pm Poster Viewing (authors present 

from 4:00 pm – 7:30 pm) 

2:00 pm – 7:30 pm Exhibit Hall Open 

4:00 pm – 7:30 pm Welcome Reception 

7:30 pm – 8:00 pm Authors Remove Posters 


ABSTRACT POSTER SESSION II 



from 4:00 pm – 7:30 pm) 


4:00 pm – 7:30 pm Networking Reception 



ABSTRACT POSTER SESSION III 



from 4:00 pm – 7:00 pm) 


4:00 pm – 7:00 pm Networking Reception 


PRESS ROOM 

The Press Room is located in America’s Center, Level 

Two, Room 280. 

Members of the working media may register for the 8th Annual Meeting in the Press Room. Press conferences on 

previously selected newsworthy abstracts, along with 

other scientific sessions, will be conducted throughout 

the meeting. Interview space, computers, telephones, and 

fax services are available for the convenience of media 

representatives covering the meeting. Press must register, 

provide credentials, and wear their press badge for 

admittance to ASGT sessions. 

PRESS ROOM HOURS 

Wednesday, June 1 3:00 pm – 6:00 pm 

Thursday, June 2 7:45 am – 5:00 pm 

Friday, June 3 7:45 am – 5:00 pm 

Saturday, June 4 7:45 am – 5:00 pm 

REGISTRATION DESK 

Name badges, final programs, and abstract supplements 

will be distributed at the registration desk located on 

Level One of America’s Center, outside of Hall 1 

(Washington Avenue Lobby). 

REGISTRATION DESK HOURS 

Wednesday, June 1 Noon – 8:00 pm 




Sunday, June 5 8:00 am – Noon 

RESTAURANT INFORMATION DESK 

St. Louis offers a variety of restaurants from casual to 

elegant, with prices and cuisine to meet all tastes and 

budgets. The Information Desk is staffed by the St. Louis 

Convention & Visitors Commission, to help you make 

the right dining choice. Staff will have sample menus 

from area restaurants and will be able to assist you in 

making your reservations. The Restaurant Information 

Desk is located on Level One of America’s Center near 

Registration, outside of Hall 1 (Washington Avenue 

Lobby).



SPEAKER READY ROOM 

The Speaker Ready Room is located in Room D (Level 

One) of America’s Center. 

Important Notice: All faculty must deliver their 

presentations to the Speaker Ready Room the day before 

their session or at least five hours prior to their 

presentations. Equipment is available for faculty to review 

their materials. Audiovisual personnel will be available for 

assistance. Please mark your materials (include name, 

session and speaker order) so the information can be 

returned to you. 

SPEAKER READY ROOM HOURS 

Wednesday, June 1 10:00 am – 9:00 pm 




Sunday, June 5 7:30 am – 12:00 noon 

The Society strongly encourages faculty to pre-load presentations in the Speaker Ready Room; 

those faculty that load presentations during the session will have that time deducted from their 

presentation time by the Chair or Moderator.

24 


2005 PROGRAM-AT-A-GLANCE 

WEDNESDAY, JUNE 1 

8:00 am – 3:00 pm Pre-Meeting Session: 2nd Stem Cell Clonality & Genotoxicity Retreat 

Noon – 8:00 pm Attendee Registration Open 

12:30 pm – 4:00 pm ASGT Board of Directors Meeting 

3:30 pm – 5:00 pm Education Sessions I 

5:15 pm – 6:45 pm Education Sessions II 

6:45 pm – 8:30 pm Dinner Break 

8:30 pm – 10:00 pm 


Education Sessions III 

7:00 am – 5:00 pm Attendee Registration Open 

8:00 am – 9:00 am George Stamatoyannopoulos Lecture and Excellence in Research Awards 

Keynote Address: Thomas Tuschl, PhD 

9:15 am – 12:30 pm Workshops 


Noon – 7:30 pm Poster Viewing 

12:45 pm – 1:45 pm Meet-the-Investigator Lunches 

2:00 pm – 4:30 pm Oral Abstract Sessions 


4:00 pm – 7:30 pm 


Poster Session, Exhibits, and Welcome Reception 



8:00 am – 10:00 am Scientific Symposia 

10:15 am – 12:15 pm Scientific Symposia 



2:00 pm – 4:30 pm Oral Abstract Sessions 


4:00 pm – 7:30 pm Poster Session, Exhibits, and Networking Reception 

4:30 pm – 5:30 pm Business Meeting 

8:30 pm – 10:00 pm 


Evening Session: Immune Responses in Neurologic Gene Therapy 



8:00 am – 10:00 am Scientific Symposia 

10:15 am – 12:15 pm Oral Abstract Sessions 



1:45 pm – 4:15 pm Presidential Symposium and Presentation of Top Abstracts 

Keynote Address: George Q. Daley, MD, PhD 

Presidential Address: Katherine A. High, MD 


4:00 pm – 7:00 pm Poster Session, Exhibits, and Networking Reception 

4:30 pm – 6:00 pm 

SUNDAY, JUNE 5 

ASGT Board of Directors Meeting 

8:00 am – Noon Attendee Registration Open 

8:30 am – 10:00 am Young Investigators Symposium 

10:15 am – 12:15 pm Oral Abstract Sessions 

12:15 pm 8th Annual Meeting Concludes


SCIENTIFIC PROGRAM 

WEDNESDAY, JUNE 1, 2005 

EDUCATION SESSION I – 3:30 PM - 5:00 PM 

Education Session 100 

Retrovirus and Lentivirus Vectors 

This session will introduce three of the most popular versions of 

retroviral delivery systems, for which an increasing repertoire of 

strains and genera becomes available: gammaretroviral (based on 

murine leukemia virus), lentiviral (based on human 

immunodeficiency virus type 1) and spumaviral (based on 

“human” foamyvirus). They share the default aspects of the 

retroviral life cycle: receptor-mediated uptake of an enveloped 

particle, reverse transcription of a plus-stranded mRNA, and more 

or less random integration of double-stranded DNA following 

nuclear uptake of the preintegration complex. However, we will 

also discuss important differences in efficiency and biosafety related 

to viral origin and vector architecture. 

Room: 263/267 

Chair: Christopher Baum, MD 

Speakers: Christopher Baum, MD - Retrovirus Vectors: 

We Are Family! 

Luigi Naldini, MD, PhD - Lentiviral Vectors: 

Challenges and Expectations 

David W. Russell, MD, PhD - Foamy Virus 

Vectors 


Nonviral Vectors 

Nonviral gene therapy approaches may be simpler and safer than 

those using viral vectors. The challenges of non-viral gene therapy 

involve 1) development of vectors that provide long-term 

expression of therapeutic genes, and 2) development of suitable 

methods to deliver vector DNA to target cells. This session will 

examine the latest integrating and non-integrating vectors for 

achieving long-term gene expression and will describe current 

physical and chemical methods for DNA delivery. 

Room: 260/264 

Chair: Michele P. Calos, PhD 

Speakers: Michele P. Calos, PhD - Integrating and 

Non-integrating Plasmids for Non-viral Gene 

Therapy 

Daniel Scherman, PhD - In Vivo Electrotransfer 

of Plasmids or Synthetic Nucleotides: Basic 

Concepts and Potential Therapeutic Applications 

Dexi Liu, PhD - What Constitutes a Better 

Synthetic Vector? 


Immune Responses 

In vivo delivery of genes for therapeutic purposes and vaccines 

induces immune responses directed both against the vector as well 

as the gene-product. The goal of this educational session is to 

provide an overview of the role of immune responses in strategies 

for development of vector systems for gene therapy and vaccines. 

The overview of the immune response will include innate 

immunity, antigen processing and presentation, lymphocyte 

activation, induction of effecter responses and regulation of the 

immune responses. There will be a presentation and discussion on 

the use of gene transfer to induce tolerance to therapeutic or 

transplant antigens. Several exciting new approaches involving 

mechanisms of deletion, energy, or suppression will be discussed. 

In addition experiences of immunologic factors influencing efficacy, 

and safety in clinical trials with viral vectors will be reviewed. 

Room: 120/124 

Chair: Maria Grazia Roncarolo, MD 

Speakers: Maria Grazia Roncarolo, MD - The Role of 

Regulatory T Cells in Suppressing Immune 

Responses to Transgenes and Inducing Immune 

Tolerance 

Jacques Banchereau, PhD - Dendritic Cells: 

Controllers of Immune Responses 


Regulatory Hurdles to Phase III Clinical Trials 

This session will discuss FDA’s perspectives on what investigator/ 

sponsors can do to help move the field of gene therapy forward and 

get products to licensure. FDA will discuss what is the most 

efficient way to establish safety and efficacy, as well as what type of 

preclinical studies should be performed prior to initiation of 

clinical trials. Questions regarding the conduct of exploratory and 

confirmatory clinical trials will be discussed. FDA will also discuss 

why product characterization is important during early product 

development. These issues will be discussed as a way of explaining 

how the academic investigator can play a role in partnering with 

industry to help bring gene therapy products to licensure. 

Room: 265/266 

Chair: Stephanie Simek, PhD 

Speakers: Andrew Byrnes, PhD - Common Challenges in 

the Development of Gene Therapy Products 

Mercedes Serabian, MS, DABT - Preclinical 

Considerations Beyond Phase 1 

Daniel Rosenblum, MD - How to Make 

Optimal Use of the Approval Process

26 





Expression Cassette Design 

Gene Therapy projects require expression of an exogenously 

introduced therapeutic gene. In The 1995 Orkin and Motulsky 

report identified inadequate knowledge of vector components 

necessary to maintain adequate expression of the therapeutic gene 

as a major fault in the field. Over the last 10 years, a significant 

amount of effort has been invested toward the development of 

optimized expression cassettes that provide sustained, high – level 

expression of therapeutic proteins in relevant disease models with 

viral and non – viral vectors. This session will provide guidance 

for the development of expression cassettes yielding high level, 

sustained expression of a desired therapeutic protein. The three 

presentations will provide (1) an overview to expression cassette 

design for viral and non – viral vectors, with a focus on using 

viral and house – keeping promoters for highest level of gene 

expression initially following gene transfer, (2) strategies that have 

been successfully used to provide sustained, high level tissue – 

specific expression, and (3) vector strategies that optimize protein 

expression through the use of optimal intron configuration and 

mRNA splicing, mRNA stability elements and translation 

initiation site configuration. A question and answer session will 

follow. 

Room: 123/127 

Chair: Mitchell Finer, PhD 

Speakers: Mitchell Finer, PhD - Expression Cassette 

Design - 20 Years in 20 Minutes 

Carol H. Miao, PhD - Establishment of 

High-Level and Tissue-Specific Gene Expression 

Cassettes 

Thomas J. Hope, PhD - Post Transcriptional 

Regulation in Gene Expression Vectors 


Cancer Gene Therapy 

Cancer has been a target application for numerous gene therapy 

trials. Recent advances in the use of oncolytic viruses, the type of 

anticancer transgenes employed for biologic effects and the effects of 

the immune system will be discussed. The understanding of these 

topics will facilitate improved applications in human trials. 

Room: 240 Complex 

Chair: E. Antonio Chiocca, MD, PhD 

Speakers: E. Antonio Chiocca, MD, PhD - HSV 

Oncolytic Viruses 

Glen Barber, PhD - VSV as an Oncolytic Vector: 

Mechanisms of Action 

Noriyuki Kasahara, MD, PhD - Retrovirus 

Vectors for Cancer Gene Therapy: Oncolytic, 

Anti-angiogenic, and Immunotherapeutic Strategies 

BREAK – 5:00 PM - 5:15 PM 

EDUCATION SESSION II – 5:15 PM - 6:45 PM 



The goal of the Adenovirus Education Session is to survey the 

current state-of-the-art in our development and use of adenovirus 

gene transfer vectors. The session will begin with a discussion of 

the molecular biology of adenovirus including the production of 

replication –deficient vectors and packaging cell lines, the benefits 

and deficits of deletions of subsets or all the viral genes in the 

vector, the logic behind conditionally replicating adenoviruses, and 

variations relating to the Transgene expression cassette. The second 

topic of discussion will address the efficiency with which 

adenovirus interacts with target cells to deliver its genome to the 

nucleus. This discussion will include the basic infection pathway, 

altered vector tropism, variables relating to target cell type and 

physiology, and in vivo vector trafficking. The final topic of the 

session will be the interaction between adenovirus and the 

immune system including innate immunity, acquired immunity, 

and strategies for circumventing immune system barriers. 

Room: 263/267 

Chair: Philip L. Leopold, PhD 

Speakers: Neil R. Hackett, PhD - Molecular Biology of 


Philip L. Leopold, PhD - Cell Biology of 

Adenovirus Infection: Subcellular Mimicry 

James M. Wilson, MD PhD - Immunology of 

Adenoviral Vectors 


HSV Vectors 

This session will describe design features and production features, 

plus experimental and therapeutic uses of recombinant oncolytic, 

replication defective and amplicon vectors derived from herpes 

simplex virus type 1. Oncolytic vectors are designed for treatment 

of cancer based on targeted delivery and selective virus replication 

in tumor cells. Clinical trials using these vectors for brain tumors 

will be described. Replication defective viruses deleted for essential 

viral functions have been engineered for peripheral nervous system 

applications since they naturally persist in sensory nerves following 

local delivery to the skin. Amplicon vectors incorporate no viral 

genes and can include up to 150 kb of genetic information, 

including complete genes, self-contained regulatory elements and 

AAV genome integrating components. These vectors provide 

versatile tools for systematic genetics and therapeutics. 

Room: 260/264 

Chair: Xandra Breakefield, PhD 

Speakers: Xandra Breakefield, PhD - Mega-multi 

Capacity HSV Amplicon Vectors 

Joseph C. Glorioso, PhD - HSV Gene Vectors 

for Treatment of Pain and Neuropathy 

E. Antonio Chiocca, MD, PhD - HSV Vectors 

for Chemotherapy Delivery





Regulation of Transgene Expression 

The ability to regulate the timing and level of transgene expression 

opened new avenues in basic research applications and might 

become prerequisites in future clinical settings. Several gene 

regulation systems have been developed and successfully 

incorporated into viral and non-viral gene delivery systems. The 

session will begin with a general overview on the characteristics 

common to the various gene expression regulating systems. The 

following parts of the session will focus on the most common gene 

regulatory systems including the tetracycline, ecdysone, and the 

dimerizer systems. The last part of this session will cover new 

developments in regulating siRNA expression cassettes. 

Room: 265/266 

Chair: Tal Kafri, PhD 

Speakers: Tal Kafri, PhD - General overview on transgene 

regulation with special emphasis on the tetracycline 

gene regulatory system 

Francesco Galimi, MD, PhD - Regulation of 

Transgene Expression: Vector Designs and 

Applications 

Jakob Reiser, PhD - Knockdown Control: 

Practical Approaches to Regulating shRNA 

Expression 


NIH Grantsmanship & Funding Opportunities 

The goal of this session is to acquaint participants with training 

and career development application mechanisms and the review 

process at the NIH. Staff will give advice on what contributes a 

successful grant application and how to pick the right mechanism 

depending on the applicant’s expertise and education. There will 

be an opportunity for questions during the presentations and NIH 

staff will be available for individual questions. This session is 

intended for new investigators but should be informative for any 

investigator planning to submit an NIH grant application. 

Room: 120/124 

Chair: Sonia I. Skarlatos, PhD 

Speakers: Sonia I. Skarlatos, PhD - Research Training and 

Career Development Programs 

Steven J. Zullo, PhD - Grant Review in the 

CSR 

Arun Srivastava, PhD - Writing and Reviewing 

NIH Grants: A Personal Odyssey 


Gene Transfer to Muscle 

This educational session will focus on applications, methods and 

strategies for gene transfer to muscle tissue. The first lecture will 

give an overview of viral vectors for muscle gene transfer in vivo. 

The advantages and disadvantages of a variety of commonly used 

vectors with examples of their application will be discussed. The 

second lecture will focus on non-viral methods for gene transfer to 

muscle, which is uniquely suited for uptake of non-viral vectors. 

The final topic will be on ex vivo methods for gene transfer and 

appropriate vectors for transducing stem cells. 

Room: 123/127 

Chair: Jeffrey Chamberlain, PhD 

Speakers: Jeffrey Chamberlain, PhD - Viral Gene 

Transfer 

Jon Wolff, MD - Non-Viral Gene Transfer 

Giuliana Ferrari, PhD - Cell Therapy for 

Muscular Dystrophy 


Building a Strategy for Translational Medicine: Focus 

on Immunodeficiencies 

The goal of this session is to highlight scientific, technological and 

logistical aspects that bear on the successful translation and clinical 

implementation of gene therapy research. This year’s session will 

focus on the severe combined immune deficiencies (SCID). The 

faculty will discuss their experience with adenosine deaminase 

(ADA) deficiency, X-linked SCID and Wiskott-Aldrich 

syndrome, with emphasis on the strategic choices they have made 

and the hurdles they have confronted on issues such as vector/ 

promoter selection, small vs. large animal models, vector 

production, infrastructure development, protocol approval, patient 

selection and recruitment, quality assurance and regulatory 

oversight. 

Room: 230/231 

Chair: Michel Sadelain, MD PhD 

Speakers: Donald B. Kohn, MD - ADA-Deficient SCID: 

Pathogenis and Treatments 

Marina Cavazzana-Calvo, MD, PhD - Severe 

Combined Immunodeficiency: Disease Models for 

New Therapetical Approaches 

Arthur Nienhuis, MD - Development of Gene 

Therapy for Wiskott-Aldrich Syndrome 

DINNER BREAK – 6:45 PM - 8:30 PM

28 




EDUCATION SESSION III – 8:30 PM - 10:00 PM 


Novel Vector Systems 

Novel adenovirus and adeno-associated virus vectors have been 

developed recently, which enable the delivery of transgenes into 

target cells and tissues with efficiency and specificity. Application of 

these novel vectors in the delivery of therapeutic genes to treat 

inherited disorders as well as acquired diseases like cancer will be 

discussed. 

Room: 260/264 

Chair: Savio L.C. Woo, PhD 

Speakers: Andre Lieber, MD, PhD - Gene Transfer Vectors 

Based on B-group Adenoviruses 

David T. Curiel, MD, PhD - Targetable/ 

Injectable Adenoviral Vectors for Cell-specific Gene 

Transfer In Vivo 

Guangping Gao, PhD - Efficient Gene Transfer 

Vectors Based on Novel Primate AAVs 


AAV Vectors 

The goal of the AAV Education Session is to provide the 

community not only with a general understanding of this popular 

vector system, but to integrate this into a broader awareness of 

expectations when using these vectors in vitro and in vivo. Three 

topics will be discussed in this session. The first speaker will begin 

with a general background of AAV biology including a discussion 

of the molecular fate of recombinant genomes in vitro and in 

vivo. The second topic will concentrate on structural differences 

between AAV serotypes and how those differences can be utilized 

to improve the vector delivery. The final topic will focus on 

applications of AAV serotype delivery to specific target organs 

including performance comparisons between different serotypes. 

Room: 263/267 

Chair: Joseph Rabinowitz, PhD 

Speakers: Joseph Rabinowitz, PhD - Exploring the 

Structure and Function of the AAV Virion 

Xiao Xiao, PhD - Recent Progress in AAV Vector 

Development 

K. Reed Clark, PhD - Wild-type and 

Recombinant AAV Biology - An Update 


RNAi 

RNA interference refers to a cells natural ability to silence gene 

expression using short interfering RNAs (siRNAs). Inhibitory 

RNAs are derived from larger precursors transcribed from the 

genome, or introduced into cells artificially. The power of RNAi 

has recently been harnessed by the bench scientist to selectively 

inhibit the expression of diverse gene products to ascertain 

biological function, or as therapeutic tools for silencing pathogenic 

protein expression. This session will provide an overview of the 

protein complexes involved in RNAi, and will also illustrate 

various methods to accomplish RNAi in cells, tissues and animals. 


Chair: Beverly L. Davidson, PhD 

Speakers: Beverly L. Davidson, PhD - RNA Interference 

John J. Rossi, PhD - The Long and Short of 

RNAi 

Michael McManus, PhD - Developmental and 

Post-developmental Phenotypes of MicroRNA 

Ablation in the Mouse 


Gene Transfer to the Central Nervous System 

In this session the three speakers will address specific issues 

pertaining to gene transfer in the nervous system. The broad goals 

are to convey to the audience the anatomic, immunologic and 

development aspects of the nervous system that are unique as a 

target for gene therapy. Dr. Fink will highlight the peripheral 

nervous system development, anatomy, function and disease 

related dysfunctions. Dr. Maria Castro will address neural 

immune interactions, locus of antigen presentation and 

consequences for gene transfer. Dr. Federoff will touch on special 

problems encountered in the translation of preclinical gene therapy. 

He will also briefly highlight issues related to the development of 

in utero CNS gene therapy. 

Room: 230/231 

Chair: Howard J. Federoff, MD, PhD 

Speakers: Howard J. Federoff, MD, PhD - Translational 

Considerations for CNS Gene Therapy 

David J. Fink, MD - Gene Transfer to DRG for 

Treatment of Neuropathy or Chronic Pain 

Maria Castro, PhD - Regulated, Long Term 

Therapeutic Gene Expression for Neurological 

Gene Therapy





Bioavailability and Pharmacokinetics 

Pharmacokinetic analysis translates the disposition characteristics 

of macromolecules into quantitative parameters that can be used for 

direct comparison of medicinal agents as well as with physiological 

parameters such as blood flow and the rate of cellular endocytosis. 

Careful collection and experimental analysis of biological samples 

as well as theoretical development of appropriate mathematical 

models are necessary for accurate prediction of drug disposition and 

comparison of treatment regimens. This session, designed for the 

bench scientist and the clinician alike, will discuss basic 

pharmacokinetic concepts, methods for modeling, study design, 

data analysis, population kinetics and their application to gene 

delivery systems. 

Room: 265/266 

Chair: Maria A. Croyle, PhD 

Speakers: Maria A. Croyle, PhD - Pharmacokinetics Made 

Easy 

Sally Choe, PhD - Bridging Preclinical and 

Clinical Pharmacokinetics in Gene Therapy 


Gene-Based Vaccines 

This session will give the fundamentals as well as updates about 

Gene-based vaccines. The technology, immune mechanisms, and 

second generation advances describing new delivery systems and 

the use of adjuvants will be presented. An overview of clinical and 

research applications will be described. 

Room: 120/124 

Chair: Margaret A. Liu, MD 

Speakers: Margaret A. Liu, MD - Gene-based Vaccines: An 

Overview 

Jeffrey Ulmer, PhD - Toward the Development 

of an Effective DNA Vaccine for Humans 

Hildegund CJ Ertl, MD - Adaptive Immune 

Responses to Viral Vectors 

THURSDAY, JUNE 2, 2005 

GEORGE STAMATOYANNOPOULOS 

LECTURE 200 

8:00 am - 9:00 am 

Room: America’s Ballroom 

Chair: 


Speaker: 

Thomas Tuschl, PhD - 

Mechanisms of siRNA- and 

miRNA-guided Gene 

Regulation in Mammals 

Presentation of Excellence in Research Awards 

BREAK – 9:00 AM - 9:15 AM 

WORKSHOPS – 9:15 AM - 10:45 AM 

Workshop 210 

Cancer: Optimization of T-Cell Gene Therapy 

Room: 123/127 

Chair: Laurence JN Cooper, MD, PhD 

Speakers: John C. Morris, MD - Role of Interleutem-15 in 

T cell Development and Function 

Laurence JN Cooper, MD, PhD - Redirected 

Specifity of T cells - Improving Their Potential 

Steven M. Albelda, MD - Augmenting T-cell 

Trafficking to Tumors 

Bruce L. Levine, PhD - Cancer: Optimization 

of T-Cell Gene Therapy 

Francesco M. Marincola, MD - Gene Profiling 

of Immune Responses Against Tumors 

Carolyn Wilson, PhD - T-cell Gene Therapy: 

Regulatory Issues

30 




Workshop 211 

Gene-Based Vaccines: New Developments for Genetic 

Vaccines 

Room: 120/124 

Chair: David B. Weiner, PhD 

Speakers: Richard Heller, PhD - Electroporation Mediated 

Immunotherapy of Solid Tumors: Preclinical 

Results and Initiation of Clinical Study 

Leaf Huang, PhD - LPD Nanoparticles: A 

Gene Vector Turned into a Potent Vaccine Carrier 

David B. Weiner, PhD - Expanding the Potency 

and Breadth of the Immune Responses Induced by 

DNA Vaccines 

Britta Elisabeth Wahren, MD, PhD - Means to 

Induce Mucosal Immunity in HIV Genetic 

Immunization 

Ning-Sun Yang, PhD - Developing Multiple 

Viral Protein Genes and Virus-like Particles as 

Gene-based Vaccines 

Workshop 212 

Hemopoietic: Hemopoietic Stem Cell Gene Therapy 

Room: Ferrara Theater 

Chair: Jan Nolta, PhD 

Speakers: Carolyn Wilson, PhD - Addressing Safety 

Concerns for Hematopoietic Stem Cell Gene 

Therapy - A FDA Perspective 

Manuel Grez, PhD - Correction of Chronic 

Granulomatous Disease by Gene Therapy 

Marina Cavazzana-Calvo, MD, PhD - 

Clinical and Biological Results from the SCID-XI 

Gene Therapy Trial 

Stanton L. Gerson, MD - In Vivo Stem Cell 

Selection without Myeloablation 

Alessandro Aiuti, MD, PhD - Gene Therapy of 

ADA-deficient SCID 

Workshop 213 

Immunology of Gene Therapy: Organ 

Specific Immunity 

Room: 260/264 

Chair: Roland W. Herzog, PhD 

Speakers: Pedro Lowenstein, MD, PhD - Immune 

Responses to Viral Vectors in the Central Nervous 

System 

John Iacomini, PhD - Reshaping the Immune 

System Using Gene Therapy 

Roland W. Herzog, PhD - In Vivo Gene 

Transfer to Liver and Muscle; Two Organs, Two 

Different Stories 

Hans-Peter Kiem, MD - Immune Responses to 

Gene-modified Hematopoietic Cells 

Workshop 214 

Industrial Liaison: Pre-Clinical Issues in Gene Therapy 

Room: 263/267 

Chair: Douglas J. Jolly, PhD 

Speakers: Rebecca Sheets, PhD - Pre-clinical 

Development of Adenovirus-vectored HIV Vaccine 

Candidate 

Joy Cavagnaro, PhD, DABT, RAC - 

Optimizing Design and Analysis of Preclinical 

Development Programs 

Boro Dropulic, PhD - Clinical Translation of a 

Lentiviral Vector for the Treatment of HIV/AIDS 

Peter K. Working, PhD, DABT - Preclinical 

Strategies: Taking an “Armed” Oncolytic 

Adenovirus to the Clinic 

Workshop 215 

Programs of Excellence in Gene Therapy 

Room: 230/231 

Chair: Sonia I. Skarlatos, PhD 

Speakers: Sonia I. Skarlatos, PhD - PEGT Overview 

Haig H. Kazazian, Jr., MD - Preclinical Gene 

Therapy of Hemophilia A with New AAV 

Serotypes 

Ronald G. Crystal, MD - Gene therapy for 

Alpha 1-antitrypsin Deficiency with Intrapleural 

Administration of Novel AAV Serotypes 

Barry London, MD, PhD - Gene Therapy 

During LVAD Support: Can We Fix the Broken 

Heart? 

Stanley Riddell, MD - Regulating T cells for 

Immunotherapy by Gene Insertion 

Richard A. Knazek, MD - The National Gene 

Vector Laboratories - Production, Toxicology, 

Repository, Clonality Testing and Long-Term 

Follow Up 

Workshop 216 

Respiratory Tract: What’s New in Animal Models for 

Lung Gene Therapy 

Room: 265/266 

Chair: Stephen Hyde, PhD 

Speakers: Bruce Bunnell, PhD - Mouse or Monkey: What 

is the FDA Looking for in Pre-clinical Data? 

Alice F. Tarantal, PhD - Monkey Model for Fetal 

Intrapulmonary Gene Transfer 

John F. Engelhardt, PhD - Are Men Mice or 

Pigs? A Lung Gene Therapy and Cell Biology 

Perspective 

Uta Griesenbach, PhD - FABp-CF Knockout 

Mice as Models for CFTR Gene Transfer 

Gerry McLachlan, PhD - Evaluation Non-viral 

Gene Transfer in the Ovine Lung




Workshop 217 

Viral: Key Design Issues for Oncolytic Vectors 


Chair: Samuel D. Rabkin, PhD 

Speakers: David L. Bartlett, MD - Options for Creating 

Mutant Poxviruses for Oncolytic Therapy 

Noriyuki Kasahara, MD, PhD - Building a 

Better Mouse Virus: Design Strategies to Improve 

Therapeutic Efficacy and Safety of Replicationcompetent 

Retrovirus (RCR) Vectors for Cancer 


John C. Bell, PhD - VSV as an Oncolytic Agent 

Stephen J. Russell, MD, PhD - Retargeting 

Attachment and Entry of Oncolytic Viruses 

William S. M. Wold, PhD - Oncolytic 

Adenovirus Vectors that Overexpress ADP 

Samuel D. Rabkin, PhD - Oncolytic Herpes 

Simplex Virus (HSV) Vectors 


WORKSHOPS – 11:00 AM - 12:30 PM 

Workshop 220 

Cardiovascular Gene Therapy: From Bench to Bedside 

Room: 120/124 

Co-Chairs: Anthony Rosenzweig, MD 

Roger J. Hajjar, MD 

Speakers: Albert J. Sinusas, MD - Imaging of Angiogenesis 

and Gene Therapy In Vivo 

R. Jude Samulski, PhD - Biological 

Nanoparticles: The Next Generation of Efficient 

Delivery Reagents 

David Kaye, FRACP, PhD, FACC - 

Percutaneous Approaches to Cardiac Gene Transfer 

Roger J. Hajjar, MD - The Road from 

Physiological Observation to Gene Therapy in 

Patients with Heart Failure 

Workshop 221 

Clinical and Regulatory Affairs: Cell and Gene Based 

Cancer Vaccines 

Room: 263/267 

Chair: Dale Ando, MD 

Speakers: Karin Jooss, PhD - Combination Therapy of a 

GM-CSF Secreting Tumor Cell Vaccine and 

anti-CTLA-4: Preclinical Evaluation and Tox 

Studies 

Drew Pardoll, MD, PhD - Targeting Immune 

Responses to Sites of Cancer Using Engineered 

Viruses and Bacteria 

Stephen J. Russell, MD, PhD - Noninvasive 

Expression Monitoring of Oncolytic Viruses in 

Phase I Clinical Trials 

Workshop 222 

Genetic Diseases: Focus on the Metabolic Diseases of 

the Liver 

Room: 265/266 

Chair: Jayanta Roy-Chowdhury, MD 

Speakers: Markus Grompe, MD - Strategies to Select 

Hepatocytes In Vivo 

Cary Harding, MD - Gene and Cell-mediated 

Therapeutic Approaches to Phenyckeonuria 

Jayanta Roy-Chowdhury, MD - SV40-based 

Vectors for Metabolic Diseases of the Liver and 

Beyond 

Brendan Lee, MD, PhD - Helper-dependent 

Adenoviral Gene Therapy for Metabolic Liver 

Diseases 

Workshop 223 

Infectious Disease: Innate Immunity and Vector 

Function 

Room: 123/127 

Co-Chairs: Bruce L. Levine, PhD 


Speakers: Hildegund CJ Ertl, MD - Innate Immunity to 

Adenovirus 

Bryan Williams, PhD - Synergistic Activation of 

Innate Immunity by Double-stranded RNA and 

CpG DNA 

Bruce E. Torbett, PhD, MSPH - Are 

Viral-based Vectors and Their Payloads Seen as 

Pathogens? 

Hua Yu, PhD - Stat3 Targeting and Immunity

32 




Workshop 224 

Musculo-Skeletal: Myogenic Stem Cells and 

Regeneration 

Room: 230/231 

Co-Chairs: Jeffrey Chamberlain, PhD 

Shin’ichi Takeda, MD, PhD 

Speakers: Shin’ichi Takeda, MD, PhD - Contribution of 

CD31-negative/CD45-negative Side Population 

Cells to Skeletal Muscle Regeneration 

Morayma Reyes, MD, PhD - Muscle 

Multipotent Adult Progenitor Cells (MAPC): 

Their Origin, Role and Clinical Potential in 

Muscle Disease 

Stephen D. Hauschka, PhD - Regulatory 

Cassette Design for Optimal Expression in Skeletal 

and Cardiac Muscle Following In Vivo and Ex 

Vivo Gene Delivery 

Thomas A. Rando, MD, PhD - Molecular 

Control of Muscle Stem Cell Activity 

Johnny Huard, PhD - Gene Therapy Based on 

Muscle-derived Stem Cells: Potential for Tissue 

Regeneration and Repair 

Emanuela Gussoni, PhD - Human Muscle 

Progenitor Cell Quiescence Maintained by Elevated 

BMP4 Expression 

Workshop 225 

Neural Disorders: Gene Therapy and Behavior 


Chair: Pedro Lowenstein, MD, PhD 

Speakers: Mary Bartlett Bunge, PhD - Using Gene 

Therapy to Improve Repair of the Injured Spinal 

Cord 

William J. Bowers, PhD - Functional 

Assessment of Gene-based A² Immunotherapy in a 

Mouse Model of Alzheimer’s Disease 

James B. Uney, PhD - Practical Approaches to 

Using Viral Vectors Provisional to Study the 

Molecular Basis of Behavioral Function 

Michael Kaplitt, MD, PhD - Gene Therapy to 

Alter Sexual Behavior 

Xandra Breakefield, PhD - HSV Amplicon 

Vector Delivery of Gonadotropin Releasing 

Hormone Gene (13 kb) to Hypothalamus Restores 

Estrous Activity in Hypogonadal Mice 

Workshop 226 

Nonviral: New Approaches in Nonviral Gene Delivery 

Room: 260/264 

Co-Chairs: Stephen Hart, PhD 

Leaf Huang, PhD 

Speakers: Esther H. Chang, PhD - Tumor Targeting 

Nanodelivery Systems: Expanding the Potential for 

Cancer Therapy and Diagnosis 

Mark E. Davis, PhD - A Systems Approach to 

the Design and Development of Non-Viral 

Delivery Systems 

Muna Naash, PhD - DNA Nanoparticles for 

Non-Viral Ocular Gene Transfer 

Lonnie D. Shea, PhD - Controlled Release 

Systems for Non-viral Vectors 

Alexander V. Kabanov, PhD - Transcriptional 

Activation of Gene Expression by Pluronic 

Ian MacLachlan, PhD - Clinical Pharmacology 

of Stable Plasmid Lipid Particles for Systemic 


Workshop 227 

Special: The Clinical Trial Data on adp53 Gene 

Therapy of Cancer 


Co-Chairs: James S. Norris, PhD 

Helen E. Heslop, MD 

Speakers: Jack A. Roth, MD - Adp53 Clinical Trials 

Dmitry Gabrilovich, MD, PhD - 

Immunotherapy of Cancer with Dendritic Cell 

Based p53 Vaccine 

Yongsong Guan, MD - Transcatheter Hepatic 

Arterial Chemoembolization and Gendicine 

Combined in Treatment of Advanced Hepatocellular 

Carcinoma(HCC) 

Workshop 228 

Special: Skin Gene Therapy 

Room: 261/262 

Chair: Ulrich R. Hengge, MD 

Speaker: Ulrich R. Hengge, MD - Naked DNA: From 

Basics to Application 

Ernst Wagner, PhD - Bioresponsive Deshielding 

of Targeted DNA Polyplexes 

Lorne Taichman, MD, MSc, PhD - Long-term 

Systemic Delivery From Genetically Modified 

Human Skin 

MEET-THE-INVESTIGATOR LUNCH 

SESSIONS – 12:45 PM - 1:45 PM 

Meet-the-Investigator 230 

Room: Washington Avenue A 

Chair: Jeffrey Chamberlain, PhD - Systemic Delivery 

of Genes to Muscle Using rAAV





Room: Washington Avenue B 

Chair: Carl H. June, MD - Translational Lessons on 

T Cell Gene Transfer 


Room: Washington Avenue C 

Chair: Donald B. Kohn, MD - Gene Therapy Using 

Hematopoietic Stem Cells 


Room: Washington Avenue E 

Chair: Gerry McLachlan, PhD - Can Animal Models 

Predict Clinical Efficacy of Respiratory Gene 

Transfer? 


Room: Washington Avenue F 

Chair: Maria Grazia Roncarolo, MD - Is Immune 

Response to Transgenes a Major Hurdle to 

Successful Gene Therapy? 

SIMULTANEOUS ORAL ABSTRACT 


Oral Abstract Session 240 

RNA Virus Vectors: Gene Transfer and Gene 

Expression 

Room: 260/264 

Co-Chairs: Stephen J. Russell, MD, PhD 


(Abstracts #1 - #10) 


AAV Vectors: Vector Biology 


Co-Chairs: Hiroyuki Nakai, MD, PhD 




Adenovirus Vectors: Vector Technology 


Co-Chairs: Kohnosuke Mitani, PhD 




Inborn Errors of Metabolism - Stem Cells and Blood 

Disorders 

Room: 120/124 

Co-Chairs: Barry J. Byrne, MD PhD 




Cardiac Gene Transfer 

Room: 123/127 

Chair: Federica Del Monte, MD, PhD 



Neurodegenerative Diseases 

Room: 263/267 

Co-Chairs: David J. Fink, MD 




Gene Therapy for Infections and Vaccines 

Room: 230/231 

Co-Chairs: Boro Dropulic, PhD 




Cancer-Targeted Gene Therapy: Non Adenoviral 

Targeting of Vectors and Non Viral Vectors 

Room: 265/266 

Co-Chairs: Ronald G. Crystal, MD 




Gene Regulation: Keeping Genes On and Turning 

Them Off 

Room: 261/262 

Co-Chairs: Punam Malik, MD 



POSTER SESSION I – 4:00 PM - 7:30 PM 

Hall 2 

RNA Virus Vectors: Gene Expression 

(Abstracts #89 through #109; and Abstract #822) 

AAV Vectors: Disease Applications 

(Abstracts #110 through #136) 

Adenovirus Vectors: General Biology and Host 

Response 


“Other” DNA Virus Vectors 


Naked DNA Gene Transfer: Chromosomal, Integration, 

Delivery Techniques 



(Abstracts #196 through #231)

34 




Inborn Errors of Metabolism: Systemic and Storage 

Diseases 


Muscle and Connective Tissue: Gene Therapy for 



Cancer - Immunotherapy: Genetically Modified 

Antigen Presenting Cells and Adjuncts 


Cancer-Apoptosis and Suicide 


Cancer - Targeted Gene Therapy: Targeting of 

Non-Viral Vectors 

(Abstracts #310 through #329; Abstract # 328 has been 

moved to Poster Session III) 

Hematologic - Disease Models 


Gene Therapy Approaches to Pulmonary Disease 


Gene Regulation: Gene Targeting and RNA 


FRIDAY, JUNE 3, 2005 

SCIENTIFIC SYMPOSIA – 8:00 AM - 10:00 AM 

Scientific Symposium 300 

Ethics: Ethical Issues in Clinical Trial Design: 

Informed Consent and Children 


Chair: R. Scott McIvor, PhD 

Speakers: Adrian J. Thrasher, MD, PhD - Clinical Trials 

of Gene Therapy for Severe Combined 

Immunodeficiency 

Robert M. Nelson, MD, PhD - Gene Transfer 

Trials in Children: What’s Ethical? What’s 

Allowable? 

Nancy M.P. King, JD - Children in Gene 

Transfer Trials: Design, Benefit and Consent Issues 

Panel: Punam Malik, MD 

Cynthia Dunbar, MD 


Gene-Based Vaccines: Cancer Vaccines 


Chair: Margaret A. Liu, MD 

Speakers: Kristen Hege, MD - GM-CSF Gene-modified 


Margaret A. Liu, MD - Clinical Gene-based 

Cancer Vaccines and Immunotherapy 

Malcolm K. Brenner, MD, PhD - Improving 

the Function and Feasibility of Gene Modified 


Dennis Panicali, PhD - Progress in the 

Development of Poxvirus-based Cancer Vaccines 


Genetic Diseases: Protein Modifications to Enhance 

Therapy of Genetic Disease 


Chair: Valder Arruda, MD, PhD 

Speakers: Jonathan B. Rothbard, PhD - Delivery of Small 

and Large Molecules into Cells and Tissues with 

Arginine Rich Transporters 

Robert H. Costa, PhD - Use of a Transducing 

p19ARF Peptide to Induce Apoptosis and 

Diminish Proliferation of Mouse Hepatocellular 

Carcinomas 

Valder Arruda, MD, PhD - Coagulation 

Proteins with Improved Biological Properties for 

Hemostasis Gene Therapy 

Nay Wei Soong, PhD - Seven Years in Tibet: 

Optimizing Therapeutic Proteins by DNA 

Shuffling





Infectious Disease: Gene Therapy for HIV: Lessons 

Learned, Paradigms to Apply to Other Areas 

Room: 260/264 

Co-Chairs: Boro Dropulic, PhD 


Speakers: Dale Ando, MD - Gene Modulation of the HIV 

Co-receptor CCR5 Using CCR5 Specific Zinc 

Finger Nucleases 

Dorothee Von Laer, MD - Treatment of HIV 

Infection with Gene Protected Autologous T cells 

Carl H. June, MD - Lentiviral Gene Therapy for 

HIV: A New Paradigm? 

John J. Rossi, PhD - RNAi Gene Therapy for 

HIV-1 


Musculo-Skeletal: Regenerative Approaches for Skeletal 

Disorders 

Room: 263/267 

Chair: Steven C. Ghivizzani, PhD 

Speakers: Christopher Evans, PhD - Tissue Regeneration 

Without Manufactured Scaffolds or Cell Culture 

Dan Gazit, DMD, PhD - Genetically 

Engineered Mesenchymal Stem Cells as a Platform 

for Skeletal Tissue Engineering 

Paul D. Robbins, PhD - Gene Therapy for Bone 

and Cartilage Regeneration 

Edward M. Schwarz, PhD - Revitalization of 

Structural Allografts via Immobilized rAAV Gene 



SCIENTIFIC SYMPOSIA – 10:15 AM - 12:15 PM 


Embryonic Stem Cells and Tissue Engineering 


Chair: Carolyn Lutzko, PhD 

Speakers: Linda G. Griffith, PhD - Microscale Models of 

Liver for Analysis of Gene Therapy 

Mervin Yoder, MD - Using RNA Interference 

Approaches to Modulate Gene Expression in ES 

Cells 

Kyunghee Choi, PhD - Stepwise Commitment 

from Embryonic Stem to Hematopoietic and 

Endothelial Cells 

Michael R. Linden, PhD - AAV-mediated Site- 

Specific Gene Addition in Embryonic Stem Cells 


Immunology of Gene Therapy: Immune Response to 

Gene Transfer Vehicles 


Chair: Hildegund CJ Ertl, MD 

Speakers: James M. Wilson, MD, PhD - T Cell 

Responses to Adenoviruses and AAV 

Katherine A. High, MD - Immune Responses to 

Vector and Transgene Product in AAV-mediated 

Gene Transfer 

Thierry C. Vandendriessche, PhD - 

Transduction of Antigen-Presenting Cells using 

Viral Vectors: A Double Edged Sword 


Neural Disorders: Inherited Metabolic Brain Disorders 


Chair: Beverly L. Davidson, PhD 

Speakers: Luigi Naldini, MD, PhD - HSC-based Gene 

Therapy of Leukodystrophies 

John H. Wolfe, VMD, PhD - Gene Therapy for 

the CNS in Lysosomal Storage Disease 

Paola Leone, PhD - Safety and Efficacy of 

AAV-mediated Gene Transfer for Canavan Disease 

Ronald G. Crystal, MD - AAV-mediated Gene 

Transfer for Batten Disease 


Nonviral: Nonviral Gene Delivery: Towards Clinical 

Applications 

Room: 260/264 

Chair: Dexi Liu, PhD 

Speakers: Lluis M. Mir, DSc - Electric Pulses-based Gene 

Transfer: Safety and Efficacy of Combinations of 

High Voltage and Low Voltage Pulses 

Hans J. Lipps, PhD - A Novel Non-viral 

Episomal Vector as Expression System for Gene 


Michael A. Barry, PhD - Endonucleases as 

Barriers to Gene Delivery 

William M. Pardridge, MD - Intravenous, 

Non-Viral Gene Therapy of Brain in Rodents and 

Primates

36 





Respiratory Tract: Respiratory Epithelium as a Model 

for Mucosal Gene Delivery 

Room: 263/267 

Co-Chairs: Daniel Weiss, MD, PhD 

Patrick L. Sinn, PhD 

Speakers: Stephen Hyde, PhD - Non-viral Vectors for 

Airway Epithelium: The Pros and Cons 

Larry G. Johnson, MD - Cell Biological 

Considerations in Gene Transfer to Respiratory 

Epithelia 

Jim Hu, PhD - Viral Vectors for Airway Gene 

Delivery: Pros and Cons 

Adriana Heguy, PhD - Microarray Analysis of 

Gene Expression in the Human Small and Large 

Airways to Identify Targets for Lung Disease 



Meet- the-Investigator 320 


Chair: Kenneth Cornetta, MD - Integrating Vectors: 

From Production to Clinic 



Chair: Cynthia Dunbar, MD - Insertional Mutagenesis 

and Retroviral Vectors 



Chair: Adrian J. Thrasher, MD, PhD - Clinical Trials 

for Immunodeficiency 


Room: Washington Avenue F 

Chair: John Tisdale, MD - Hematopoietic Stem Cell 



SESSIONS – 2:00 PM – 4:30 PM 


RNA Virus Vectors: Integration and Vector Safety 


Co-Chairs: Isabelle Riviere, PhD 


(Abstracts #386 - #395) 


AAV Vectors: Vector Development 


Co-Chairs: Saswati Chatterjee, PhD 

Guangping Gao, PhD 

(Abstracts #396 - #405) 


Adenovirus Vectors: Innate Immunity 

Room: 120/124 

Co-Chairs: Joseph T. Bruder, PhD 

Andrea Amalfitano, DO, PhD 

(Abstracts #406 - #415) 


Inborn Errors of Metabolism - Liver and Storage 

Disorders 

Room: 260/264 

Co-Chairs: Seng H. Cheng, PhD 


(Abstracts #416 - #425) 


Neurologic Disorders 

Room: 265/266 

Chair: Pedro Lowenstein, MD, PhD 

(Abstracts #426 - #435) 


Infectious Diseases and Vaccines: RNAi in Gene 

Therapy for Infections 

Room: 230/231 

Chair: Bruce E. Torbett, PhD, MSPH 

(Abstracts #436 - #443) 


Targeting of Adeno Viral Vectors and Cancer Treatment 

Room: 123/127 

Co-Chairs: Estuardo Aguilar-Cordova, PhD 


(Abstracts #444 - #453) 


Advances in Hematologic Gene Therapy 

Room: 263/267 

Co-Chairs: Karen E. Pollok, PhD 


(Abstracts #454 - #463 


Gene Regulation: Regulated Systems and Cell 

Engineering 

Room: 261/262 

Co-Chairs: David Spencer, PhD 

James M. Wilson, MD, PhD 

(Abstracts #464 - #473)




POSTER SESSION II – 4:00 PM - 7:30 PM 

Hall 2 

RNA Virus Vectors: Gene Transfer, Vector Production 




Adenovirus Vectors: Applications 


Naked DNA Gene Transfer: Optimizing Expression, 

Animal Models 


Lipid Mediated Gene Transfer: Non-Viral Delivery 

System 

(Abstracts # 576 through #596) 

Inborn Errors of Metabolism: Hemophilia 


Cardiovascular: Angiogenesis and Vascular Gene 



Neurologic: Pain 


Neurologic: Spinal Cord Injury and Motor Neuron 

Disease 


Neurologic: Advances in Vectors, Delivery and Imaging 


Neurologic: Sensory 


Gene Therapy for Connective Tissue 


Cancer - Immunotherapy: Cytokine Transgenes and 

Vaccinations 



Non-Adenoviral Vectors 


Gene Regulation: Overcoming Obstacles to Gene 

Transfer and Expression 


EVENING SESSION – 8:30 PM – 10:00 PM 

Evening Session 340 

Immune Responses in Neurologic Gene Therapy - Basic 

Bench Perspectives 

Target Audience: Anyone interested in immune responses as well 

as neural gene therapy in the brain. 

Room: 261/262 

Co-Chairs: Pedro Lowenstein, MD, PhD 

Hildegund CJ Ertl, MD 

Speakers: Ronald G. Crystal, MD 

Paola Leone, PhD 

James Wilson, MD, PhD 

Michael Kaplitt, MD, PhD 

John Iacomini, PhD 



Samuel D. Rabkin, PhD 

Jean Bennett, MD, PhD 

David J. Fink, MD 

Ronald Mandel, PhD

38 



SATURDAY, JUNE 4, 2005 

SCIENTIFIC SYMPOSIA – 8:00 AM - 10:00 AM 


Cancer: Clinical Trials 


Chair: Steven M. Albelda, MD 

Speakers: Daniel H. Sterman, MD - Gene Therapy 

Clinical Trials for Pleural Malignancies 

John J. Nemunaitis, MD - Oncolytic Viral 

Gene Delivery Vehicles 

Chiara Bonini, MD - Suicide Gene Therapy in 

Stem Cell Transplantation 

Kristen Hege, MD - GVAX Cancer Vaccines - 

Clinical Development 


Cardiovascular: Advances in Cardiovascular Cell and 



Co-Chairs: Lawrence C. Chan, MD 

Douglas Losordo, MD 

Speakers: Lawrence C. Chan, MD - Novel Therapy of 

Obesity: Implications for Cardiovascular Disease 

Douglas Losordo, MD - Adult Stem Cells: Fact 

or Fiction 

H. Kirk Hammond, MD - Adenylyl Cyclase 

Gene Transfer for Heart Failure 


Hemopoietic: Advances in Stem Cell Self Renewal 

Room: 260/264 

Co-Chairs: David W. Emery, PhD 


Speakers: R. Keith Humphries, MD, PhD - Emerging 

Strategies for High Level Expansion of 


C. Anthony Blau, MD - Pharmacologically 

Regulated Cell Therapy 

Akihiro Kume, MD, PhD - On-demand 

Expansion of Genetically Corrected Blood Cells 


Oligonucleotide Based Therapies: Emerging 

Applications of Oligonucleotide Therapeutics 


Chair: Dieter Gruenert, PhD 

Speakers: Bruce Sullenger, PhD - Aptamer-Antidote Pairs 

as a Novel Approach to Safer Drug Design 

Mark A. Kay, MD, PhD - RNAi Based 

Approaches for Treatment of Human Hepatitis 

Viral Infection 

Peter Glazer, MD, PhD - Targeted Gene 

Correction via Triplex Formation 

Andrew Ellington, PhD - Aptamer Inhibition of 

HIV 


Viral: Vector Cell Interactions 

Room: 263/267 

Chair: Kenneth Cornetta, PhD 

Speakers: Glen R. Nemerow, PhD - Adenovirus Cell 

Entry Mechanisms 

Arun Srivastava, PhD - AAV-mediated 

Transgene Expression: Second-strand Synthesis 

Versus Strand-annealing 

Nicholas Muzyczka, PhD - Targeting AAV 

Vectors 



SESSIONS – 10:15 AM - 12:15 PM 


DNA Viruses: Vaccines, HSV, Baculovirus and SV40 


Co-Chairs: David Krisky, MD, PhD 


(Abstracts #755 - #762) 


Naked DNA Gene Transfer: Optimizing Expression/ 

Animal Models 

Room: 260/264 

Co-Chairs: Ronald K. Scheule, PhD 


(Abstracts #763 - #770) 


Lipid Mediated Gene Transfer: Non Viral Delivery 

Systems 

Room: 263/267 

Co-Chairs: Ralph W. Paul, PhD 


(Abstracts #771 - #778)





Molecular Conjugates: Therapeutic Applications 

Room: 120/124 

Co-Chairs: Lonnie D. Shea, PhD 


(Abstracts #779 - #786) 




Room: 230/231 

Co-Chairs: Olivier Danos, PhD 

Paula Clemens, MD 

(Abstracts #787 - #794) 


Cellular Immunotherapy of Cancer Using Genetically 

Modified Effectors 


Chair: Michael Jensen, MD 

(Abstracts #795 - #802) 



Room: 123/127 

Co-Chairs: Sunil Chada, PhD 


(Abstracts #803 - #810) 



Room: 261/262 

Co-Chairs: Terence R. Flotte, MD 

Christine L. Halbert, PhD 

(Abstracts #811 - #818) 





Chair: Harry L. Malech, MD - Ex Vivo Hematopoietic 

Stem Cell Therapy for Inherited Immune Disease 



Chair: R. Scott McIvor, PhD - Drug Resistance and 

Transposons in Gene Therapy Research 



Chair: Luigi Naldini, MD, PhD - Lentiviral Vectors for 

Stem Cell Gene Transfer 

PRESIDENTIAL SYMPOSIUM 430 

1:45 pm - 4:15 pm 


Chair: 


Speaker: 

George Q. Daley, MD, PhD 

Presidential Address: Katherine A. High, MD 

Speaker: George Q. Daley, MD, PhD - Combining 

Gene Therapy with Cell Therapy 

Presentation of Top Three Abstracts 

#819 – Alessandra Recchia, PhD - Molecular 

Follow-Up of Patients Treated with Allogeneic 

Hematopoietic Stem Cell Transplantation and Donor 

Lymphocytes Transduced with a Retroviral Vector 

Expressing HSV-TK and ∆LNGFR 

#820 – Aurelie Goyenvalle - Highly Efficient 

Exon-Skipping and Sustained Correction of Muscular 

Dystrophy Using an Adeno-Associated Viral Vector 

#821 – Anja Ehrhardt, PhD - Development of a 

Site-Specific Integrating Gene-Deleted Adenoviral Vector 

for Sustained Transgene Expression Levels In Vivo

40 




POSTER SESSION III – 4:00 PM - 7:00 PM 

Hall 2 

RNA Virus Vectors: Safety, Therapeutics 

(Abstracts #823 through #846; Abstract #822 has been 

moved to Poster Session I) 



Adenovirus Vectors: Vector Technologies 


Inborn Errors of Metabolism: Liver and Pancreatic 

Diseases 


Cardiovascular: Myocardial Gene Therapy 

(Abstracts #916 through #938; Abstract #921 has been 

moved to Poster Session II) 



Neurologic: Other 




Infectious Diseases and Vaccines: RNAi for Gene 

Therapy of Infections 





Hematologic - New Advances 


Gene Regulation: Regulated Systems and Tissue 

Specific Expression 


SUNDAY, JUNE 5, 2005 

SCIENTIFIC SYMPOSIUM – 8:30 AM - 10:00 AM 


Young Investigators 


Co-Chairs: Joseph C. Glorioso, PhD 

David Bodine, PhD 

Speakers: Chiara Bonini, MD - Gene Transfer into 

Peripheral Blood T Lymphocytes: Clinical Benefits 

and Safety Profile 

Introduced by Fabio Candotti, MD 

Michael Kaplitt, MD, PhD - Development of 

Human Gene Therapy for Neurodegenerative 

Disorders 

Introduced by Ronald G. Crystal, MD 


SESSIONS – 10:15 AM - 12:15 PM 


Clinical Gene Therapy 


Co-Chairs: Arthur Nienhuis, MD 


(Abstracts #1074 - #1079) 



Room: 260/264 

Co-Chairs: Roland W. Herzog, PhD 

Xiao Xiao, PhD 

(Abstracts #1080 - #1087) 


Adenovirus Vectors: Adaptive Immunity and Vaccines 

Room: 120/124 

Co-Chairs: Victor Krasnykh, PhD 

Robin J. Parks, PhD 

(Abstracts #1088 - # 1095) 


Naked DNA Gene Transfer: Chromosomal Integration 

Room: 263/267 

Co-Chairs: R. Scott McIvor, PhD 

Cary Harding, MD 

(Abstracts #1096 - #1103) 


Molecular Conjugates: Vector Engineering 

Room: 265/266 

Co-Chairs: Suzie H. Pun, PhD 


(Abstracts #1104 - #1111)



SUNDAY, JUNE 5, 2005 




Room: 261/262 

Chair: Andrew H. Baker, PhD 

(Abstracts #1112 - #1119) 



Connective Tissue 

Room: 230/231 

Co-Chairs: Paul D. Robbins, PhD 


(Abstracts #1120 - #1127) 

Oral Abstract 517 

Cancer - Immunotherapy: Genetic Strategies to Induce 

and Augment Anti-Tumor Immunity 

Room: 123/127 

Chair: Michael Jensen, MD 

(Abstracts #1128 - 1135) 

12:15 PM – ASGT 8 TH ANNUAL MEETING 

CONCLUDES 

See you in 2006! 

ASGT 9 th Annual Meeting 

May 31 - June 4, 2006 

Baltimore, Maryland

42 


AMERICA’S CENTER FLOOR PLAN 

LEVEL 1 

America’s Center Floor Plan 

Level One: 

· ASGT Registration 

· CME Sign In 

· Internet Kiosk 

· Education Sessions 

· Workshops 

· Scientific Symposia 

· Oral Abstract Sessions 

· Exhibits 

· Posters 

· Speaker Ready Room 

· Meet-the-Investigator Lunches


AMERICA’S CENTER FLOOR PLAN 

LEVEL 2 

America’s Center Floor Plan 

Level Two: 

· Plenary Sessions 

· Education Sessions 

· Workshops 

· Scientific Symposia 

· Oral Abstract Sessions 

· Press Room 

· Internet Kiosk

44 


HOUSING INFORMATION 

A RENAISSANCE GRAND HOTEL* 

Headquarters Hotel 

800 Washington Avenue 

St. Louis, Missouri 63101 

Phone: 314-621-9600 

B RENAISSANCE ST. LOUIS SUITES HOTEL 

827 Washington Avenue 

St. Louis, Missoiuri 63101 

Phone: 314-621-9700 

C HOLIDAY INN SELECT DOWNTOWN 

Ninth Street at Convention Center 

St Louis, Missouri 63101 

Phone: 314-421-4000


MAP OF ST. LOUIS

46 


ABSTRACTS 



RNA Virus Vectors: Gene Transfer and Gene 

Expression 

Room: 260/264 

1 2:00 PM 

Persistent Gene Expression in Mouse Nasal Epithelia 

Following Baculovirus GP64 Pseudotyped FIV-Based 


Patrick L. Sinn, Erin R. Burnight, Melissa A. Hickey, Gary W. 

Blissard, Paul B. McCray, Jr. 

2 2:15 PM 

Cellular Mechanisms Involved with Lentiviral Gene 

Transfer 

Shangming Zhang, Karen Pollok, Lakshmi Sastry, Lionel 

Berthoux, Jeremy Luban, Kenneth Cornetta 

3 2:30 PM 

Effects of the cHS4 Insulator Elements on Transgene 

Expression and Biological Titer of Lentiviral Vector 

Hideki Hanawa, Takashi Shimada 

4 2:45 PM 

Optimization of Globin Lentiviral Vector Design for the 

Treatment of bð-Thalassemia 

Leszek Lisowski, Stefano Rivella, Michel Sadelain 

5 3:00 PM 

Differential Expression of PIT1 and PIT2 after G-CSF 

Mobilization Is Associated with Efficient Gene Transfer 

Using GALV-Pseudotyped Gammaretroviral Vectors 

Brian C. Beard, Pau Mezquita, Julia C. Morris, Hans-Peter 

Kiem 

6 3:15 PM 

In Vivo Adult Stem Cell Gene Transfer in Mice by In Situ 

Delivery of a Self-Inactivating Lentiviral Vector Using 

Intrafemoral Injection 

D. Nicole Worsham, Kimberly Bohn, David Kuhel, David A. 

Williams, Christof von Kalle, Dao Pan 

7 3:30 PM 

Transduction of Quiescent Human Cells Using an 

SIVsmmPBj-Derived Lentiviral Vector 

Matthias Schweizer, Nina Wolfrum, Michael Muehlebach, Julia 

Kaiser, Silke Schuele, Klaus Cichutek 

8 3:45 PM 

Potent Inhibition of HIV-1 Replication by Lentiviral 

Vectors Carrying Anti-Vif siRNA 

S. Li, G. Wang, K. Meinking, M. J. Li, J. Ji, J. A. Zaia, J. J. 

Rossi 

9 4:00 PM 

Coronaviruses: Development of Novel Oncolytic Vectors 

Tom Würdinger, Hélène M. Verheije, Victor W. van Beusechem, 

Xander A. M. de Haan, Winald R. Gerritsen, Peter J. M. Rottier 

10 4:15 PM 

The Effect of Globin Locus Control Region (LCR) 

Elements or the MSCV-LTR on Promoter Trapping by 

Integrated Lentiviral Vector Genomes 

Byoung Y. Ryu, Derek A. Persons, Arthur W. Nienhuis 




11 2:00 PM 

An Additional Role of Cellular FKBP52 in Recombinant 

Adeno-Associated Virus 2 Vector-Mediated Gene 

Transfer and Transgene Expression 

Weihong Zhao, Li Zhong, Jianqing Wu, Liyuan Chen, Keyun 

Qing, Kirsten A. Weigek-Kelley, Steven H. Larsen, Arun 

Srivastava 

12 2:15 PM 

Self-Complementary AAV2 Vectors Transduce Liver with 

the Same Efficiency as AAV8: The Critical Role of 

Second-Strand Synthesis in AAV Biology 

Zhijian Wu, Hongzhe Duan, R. Jude Samulski 

13 2:30 PM 

Persistence of AAV Capsid in Transduced Cells 

Mo-Ying Hsieh, Fayaz R. Khazi, Alexander Schlachterman, Yi- 

Lin Liu, Katherine A. High 

14 2:45 PM 

rAAV2 Integration Junctions Isolated without In Vivo 

Selection Bias Show a Preference to 5’ Regions of Open 

Reading Frames 

Fayaz R. Khazi, Alexander Schlachterman, Katherine A. High 

15 3:00 PM 

Large-Scale Analysis of Adeno-Associated Virus Vector 

Integration Sites in Normal Human Cells 

Daniel G. Miller, Grant D. Trobridge, Lisa M. Petek, Michael 

A. Jacobs, Rajinder Kaul, David W. Russell 

16 3:15 PM 

Single-Polarity Recombinant Adeno-Associated Virus 2 

Vector-Mediated Transgene Expression In Vitro and In 

Vivo: Mechanism of Transduction 

Li Zhong, Xiaohuai Zhou, Yanjun Li, Keyun Qing, Richard J. 

Samulski, Arun Srivastava 

17 3:30 PM 

Visualization of the Intranuclear rAAV Replication 

Centers and Their Co-Localization with Double- 

Stranded DNA Break Repair Proteins 

Cervelli Tiziana, Lorena Zentilin, Alessandro Marcello, Mauro 

Giacca


ABSTRACTS 


18 3:45 PM 

Facilitation of AAV Genome Circularization by Host Cell 

Factors Involved in DNA Replication and Double-Strand 

Break Repair 

Vivian W. Choi, Douglas M. McCarty, Richard J. Samulski 

19 4:00 PM 

rAAV-2 Transduction of Primitive Human Hematopoietic 

Stem Cells Capable of Serial Engraftment in Immune 

Deficient Mice 

Helicia Paz, Leah Santat, Lijing Li, Christie Wong, Stephen J. 

Forman, K. K. Wong, Saswati Chatterjee 

20 4:15 PM 

Targeted Gene Delivery to Embryonic Stem Cells 

Els Henckaerts, Nadja Zeltner, Steven Kattman, Nathalie 

Dutheil, Peter Ward, Marion Kennedy, Nathalie Clement, 

Patricia Rebollo, Gordon Keller, R. Michael Linden 


Adenovirus Vectors: Vector Technology 


21 2:00 PM 

Application of Splice Acceptor Transposons to the 

Development of Oncolytic Viruses 

Fang Jin, Peter Kretschmer, Cecile Chartier, Irene Kuhn, Terry 

Hermiston 

22 2:15 PM 

Generation of Circular Sleeping Beauty DNA Transposition 

Substrates by RU486-Induced Flp Recombination In 

Vitro – Steps towards a Single Integrating Adeno- 

Transposon Vector 

Jacob G. Mikkelsen, Stephen R. Yant, Anja Ehrhardt, Mark A. 

Kay 

23 2:30 PM 

Frequency of Random and Targeted Chromosomal 

Integration of Helper-Dependent Adenoviral Vector 

Fumi Ohbayashi, Emi Aizawa, Atshuhiro Kishimoto, Ko 

Mitani 

24 2:45 PM 

Context-Specific Peptide-Presenting Phage Libraries for 

Adenoviral Vector Targeting 

Debadyuti Ghosh, Michael A. Barry 

25 3:00 PM 

Genetic Incorporation of HSV-1 Thymidine Kinase into 

the Adenovirus Protein IX for Functional Display on the 

Virion 

Jing Li, Long Le, Don A. Sibley, J. Michael Mathis, David T. 

Curiel 

26 3:15 PM 

Microsphere Delivery of Adenoviral-Liposome Complexes 

Reduces Adenoviral Immunogenicity and Allows 

Successful Re-Administration of the Virus without Loss 

of Gene Expression Efficiency 

Jason C. Steel, John C. Morris, Mark A. Burton, Heather M. 

A. Cavanagh, Wouter H. J. Kalle 

27 3:30 PM 

A Novel “Geneti-Chemical” Platform for Flexible and 

Efficient De- and Retargeting of Adenovirus Vector 

Particles 

Florian Kreppel, Judith Gackowski, Erika Schmidt, Stefan 

Kochanek 

28 3:45 PM 

Adenovirus Targeting Via Leucine Zipper Peptide- 

Mediated Ligand Attachment 

Joel N. Glasgow, Nikolay Korokhov, David T. Curiel 

29 4:00 PM 

Redirection of Adenoviral Tropism through Capsomers 

Other Than Fiber Is Generally Inefficient and Dependent 

on Ligand-Receptor Biology 

Samuel K. Campos, Michael A. Barry 

30 4:15 PM 

AdenoLibrary for Construction and Selection of Targeted 

Adenoviruses 

Sam C. Noureddini, Alexander Krendelshchikov, Vera 

Simonenko, Joanne T. Douglas, David T. Curiel, Nikolay 

Korokhov 


Inborn Errors of Metabolism - Stem Cells and Blood 

Disorders 

Room: 120/124 

31 2:00 PM 

Somatic Gene Therapy for ADA-SCID Following 

Cessation of PEG-ADA and Use of a Mild Conditioning 

Regime 

Hubert B. Gaspar, Emma Bjorkegren, Katherine Parsley, 

Kimberly C. Gilmour, Jo Sinclair, Fang Zhang, Lynette D. 

Fairbanks, Doug King, Graham Davies, Paul A. Veys Christine 

Kinnon, Adrian J. Thrasher 

32 2:15 PM 

Tolerance after Neonatal Gene Transfer of a Human 

Factor IX-Expressing Retroviral Vector May Involve 

Clonal Deletion in C3H Mice and Is Effective in Hemophilia 

B Dogs 

Lingfei Xu, Mark E. Haskins, Timothy C. Nichols, Manxue 

Mei, Patty O’Donnell, Dwight A. Bellinger, Stephanie 

McCorquodale, Katherine P. Ponder

48 


ABSTRACTS 


33 2:30 PM 

Non-Random Genomic Distribution of Retrovirus 

Vector Integration in Successful SCID-X1 Gene Therapy 

Kerstin Schwarzwaelder, Manfred Schmidt, Steven Howe, 

Hanno Glimm, Claudia Prinz, Manuela Wissler, Annette 

Deichmann, Sonja Schmidt, Bobby Gaspar, Adrian Thrasher 

Christof von Kalle 

34 2:45 PM 

Transposon-Based Gene Therapy of Hemophilia A 

Targeting Endothelial Cells in Neonates 

Li Liu, Bradley S. Fletcher 

35 3:00 PM 

Generalized Detoxification Associated with Engraftment 

of Gene-Corrected Repopulating Cells Achieved in 

ADA-SCID Patients by Stem Cell Gene Therapy without 

Myelopreparative Pre-Conditioning 

Makoto Otsu, Satoru Nakajima, Miyuki Kida, Yoshihiro 

Maeyama, Nariaki Toita, Norikazu Hatano, Nobuaki 

Kawamura, Motohiko Okano, Ryouji Kobayashi, Osamu 

Tatsuzawa Masafumi Onodera, Fabio Candotti, Michael S. 

Hershfield, Yukio Sakiyama, Tadashi Ariga 

36 3:15 PM 

Improved Lentiviral Vectors for Systemic Gene Transfer 

in the Absence of an Immune Response 

Brian D. Brown, Ehud Hauben, Angelo Lombardo, Lucia 

Sergi Sergi, Maria Grazia Roncarolo, Luigi Naldini 

37 3:30 PM 

A Novel Form of Enzyme Replacement Therapy for 

ADA-Deficiency: In Vivo Transduction by Neonatal 

Injection of Lentivirus Expressing ADA 

Denise A. Carbonaro, Xiangyang Jin, Denise Petersen, Donald 

B. Kohn 

38 3:45 PM 

Inhibition of Apolipoprotein B-100 Expression by 

Lentivirus Mediated RNA Interference in Mice after 

Stem Cell Transduction and Transplantation 

Mikko P. Turunen, Petri I. Makinen, Pia Leppanen, Juulia 

Enback, Suvi Koota, Tero Vatanen, Seppo Yla-Herttuala 

39 4:00 PM 

Effects of Long-Term Expression of Activated Murine 

FVII in Normal and Hemophilic Mice 

Majed N. Aljamali, Paris Margaritis, Rodney M. Camire, 

Katherine A. High 

40 4:15 PM 

Enhanced Factor IX Delivery from Bioengineered Hybrid 

Human Skeletal Muscle Co-Expressing VEGF 

Lieven Thorrez, Herman Vandenburgh, Desire Collen, Janet 

Shansky, Thierry VandenDriessche, Marinee Chuah 


Cardiac Gene Transfer 

Room: 123/127 

41 2:00 PM 

High Efficiency, Catheter-Based, Gene Transfer to the 

Large Animal Heart 

David M. Kaye, Kenneth Chien, Masahiko Hoshijima, 

Krisztina Zsebo, John Power 

42 2:15 PM 

Uniform Scale-Independent Gene Transfer to Striated 

Muscle after Transvenular Extravasation of Vector 

Kapil Gopal, Leonard T. Su, Zhonglin Wang, Xiaoqing Yin, 

Anthony Nelson, Benjamin W. Kozyak, James M. Burkman, 

Marilyn A. Mitchell, David W. Low, Charles R. Bridges 

Hansell H. Stedman 

43 2:30 PM 

Targeting the Biology of Heart Disease: Engineered Zinc 

Finger Protein Repressors of Phospholamban as a 

Potential Therapy for Congestive Heart Failure 

H. Steve Zhang, Lei Zhang, Yan Huang, Dinggang Liu, Yuxin 

Liang, Reed Hickey, Dmitry Guschin, Simon Chandler, Mike 

Kunis, Linda Hinh Danny Xia, Xiaohong Zhong, S. Kaye 

Spratt, J. Tyler Martin, Casey C. Case, Dale Ando, Edward J. 

Rebar, Philip D. Gregory, Frank Giordano 

44 2:45 PM 

Development of AAV-Mediated Gene Therapy for 

Murine Models of Genetic Diseases Affecting the Heart 

Christina A. Pacak, Cathryn Mah, Gabriel Gaidosh, Melissa 

Lewis, Raquel Torres, Kevin Campbell, Glenn A. Walter, Barry 

J. Byrne 

45 3:00 PM 

Adenoviral Gene Transfer of SERCA2a Restores Mechanical 

and Energetic Left-Ventricular Function in 

Spontaneously Diabetic Rats 

Susumu Sakata, Yuri Sakata, Elie R. Chemaly, Prabhu M. 

Padmanabhan, Djamel Lebeche, Miyako Takaki, Federica del 

Monte, Roger J. Hajjar 

46 3:15 PM 

In Vivo Adenoviral Gene Transfer of Activated PI3- 

Kinase Rescues Cardiac Dysfunction and Injury Induced 

by Ischemia-Reperfusion after Chronic Cardiac Akt 

Activation 

Tomohisa Nagoshi, Takashi Matsui, Takuma Aoyama, Ling Li, 

David A. Kass, Hunter C. Champion, Anthony Rosenzweig 

47 3:30 PM 

Ex Vivo Hypothermic Recirculatory Adenoviral Gene 

Transfer to the Transplanted Pig Heart 

Keiji Oi, William R. Davies, Henry D. Tazelaar, Kent R. 

Bailey, Mark J. Federspiel, Stephen J. Russell, Christopher G. 

A. McGregor


ABSTRACTS 


48 3:45 PM 

The Long-Term Effects of SERCA2a Overexpression in 

Large Animal Model of Heart Failure 

Yoshiaki Kawase, Ryuichi Yoneyama, Kozo Hoshino, Jennifer 

McGregor, Thanh-Thao Ton-Nu, Tom G. Neilan, Robert A. 

Levine, Judy Hung, Motoya Hayase, Federica del Monte Roger 

Hajjar 

49 4:00 PM 

Evaluation of Catheter-Based Deliveries of 10um Gelatin 

Hydrogel Microsphere, a Novel Non-Viral Vehicle, in a 

Porcine Heart 

Kozo Hoshino, Ryuichi Yoneyama, Yoshiaki Kawase, Alec M. 

De Grand, John V. Frangioni, Yasuhiko Tabata, Takeshi 

Kimura, Toru Kita, Roger J. Hajjar, Motoya Hayase 

50 4:15 PM 

Transcript Profiling Identifies Novel Role for SGK1 in 

Promoting Cardiomyocyte Survival 

Takuma Aoyama, Takashi Matsui, Mikhail Novikov, Anthony 

Rosenzweig 



Room: 263/267 

51 2:00 PM 

Adenoviral BDNF/Noggin-Induced Neurogenesis from 

Endogenous Neural Stem Cells Delays Motor Impairment 

and Extends Survival in a Transgenic Model of 

Huntington’s Disease 

Sung-Rae Cho, Eva Chmielnicki, Aris Economides, Steven A. 

Goldman 

52 2:15 PM 

AAV-Delivered RNAi Improves Cellular and Motor 

Phenotypes in a Mouse Model for Huntington’s Disease 

Scott Q. Harper, Patrick D. Staber, Xiaohua He, Ines H. 

Martins, Qinwen Mao, Henry L. Paulson, Robert M. Kotin, 

Beverly L. Davidson 

53 2:30 PM 

Post-Transcriptional Suppression of Striatal Mutant 

Huntingtin Leads to Mild Phenotypic Improvements in 

the R6/1 Mouse 

Edgardo Rodriguez, Eileen Denovan-Wright, Kevin Nash, 

Alfred S. Lewin, Ronald J. Mandel 

54 2:45 PM 

Lentiviral-Mediated Silencing of SOD1 through RNA 

Interference Delays Disease Onset and Progression in a 

Mouse Model of ALS 

Cedric Raoul, Patrick Aebischer 

55 3:00 PM 

Lentiviral-Mediated Silencing of Mutant SOD-1 Using 

RNAi Causes Long Term Correction of ALS in a 

Transgenic Mouse Model 

Gareth S. Ralph, Pippa A. Radcliffe, Denise M. Day, Janine M. 

Carthy, Susan M. Kingsman, Kyriacos A. Mitrophanous, 

Mimoun Azzouz, Nicholas D. Mazarakis 

56 3:15 PM 

Amelioration of Neurodegenerative and Neuropathological 

Alterations in a Transgenic Model of Alzheimer’s 

Disease: Targeting BACE1 with Small Interfering RNAs 

Oded Singer, Robert A. Marr, Edward Rockenstein, Leslie 

Crews, Fred H. Gage, Inder M. Verma, Eliezer Masliah 

57 3:30 PM 

Therapy for Alzheimer’s Disease by Neprilysin Gene 

Transfer 

Robert A. Marr, Edward Rockenstien, Atish Mukherjee, Mark 

S. Kindy, Louis B. Hersh, Fred H. Gage, Eliezer Masliah, 

Inder M. Verma 

58 3:45 PM 

Rett Syndrome Is Reversible and Treatable by MeCP2 

Gene Therapy into the Striatum in Mice 

Ken-ichiro Kosai, Akira Kusaga, Takeo Isagai, Koji Hirata, 

Satoshi Nagano, Yoshiteru Murofushi, Tomoyuki Takahashi, 

Sachio Takashima, Toyojiro Matsuishi 

59 4:00 PM 

rAAV-Mediated Nigral Parkin Over-Expression Is 

Neuroprotective in the 6-OHDA Rat Model of 

Parkinson’s Disease 

Fredric P. Manfredsson, Alfred S. Lewin, Nicholas Muzyczka, 

Corinna Burger, Ronald J. Mandel 

60 4:15 PM 

Recovery of Vestibular Function Induced by math1 Gene 

Delivery 

Hinrich Staecker, Mark Praetorius, Douglas E. Brough 



Room: 230/231 

61 2:00 PM 

Higher In Vivo Maintenance, Biodistribution and 

Immunopotency of Dendritic Cell Vaccines Self-Differentiated 

through Lentiviral Vector Programming 

Richard C. Koya, Takahiro Kimura, Antoni Ribas, Nori 

Kasahara, Renata Stripecke 

62 2:15 PM 

Transduction of CD34+ Hematopoietic Progenitor Cells 

with a SIV-Based Lentiviral Vector Expressing Antisense 

SIV Env Protects CD4+ T Cell Progeny from 

SIVmac239 Infection 

S. E. Braun, F. Eng, M. Connole, C. Chen, B. Dropulic, G. 

K. Binder, X. Lu, R. P. Johnson

50 


ABSTRACTS 


63 2:30 PM 

A Nonhuman Primate Model for Testing Immuno Gene 

Therapies for AIDS Using Gene-Protected T Cells 

Joern E. Schmitz, Felix Hermann, Sebastian Newrzela, Melisa 

Ricketts, Patricia Schult-Dietrich, Alexander Szyroki, Robert P. 

Johnson, Dorothee von Laer 

64 2:45 PM 

Characterization of the Mechanism of Action of the Anti- 

HIV-1 Transgene F12-Vif 

Rossella Lupo, Giuliana Vallanti, Maurizio Federico, Fulvio 

Mavilio, Chiara Bovolenta 

65 3:00 PM 

Protective Immunity Against Anthrax Lethal Toxin in 

Mice Immunized with an Ad-Based Vaccine Vector 

Expressing Lethal Factor 

Julie L. Boyer, Tiffany B. Niven, Neil R. Hackett, Ronald G. 

Crystal 

66 3:15 PM 

A Protective Vaccine for the Rapid Response to Pandemic 

AVian Influenza 

Wentao Gao, Adam Soloff, Xiuhua Liu, Angela Montecalvo, 

Yumiko Matsuoka, Paul D. Robbins, Ruben O. Donis, 

Jacqueline M. Katz, Simon Barratt-Boyes, Andrea Gambotto 

67 3:30 PM 

Protective Immunity Against Yersinia pestis in Mice 

Immunized with an Ad-Based Vaccine Vector Expressing 

the Y. pestis F1 and V Antigens 

Maria Chiuchiolo, Julie L. Boyer, Tiffany Niven, Anja Krause, 

Neil R. Hackett, Ronald G. Crystal 

68 3:45 PM 

Development of a Novel Non-Viral DNA Vaccine- 

Delivering Gene into the Nucleus of Dendritic Cells 

Takashi Nakamra, Kentaro Kogure, Rumiko Moriguchi, Shiroh 

Futaki, Masahiro Fujimuro, Hideyoshi Yokosawa, Tsukasa 

Seya, Hideyoshi Harashima 


Cancer-Targeted Gene Therapy: Non Adenoviral 

Targeting of Vectors and Non Viral Vectors 

Room: 265/266 

69 2:00 PM 

Tumor-Targeting Innumogene Therapy by Mesenchymal 

Stem Cells Expressing CX3CL1 

Hong Xin, Masahiko Kanehira, Hiroyuki Mizuguchi, Takao 

Hayakawa, Toshihiro Nukiwa, Yasuo Saijo 

70 2:15 PM 

Systemic Administration of siRNA Against EWS-FLI1 

Using a Targeted, Non-Viral Formulation Inhibits 

Growth in a Disseminated Murine Model of Ewing’s 

Sarcoma 

Jeremy D. Heidel, Siwen Hu-Lieskovan, Derek W. Bartlett, 

Timothy J. Triche, Mark E. Davis 

71 2:30 PM 

Intratumoral Delivery of Small Interfering RNA for 

Inhibition of Tumor Progression in Mice 

Yuki Takahashi, Makiya Nishikawa, Yoshinobu Takakura 

72 2:45 PM 

Capsid Modifications Overcome Low Heterogeneous 

Expression of Heparan Sulfate Proteoglycan That Limits 

AAV2-Mediated Gene Transfer and Therapeutic Efficacy 

in Human Ovarian Carcinoma 

Jeffrey S. Bartlett, Wenfang Shi 

73 3:00 PM 

Fully Retargeted Oncolytic Measles Viruses for Cancer 


Takafumi Nakamura, Mary Harvey, Suzanne Greiner, Kah- 

Whye Peng, Stephen J. Russell 

74 3:15 PM 

Specific Delivery of hTRAIL and Inhibition of Ewing’s 

Sarcoma Growth by Transplantation of Viral Vector- 

Transduced Bone Marrow-Derived Mesenchymal Stem 

Cells (MSC) 

Jeffrey S. Bartlett, Wenfang Shi, Carmel Lawrencia 

75 3:30 PM 

Molecular Imaging of an EGFP-DRD2-Expressing 

Vaccinia Virus 

Navneet Mehta, Nan Tang, Hui Deng, Deborah Scollard, James 

Jonkman, Brian C. Wilson, Raymond M. Reilly, Judith A. 

McCart 

76 3:45 PM 

New Strategy To Improve Imaging and Retargeting of 

HSV-1 Vectors 

Paola Grandi, Trevor D. McKee, Wilson Mok, Juliet L. 

Fernandez, Xandra O. Breakefield, Rakesh K. Jain 

77 4:00 PM 

Efficient Transduction and Therapy of Malignant Glioma 

by Lentiviral Vectors Pseudotyped with LCMV Glycoproteins 

Hrvoje Miletic, Yvonne Fischer, Harald Neumann, Tsanan 

Giroglou, Manuel M. Hermann, Werner Stenzel, Maria A. 

Rueger, Yannic Waerzeggers, Uwe Himmelreich, Mathias Hoehn 

Andreas H. Jacobs, Martina Deckert, Dorothee von Laer


ABSTRACTS 


78 4:15 PM 

Expression of the Herpes Simplex Virus Thymidine 

Kinase Gene by a Promoter Region of the Human 

SPARC Gene Inhibits Human Melanoma Cell Growth In 

Vitro and In Vivo 

Maria V. Lopez, Patricia V. Blanco, Diego L. Viale, Eduardo A. 

Cafferata, David Gould, Yuti Chernajovsky, Osvaldo Podhajcer 


Gene Regulation: Keeping Genes On and Turning 

Them Off 

Room: 261/262 

79 2:00 PM 

Real Time Fluorescent Tracking of Dynamic Variegation 

by Retrovirus Vectors in Stem Cells 

James Ellis, John Ramunas, Liam Kelly, Tanya Sukonnik, Eric 

Jervis 

80 2:15 PM 

Insulation of Oncoretrovirus Vectors with the cHS4 

Chromatin Insulator Is Associated with an Open Chromatin 

Histone Code 

Changlong Li, David W. Emery 

81 2:30 PM 

A Retrovirus Vector Screen for the Identification of 

Novel Chromatin Insulators and Enhancers 

Amy C. Groth, George Stamatoyannopoulos, David W. Emery 

82 2:45 PM 

Targeted Pancreatic Cancer-Specific Gene Therapy 

Controlled by a Molecular-Engineered Human Cholecystokinin 

Type-A Receptor (CCKAR) Promoter in In 

Vivo Imaging Models 

Xiaoming Xie, Zheng Li, Qingqing Ding, Hus-Ping Kuo, 

James L. Abbruzzese, Mien-Chie Hung 

83 3:00 PM 

Persistent and Position Independent Transgene Expression 

in Erythroid Cells Transduced by Lentiviral Vectors 

Annarita Miccio, Francesco Lotti, Claudia Rossi, Giuliana 

Ferrari 

84 3:15 PM 

Assessing Hemostatic Efficacy of Continuous Expression 

of Factor VIIa after AAV-Mediated, Liver-Directed Gene 

Transfer in Hemophilia A and B Mice 

Alexander Schlachterman, Paris Margaritis, Majed Aljamali, 

Jianhua Liu, Valder Arruda, Katherine High 

85 3:30 PM 

Development of Zinc Finger Nucleases for Therapeutic 

Gene Correction of Sickle Cell Anemia 

Jeffrey C. Miller, Fyodor D. Urnov, Ya-li Lee, Jeremy Rock, 

Tyler Sorba, Sarah Patterson, Philip D. Gregory, Michael C. 

Holmes, Edward J. Rebar 

86 3:45 PM 

Transcriptional Gene Silencing in Human Cells 

Zhaoxia Chen, Ali Ehsani, Riggs Art, Rossi J. John, Kevin V. 

Morris 

87 4:00 PM 

Determinants of Toxicity Induced by In Vivo Expression 

of Short Hairpin RNA from AAV Vectors 

Dirk Grimm, Konrad L. Streetz, Kusum Pandey, Theresa A. 

Storm, Mark A. Kay 

88 4:15 PM 

Driving shRNA Transcription from PolII Promoters in 

Mammalian Cells 

Jeffery C. Giering, Mark A. Kay 

Poster Session I 

Exhibit Hall 2 

RNA Virus Vectors: Gene Expression 

89 

Amelioration of Gamma Retroviral Silencing during 

Embryonic Stem Cell-Derived Hematopoiesis: Preclinical 

Assessment of Safety-Improved Vectors 

Ali Ramezani, Teresa S. Hawley, Robert G. Hawley 

90 

In Vitro and In Vivo Gene Expression of Lentiviral 

Vectors in CD4+ T Cells 

Kit L. Shaw, Karen Pepper, Denise Petersen, Vesa Kaartinen, 

Donald B. Kohn 

91 

Insertion of the cHS4-Insulator into Lentiviral Vectors: 

Use of Real-Time PCR in Order To Analyze the Effects 

on Transgene Expression and Viral Titre 

Johan Jakobsson, Troels Tolstrup Nielsen, Nina Rosenqvist, 

Cecilia Lundberg 

92 

Efficient Trangene Expression in Central Nervous System 

through a Non-Integrative Lentiviral Vector 

Stephanie Philippe, Che Serguera, Sebastien Bonnel, Christelle 

Vetu, Martine Barkats, Caroline Petit, Marc Abitbol, Chamsy 

Sarkis, Jacques Mallet 

93 

Diminished Mobilization of Self-Inactivating (SIN) 

Lentiviral Vectors Containing Globin Regulatory Elements 

Compared to Those Containing a Retroviral Long 

Terminal Repeat 

Hideki Hanawa, Derek A. Persons, Takashi Shimada, Arthur 

W. Nienhuis 

94 

Transgene Expression with Integration-Deficient HIV-1 

Based Lentiviral Vectors Expressing Class I Integrase 

Mutants 

G. Joseph, J. Marsh, T. Johnson, K. Cornetta

52 


ABSTRACTS 


95 

Transduction and Expression of shRNAs Via Lentiviral 

Bectors Bearing a U6 RNA Polymerase III Promoter, but 

Not an H1 Promoter, Induce Elevated Apoptosis in 

Human Primary Peripheral Blood T Lymphocytes 

Dong Sung An, Xiao-Feng Qin, Si-Hua Mao, Vincent C. 

Auyeung, David Baltimore, Irvin S. Y. Chen 

96 

Ocular Gene Transfer Using EIAV Vectors and an RPE- 

Specific Promoter 

Shu Kachi, Katie Binley, Naoyasu Umeda, Hideo Akiyama, 

Raquel Lima e Silva, Wei-Hong Xiao, Michiko Kachi, Margaret 

Esapa, Sharifah Iqball, Stuart Naylor Peter A. Campochiaro 

97 

Type I Interferon Response Against Virus Vectors and 

Non-Viral Gene Transfer 

Riikka Pellinen, Outi Meriläinen, Saara Lehmusvaara, Tuula 

Salonen, Katja Häkkinen, Tanja Hakkarainen, Elisa 

Vähäkangas, Kari Airenne, Ilkka Julkunen, Ari Hinkkanen 

Jarmo Wahlfors 

98 

Stable Lentivirus-Mediated RNA Interference: Comparison 

of U6 and H1 Promoters in Endothelial Cells and 

GFP Knockdown in Mouse Brain 

Petri I. Makinen, Jonna K. Koponen, Anna-Mari Turunen, 

Tarja M. Malm, Jari Koistinaho, Mikko P. Turunen, Seppo 

Yla-Herttuala 

99 

Use of a Liver-Specific Lentivirus Vector for Characterization 

of Hepatocyte-Like Cells from Human Differentiated 

Embryonic Stem Cells (hESC) 

Yuyou Duan, Naoki Yamamoto, Jian Wu, Sanjeev Gupta, 

Mark A. Zern 

100 

Development of an Integrase Deficient FIV Vector for 

Transient Gene Expression 

Marisa Banasik, Melissa Hickey, Paul McCray Jr. 

101 

Development of Lentiviral Vectors with Respiratory 

Epithelial Specific Transgene Expression 

Dinithi Senadheera, Benjamin Hendrickson, Denise Petersen, 

Karen Pepper, Carolyn Lutzko 

102 

Overexpression of the Myostatin Antagonist Follistatin in 

Normal Myoblasts Genetically Modified with a 

Lentivirus 

Basma F. Benabdallah, Joel Rousseau, Manaf Bouchentouf, 

Jacques P. Tremblay 

103 

PGK and CMV Promoters Exert the Strongest Activity in 

Lentiviral Gene Transduction of Myeloid Cells Including 

Mature Neutrophils 

Yasushi Soda, Kenzaburo Tani, Xiaojin Li, Yuansong Bai, 

Seok-Goo Cho, Muneyoshi Futami, Minghan Chen, Seiichiro 

Kobayashi, Hiroyuki Miyoshi, Hideki Sumimoto Shoichi 

Ohga, Toshiro Hara, Arinobu Tojo, Shigetaka Asano 

104 

Stable High Level Gene Expression by Minimum Sized 

Murine Leukemia Virus (MLV)-Based Retroviral Vectors 

in the Mouse Model 

Seung Shin Yu, Youngtae Hong, Sujeong Kim, Chang Wan Joo, 

Nam Kyung Yoon, Sunyoung Kim 

105 

Comparative Study on the Gene Expression Profile of 

HIV-1 Based Lentiviral Vectors Transduced Human and 

Murine Cells 

Yuan Zhao, Selina Azam, Robin Thorpe 

106 

Measles Virus Host Defense Evasion Proteins V and C 

Have Also Transcription Modulation and Infectivity 

Factor Functions 

Patricia Devaux, Warangkhana Songsungthong, Christoph 

Springfeld, Veronika von Messling, Roberto Cattaneo 

107 

Sustained Stable Gene Expression Induced by a Cytoplasmic 

RNA-Virus Vector Based on Mutant Sendai Virus 

Strain cl.151 

Ken Nishimura, Hiroaki Segawa, Takahiro Goto, Mariko 

Morishita, Takemasa Sakaguchi, Tetsuya Yoshida, Kozo 

Takayama, Mahito Nakanishi 

108 

The Complete Nucleotide Sequence of Sendai Virus 

Isolate BB1 and Comparison with Other Isolates 

Yu Yang, Lufeng Ren, Xiaoyan Dong, Xiaobing Wu 

109 

Expression of Porcine að(1-3)galactosyltransferase in 

Non-Human Primate Hematopoietic Progenitors 

Following Retroviral Mediated Gene Transfer 

Lorenzo Benatuil, John Iacomini 

(Note: Abstract 822 has been moved from Poster 

Session III to Poster Session I and will follow Abstract 

109. See page 96 for abstract title and authors.)


ABSTRACTS 



110 

Performance of Different AAV Serotype Vectors Following 

Injection into the Deep Cerebellar Nuclei of 

ASMKO Mouse Brain 

James C. Dodge, Jennifer Clarke, Anthony Song, Jie Bu, Qi 

Zhao, Tatyana V. Taksir, Denise Griffiths, Lamya S. 

Shihabuddin, Catherine R. O’Riordan, Marco A. Passini Ed H. 

Schuchman, Gregory R. Stewart 

111 

AAV7, 8, and 9 Are More Efficient and Less Immunogenic 

Vectors for Muscle-Directed Gene Therapy for 

Hemophilia B 

Lili Wang, Jean-Pierre Louboutin, Yan Li, James M. Wilson 

112 

Strong Transgene-Specific Immune Responses Can Be 

Elicited with Novel Adeno-Associated Virus Vectors in 

Mice 

Yan Zhi, Julie Johnston, Di Wu, Joanita Figueredo, Martin 

Lock, Peter Bell, Guangping Gao, James M. Wilson 

113 

Manipulating AAV2 Tropism for Enhanced Delivery of 

AAV Vectors to the Spinal Cord 

Adam Davis, Matthew Stachler, Wenfang Shi, James Liu, 

Nicholas Boulis, Jeffrey Bartlett 

114 

Efficient Arterial Formation at the Sites of Adult Neo- 

Angiogenesis Requires the Recruitment of Bone Marrow 

Cells through the Neuropilin-1 (NP-1) Receptor 

Serena Zacchigna, Nikola Arsic, Lucia Pattarini, Silvia 

Moimas, Alessandro Carrer, Lorena Zentilin, Alessandro Salvi, 

Gianfranco Sinagra, Mauro Giacca 

115 

In Vivo Expression of Human Cob(I)alamin 

Adenosyltransferase Using rAAV Serotypes 1,2 and 8, an 

Approach to Gene Therapy for Methylmalonic Aciduria 

(MMA) 

Kirsten E. Erger, Thomas J. Conlon, Travis L. Cossette, Thomas 

A. Bobik, Terence R. Flotte 

116 

Functional Comparison of Four CFTR rAAV Vectors 

with Alternative Transcriptional Elements in Cell Lines 

and Polarized Human CF Airway Epithelia 

Liang Zhang, Benjamin Dutzar, Keith Munson, Barrie Carter, 

John F. Engelhardt 

117 

AAV-Mediated Erythropoietin Gene Transfer Protects 

from Genetic and Light-Induced Retinal Degeneration 

Mariacarmela Allocca, Tonia S. Rex, Luciano Domenici, Jean 

Bennet, Victor M. Rivera, Alberto Auricchio 

118 

Self-Complementary AAV Vectors Pseudotyped with 

Either Serotype 5 or 8 Capsids Give Therapeutic Human 

Factor IX Levels at Significantly Reduced Doses in Non- 

Human Primates 

John T. Gray, Andrew M. Davidoff, Amit C. Nathwani 

119 

Intraperitoneal Transduction of Adeno-Associated Virus 2 

Expressing Angiostatin and Endostatin Synergistically 

Augments Paclitaxel Therapy and Tumor-Free Survival in 

a Mouse Model of Epithelial Ovarian Cancer 

Tatyana Isayeva, Changchun Ren, Selvarangan Ponnazhagan 

120 

Stimulation of the Synthesis of Type-II Collagen and 

Extracellular Matrix in Human Normal and Osteoarthritic 

Articular Cartilage Explants In Situ by 

Overexpression of Human sox9 by rAAV-Mediated Gene 

Transfer 

Henning Madry, Tanja Thurn, Dieter Kohn, Ernest F. 

Terwilliger, Magali Cucchiarini 

121 

VEGF-Induced Angiogenesis Does Not Have a Favorable 

Effect on Muscle Perfusion: A Relevant Role of Long- 

Term Angiopoietin-1 Expression in the Formation of 

Functional Blood Vessels 

Serena Zacchigna, Ennio Tasciotti, Nikola Arsic, Oreste Sorace, 

Marini Cecilia, Silvia Pardini, Debora Petroni, Claudia 

Kusmic, Lucia Pattarini, Silvia Moimas GianMario Sambuceti, 

Mauro Giacca 

122 

Enhanced Expression of Glutamate Decarboxylase 65 

Significantly Improves Rat Parkinsonian Symptom 

Boyoung Lee, Young Ran Nam, Sung Jin Kim, Jin Hwan Oh, 

Yoon Hee Choi, Jin Woo Chang, Heuiran Lee 

123 

rAAV-pdx-1 Augmented the Insulin-Producing Cells 

Hua Li, Karen S. Lam, Sidney Tam, Weidong Xiao, Paul Tam, 

Ruian Xu 

124 

The “Perivascular Pump” Driven by Arterial Pulsation Is 

a Powerful Mechanism for the Distribution of Therapeutic 

Molecules within the Brain 

Piotr Hadaczek, Hanna Mirek, Laszlo Tamas, Martha C. 

Bohn, Charles Noble, John W. Park, Krystof Bankiewicz 

125 

AAV Vector-Mediated Neonatal Gene Transfer: Efficient 

Transgene Expression in Muscles after Intraperitoneal 

Cavity Vector Injection 

Tsuyoshi Ogura, Hiroaki Mizukami, Jun Mimuro, Takashi 

Okada, Hiromi Hamada, Akihiro Kume, Hiroyuki Yoshikawa, 

Yoichi Sakata, Keiya Ozawa

54 


ABSTRACTS 


126 

The Formation of Stable and Mature Blood Vessels 

Requires the Proper Timing of VEGF Expression 

Sabrina Tafuro, Serena Zacchigna, Lorena Zentilin, Marina 

Dapas, Mauro Giacca 

127 

Intraarticular Gene Transfer of Rat TNFR:Fc Suppressed 

CIA Arthritis 

Ai Zhi Zhao, Kai Gao, Xiao Yan Dong, Jian Qiang Peng, 

Xiao Bing Wu 

128 

Transduction of Hepatic Oval Cells, Stem Cells, In Vitro 

by Adeno-Associated Virus Vectors 

Hong You, Ping Wang, Jidong Jia, Liu Yong, Paul L. Hermonat 

129 

Development of Vectors Utilizing Strong Neuronal Cell 

Type Specific Promoters for High Level Expression of 

Beta Glucuronidase in the Central Nervous System 

Tupur Husain, John H. Wolfe 

130 

Characterization of a Murine Adeno-Associated Virus 

(AAV-mo.1) Capsid 

Michael Lochrie, Gwen Tatsuno, Alejandra Arbetman, Peter 

Smith, Jennifer Wellman, Shang-zhen Zhou, Glenn Pierce, 

Peter Colosi 

131 

Recombinant Adeno-Associated Virus 2-Mediated 

Transfer of the Human Superoxide-Dismutase Gene 

Does Not Confer Radioresistance on HeLa Cervical 

Carcinoma Cells 

Marlon R. Veldwijk, Carsten Herskind, Stephanie Laufs, W. 

Jens Zeller, Stefan Fruehauf, Frederik Wenz 

132 

An Optimized CsCl Gradient -Based Method to Generate 

High Concentration AAV Vector Stocks of High 

Purity and In Vivo Potency 

Mario Cooper, Bernd Hauck, Jeff Watkins, Megan Erne, 

Federico Mingozzi, Roland Herzog, Fraser Wright 

133 

Comparison of Adeno-Associated Viral Vector Serotypes 

1, 2, 5 and 6 for Gene Transfer to Neurons and Glia In 

Vitro 

Doug B. Howard, Juan Li, Yun Wang, Brandon K. Harvey 

134 

Toxicology and Biodistribution Studies of a Recombinant 

Adeno-Associated Virus 1 (rAAV1) Alpha-1 Antitrypsin 

(AAT) Vector 

Amy E. Poirier, Thomas J. Conlon, Lynn A. Combee, Kirsten 

E. Erger, Terence R. Flotte 

135 

AAV-Mediated Gene Delivery To Evaluate the Biology of 

an Inherited Macular Degeneration 

Nicholas W. Keiser, Daniel C. Chung, Waixing Tang, 

Zhanyong Wei, Albert Maguire, Jean Bennett, Jeannette L. 

Bennicelli 

136 

Recombinant Adeno-Associated Virus 2-Mediated 

Ectopic Expression of að4bð1 Integrin in Mouse Mesenchymal 

Stem Cells Enhances Repopulation to Bone upon 

Syngeneic Transplantation in an Immunocompetent 

Mouse Model 

Sanjay Kumar, Selvarangan Ponnazhagan 

Adenovirus Vectors: General Biology and Host 

Response 

137 

Localization of Regions in CD46 That Interact with 

Adenovirus 

Anuj Gaggar, Dmitry M. Shayakhmetov, M. Kathryn 

Liszewski, John P. Atkinson, Andre Lieber 

138 

Blood Factors Affect Adenovirus Infectivity and Bio- 

Distribution In Vivo 

Dmitry M. Shayakhmetov 

139 

Priming and Boosting Efficiency of Chimp and Human 

Adenoviral Vectors in the Setting of Pre-Existing Immunity 

in Non-Human Primates 

Joanita Figueredo, Roberto Calcedo, Jim Miller, Gary Kobinger, 

Rebecca Grant, Guangping Gao, James M. Wilson 

140 

Primary Adenovirus Specific Cytotoxic T Lymphocyte 

Response Occurs after Viral Clearance and Liver Enzyme 

Elevation 

Jian Chen, Allan J. Zajac, Sylvia A. McPherson, Xin Xu, 

PingAr Yang, Qi Wu, David T. Curiel, John D. Mountz 

141 

Adenovirus Vaccine to Ebola Virus: Mechanism of 

Protection and Impact of Pre-Existing Immunity to the 

Vaccine Carrier 

Gary P. Kobinger, Heinz Feldmann, Yan Zhi, Gregory 

Schumer, Guangping Gao, Frederik Feldmann, Steven Jones, 

James M. Wilson 

142 

Molecular and Macromolecular Alterations of Recombinant 

Adenoviral Vectors Do Not Eliminate Changes in 

Hepatic Drug Metabolism 

Shellie M. Callahan, Michael P. Boquet, Piyanuch Wonganan, 

Maria A. Croyle


ABSTRACTS 


143 

Regulated, High Capacity Adenoviral Vectors Mediated 

Long-Term Gene Expression in the Brain Even in the 

Presence of a Peripheral Immune Response to Adenovirus 

Weidong Xiong, Shyam Goverdhana, James F. Curtin, Carlos 

Barcia-Gonzalez, Jeffrey M. Zirger, Gwendalyn D. King, 

Sandra A. Sciascia, Mariana Puntel, Marianela Candolfi, 

Donna Palmer Philip Ng, Pedro R. Lowenstein, Maria G. 

Castro 

144 

In Vivo Studies on the Toxicity of Adenovirus Vectors for 

Kupffer Cells 

Elanchezhiyan Manickan, Jeffrey S. Smith, Jie Tian, Andrew P. 

Byrnes 

145 

Evaluation of Biodistribution and Safety of Adenovirus 

Vectors Containing B-Group Fibers after Intravenous 

Injection into Baboons 

Shaoheng Ni, Kathrin Bernt, Zong-Yi Li, Hans-Peter Kiem, 

Andre Lieber 

146 

T Cell Response Against the Cystic Fibrosis Transmembrane 

Conductance Regulator (CFTR) Gene 

Maria Limberis, Joanita Figueredo, James M. Wilson 

147 

Adenovirus Vector E4 Gene Regulates Connexin 40 and 

43 Expression in Endothelial Cells Via PKA and PI3K 

Signal Pathways 

Joseph Cheng, Fan Zhang, George Lam, David Jin, Loic 

Vincent, Neil R. Hackett, Shiyang Wang, Lauren M. Young, 

Barbara Hempstead, Ronald G. Crystal Shahin Rafii 

148 

The Transgene Cassette Is Not Fully Responsible for 

Alterations of Renal Cytochrome P450 Expression after 

Systemic Administration of Recombinant Adenovirus 

Hong T. Le, Michael P. Boquet, Erin A. Clark, Shellie M. 

Callahan, Maria A. Croyle 

149 

Transient Kupffer Cell Saturation, Via Adenovirus or 

Adenoviral Protein, Enhances Expression of Reporter 

Virus 

Viraj P. Mane, Christian Clarke, Lali Medina-Kauwe, Milton 

Finegold, Brendan Lee 

150 

Pre-Existing Nab and Neutralization of Therapeutic 

Dose Adenovirus Serotypes In Vitro 

Edward C. Nwanegbo, Wentao Gao, Andrea Gambotto, Paul 

Robbins 

151 

Evaluation of a Serotype 35 Fiber Containing Adenovirus 

Vector for Vaccination 

Nelson C. Di Paolo, Shaoheng Ni, Anuj Gaggar, Zong-Yi Li, 

Andre Lieber 

152 

Coxsackievirus-Adenovirus Receptor (CAR) and α β or v 3 

α β Integrin Independent Internalization of Porcine 

v 5 

Adenoviral Vectors: Implications in Gene Therapy 

Dinesh S. Bangari, Suresh K. Mittal 

153 

Adenoviral Gene Therapy Vectors Expressing the 

Adenovirus Death Protein Demonstrate Increased 

Transgene Expression In Vivo as Demonstrated by Dose- 

Volume Histogram Analysis of Reporter Gene Activity 

Kenneth N. Barton, Stephen L. Brown, Jae Ho Kim, Hans 

Stricker, Kastytis Karvelis, Mei Lu, Svend O. Freytag 

154 

Relationship between the Expression of Membrane 

Receptors (CAR, Integrin aðvbð3 and Integrin aðvbð5 ) 

and Adenoviral Transduction Efficiency 

Hiroyuki Inoue, Terumasa Hisano, Yukoh Nakazaki, Gaku 

Sakaguchi, Ryo Kurita, Koichi Takayama, Yoichi Nakanishi, 

Kenzaburo Tani 

“Other” DNA Virus Vectors 

155 

Oral Delivery of Recombinant Vaccinia Viruses Expressing 

Adjuvanted Islet Autoantigens Protects NOD Mice 

from Autoimmune Diabetes 

William H. R. Langridge, Bela Denes, Nadia Fodor, Valentina 

Krausova, Tatyana Timiryasova, David Henderson, John 

Hough, Jie Yu, Istvan Fodor 

156 

Update on Genomic HSV Vector-Related Cytotoxicity: A 

Vector Preserving the Differentiation Potential of 

Embryonic and Adult Adipose Stem Cells 

Julie Fradette, Steven K. Wendell, Jim Wechuck, David M. 

Krisky, Darren Wolfe, William F. Goins, Joseph C. Glorioso 

157 

Quantitation of Long-Term Gene Expression from the 

HSV-1 Latency-Associated Promoter in the Mouse Brain 

Following Intracranial Injection 

Bradford K. Berges, Ananya Gupta, John H. Wolfe, Nigel W. 

Fraser 

158 

Gene Therapy for Chronic Pain Control: Perspectives of 

HSV-1 Mediated Gene Transfer to Supraspinal Centres 

Isabel Martins, Marta Pinto, Steven Wilson, Deolinda Lima, 

Isaura Tavares

56 


ABSTRACTS 


159 

Unexpected Genetic Alterations Occur in Untargeted 

Genes during the Construction of Viral Vectors 

Megan J. Dambach, Jordan Trecki, Natalia Martin, Nancy S. 

Markovitz 

160 

Systemic Oncolytic Herpes Simplex Virus Therapy of 

Spontaneously Arising Prostate Cancer in Transgenic 

TRAMP Mice 

Susan Varghese, Samuel D. Rabkin, Petur G. Nielsen, Renbin 

Liu, Wenzheng Wang, Robert L. Martuza 

161 

Targeting HSV Infection to gD Receptor-Negative Cells 

Using Soluble gD Receptors 

Heechung Kwon, Qing Bai, Kelly Felmet, Hyun-Jung Baek, 

Gyuyoup Kim, William F. Goins, Justus B. Cohen, Joseph C. 

Glorioso 

162 

Targeted HSV-1 Infection by Adapters for Glycoprotein 

D: Variable Infection Efficiency Dependent on Ligand 

Choice 

Kenji Nakano, William F. Goins, Justus B. Cohen, Joseph C. 

Glorioso 

163 

Engineered of a Highly Efficient Cell Cycle Regulatable- 

HSV-1 Amplicon Viral Vector 

Grace Ying Wang, Ivy A. W. Ho, Kam M. Hui, Paula Y. P. 

Lam 

164 

Bioluminescence Imaging in the CNS after HSV 

Amplicon Vector Delivery 

Sam Wang, Lisa Pike, Jeremy Petravicz, Xandra O. Breakefield 

165 

Tracking of Baculovirus Biodistribution In Vivo by 

Magnetic Resonance Imaging 

Jani K. Raty, Thomas Wirth, Kari J. Airenne, Tuulia Huhtala, 

Timo Liimatainen, Ale Narvanen, Juhana Hakumaki, Seppo 

Yla-Herttuala 

166 

Recombinant Baculovirus Vectors Containing Inverted 

Terminal Repeats from Adeno-Associated Virus Improve 

Gene Expression in Neurons and Astrocytes 

Cao Yang Wang, Shu Wang 

167 

Both Oncolysis and Tumor Immunity Are Involved in an 

Autitumoral Efficacy by Intratumoral Injection of 

Recombinant Vaccinia Virus (TK Deleted, hGM-CSF 

Inserted Wyeth Strain) in a VX2 Rabbit Model 

Joon-Hyeong Kim, Byeong-Ho Park, Jong-Young Oh, David 

H. Kirn, Steve H. Thorne, Jae-Seok Kim, Tae-Ho Hwang 

168 

Antitumoral Efficacy of Multiple Injection of JX-594 

(Thymidine Kinase (TK) Deleted, Human GM-CSF 

Inserted Wyeth Strain) Via Tail Vein in N- 

Nitrosomorpholine (NNM) Treated Rats 

Jae-seok Kim, June-Hyung Kim, Jong-yeong Oh, Byeong-ho 

Park, Jin-Han Yoon, Steve H. Thorne, David H. Kirn, Tae-ho 

Hwang 

169 

The Creation of Novel Oncolytic Vaccinia Virus Vectors 

for Efficient Systemic Delivery of Transgenes to Tumors 

Steve H. Thorne, David H. Kirn 

170 

In Vitro Transduction of Cells To Determine Tropism 

Using Viral-Like Particles Derived from JC Virus VP1 

Linda Cashion, Oliver Ast, Andrzej Citkowicz, Susan Harvey, 

Branka Mitrovic, Mary Rose Masikat, Katalin Kauser, Brent 

Larsen, Gabor M. Rubanyi, Richard N. Harkins Harald Petry 

171 

Changing of Viral Tropism Alters Natural Proviral 

Property: Implications for Viral Vector Design 

Peng H. Tan, Shoa-An Xue, Hans J. Stuass, George J. T. 

George 

172 

In Vitro DNA Packaging and Gene Delivery Using JC 

Virus-Like Particles 

Oliver Ast, Andrezj Citkowicz, Linda Cashion, Brent Larsen, 

Gabor M. Rubanyi, Richard N. Harkins, Harald Petry 

173 

Process Design and Scale-Up of New Vaccine Concepts 

for Biodefense 

Sibylle U. Herzer, Peder Bergvall, Raf Lemmens, Matt Coffey, 

Walter T. Leps, Peter M. Moore 

174 

Process Development of a Scaleable Purification Protocol 

for the Production of Reolysin, an Oncolytic, Reovirus- 

Based Therapeutic 

Sibylle U. Herzer, Bergvall Peder, Lemmens Raf, Coffey Matt, 

Moore M. Peter 

175 

Titering Gene Delivery Vectors by QPCR: Application to 

rSV40-Derived Vectors 

Christine B. Mitchell, David S. Strayer 

Naked DNA Gene Transfer: Chromosomal, Integration, 

Delivery Techniques 

176 

Target-Site Preferences of Sleeping Beauty Transposons 

in an Intron of the B-raf Oncogene 

Jason B. Bell, Christopher S. Hackett, Aron M. Geurts, Corey 

M. Carlson, Lara S. Collier, Geyi Liu, David A. Largaespada, 

Perry B. Hackett


ABSTRACTS 


177 

Analysis of the High Shear Cell Lysis Device and the Air- 

Mixed Neutralization Column Used in the Recovery of 

Plasmid DNA from E. coli Cells 

Sriram Ramakrishnan, Hugo Gonzalez, Henry L. Hebel 

178 

A Novel RNA Quantitation Method Using Hydrophobic 

Interaction Chromatography-High Performance Liquid 

Chromatography 

Shane Johnson, Andrew Doig, Henry Hebel, Hugo Gonzalez, 

Heather Attra 

179 

Transgenesis of B Lymphocytes by Bacterial DNA Is 

TLR9 Independent 

Mara Gerloni, Matthew Wheeler, Xocthil Cortez-Gonzales, 

Ilenia Pellicciotta, Maurizio Zanetti 

180 

pDNAVACC Vector Family: High Throughput Intracellular 

Targeting DNA Vaccine Plasmids Created by Gene 

Self Assembly 

James A. Williams, Jeremy Luke, Lance Johnson, Clague P. 

Hodgson 

181 

The Effect of Dissolved Oxygen Fluctuations on Cell 

Growth and Plasmid Yield 

Sriram Ramakrishnan, Justin Miller, Jeffrey Darnell, Henry L. 

Hebel 

182 

An Effective and Valuable Process Using Disposable 

Membrane-Based Technologies for Pure Plasmid DNA 

Isolation and Purification: Throughput, Quality, and 

Cleaning Validation Advantages 

Hugo L. Gonzalez, Sriram Ramakrishnan, Shane Johnson, 

Andrew Doig, Henry L. Hebel 

183 

Development of Ex-Vivo Gene Therapy Procedures for 

Central Nervous System Diseases, Based on Homologous 

Recombination in Human Neural Stem Cells 

Carlos A. Bueno Lopez, Alberto Martinez Serrano 

184 

Nonvirally Electrotransfected Human Mesenchymal 

Stem Cells: A Novel Platform for Bone Gene Therapy 

Based on Autocrine and Paracrine Mechanisms 

Hadi Aslan, Yoram Zilberman, Jin Zhong Li, Gregory A. 

Helm, Zulma Gazit, Dan Gazit 

185 

Poly-lactide-co-glycolide Acid (PLGA) Nanoparticles for 

DNA Vaccination 

Jacob Gvili, Marcelle Machluf 

186 

Secreted Extracellular Vesicles of Baculovirus GP64 

Interact with Plasmid DNA to Enhance Transfection 

Ghiabe H. Guibinga, Andrew C. Hinton, Atsushi Miyanohara, 

Theodore Friedmann 

187 

Strategies of Intravascular Delivery of Naked DNA into 

Rat Skeletal Muscle 

Zhen Jia, István Dankó 

188 

Transfer of DNA in Tissues Using Local Fast Injection 

Franck M. Andre, Christel O. Cournil-Henrionnet, Paule 

Opolon, Lluis M. Mir 

189 

Improvement of In-Vivo Transfer of Plasmid DNA in 

Muscle: Comparison of Electroporation Versus Ultrasound 

Geke A. Hospers, Wendy A. Dam, Marc H. DeBaets, Mario H. 

Losen, Coby Meijer, Nanno H. Mulder 

190 

Improvement of Electroporation Devices and Methods 

for Use with Plasmid-Based Therapeutics and DNA 

Vaccine Applications in Small and Large Animals 

Amir S. Khan, Ruxandra Draghia-Akli 

191 

Identification of a Novel Functional Domain in the 

Sleeping Beauty Transposase: Towards Alleviating the 

Restriction of SB Overproduction Inhibition 

Julie Park, Stephen R. Yant, Mark A. Kay 

192 

Non-Viral, Long-Term Gene Expression in Hematopoietic 

Cells Using the Sleeping Beauty Transposon System 

Linda N. Liu, Jeffrey J. Essner, Rama Shivakumar, Linhong Li, 

Shannon A. Wadman, Karl J. Clark, Aidas Nasevicious, Joseph 

C. Fratantoni, R. Scott McIvor, Perry B. Hackett 

193 

Direct Comparison of Integration Efficiency and Stable 

Gene Expression Mediated by the Sleeping Beauty 

Transposon System and by the PhiC31 Phage Integrase 

System in Cultured Human Fibroblasts and in Various 

Stem Cell Targets 

Andy Wilber, Fernando Montoya, Aron M. Geurts, David A. 

Largaespada, Uma Lakshmipathy, R. Scott McIvor 

194 

Characteristics of Site-Specific Integration as Mediated 

by PhiC31 Integrase in the Human Genome 

Joylette L. Portlock, Thomas W. Chalberg, Eric C. Olivares, 

Bhaskar Thyagarajan, Patrick J. Kirby, Michele P. Calos

58 


ABSTRACTS 


195 

Non-Viral, Site-Specific Gene Transfer into Murine 


Jason J. Hoyt, Annahita Keravala, Jennifer Duda, Chris H. 

Contag, Michele P. Calos 


196 

Role of Kupffer Cells in Hepatic Transgene Expression 

Mediated by Intrabiliary Infusion of Nonviral Vectors 

Hui Dai, Xuan Jiang, Yong Chen, Hai-Quan Mao, Kam W. 

Leong 

197 

Hepatocyte-Specific Delivery of Short Interfering RNAs 

Using Reducible Polycations 

Mark Stevenson, Wei-li Liu, Surjeet Singh, Jon A. Preece, 

Simon S. Briggs, Uday Tilapur, Martin L. Read, Leonard W. 

Seymour 

198 

Design and Testing of Intrinsic Proteasome Inhibitors To 

Increase Gene Transfer Efficiency 

Molly E. Sinclair, Ji-seon Kim, Changpo Chen, Dijie Liu, 

Kevin G. Rice 

199 

Efficient Inhibition of Bone Metastasis Via Systemic 

Delivery of siRNA Using Atelocollagen-Mediated 

Delivery System 

Shunji Nagahara, Fumitaka Takeshita, Kimi Honma, Yoshiko 

Minakuchi, Akihiko Sano, Takahiro Ochiya 

200 

Factor VIII Gene Expression after Catheter-Based DNA 

Nanoparticle Delivery 

Katherine Bowman, Xiangying Du, Catherine LeVisage, Rita 

Sarkar, Xiaoming Yang, Kam W. Leong 

201 

C105Y, a Novel Cell Penetrating Peptide Enhances Gene 

Transfer of Sec-R Targeted Molecular Conjugates 

Melanie Rhee, Pamela B. Davis 

202 

DNA Delivery to the Mitochondria Sites Using Leader 

Peptide Conjugated Polyethylenimine 

Minhyung Lee, Joon Sig Choi, Kyung Soo Ko 

203 

Gene Transfer Activity Is Enhanced Significantly by 

Allowing Cationic Polymer to Interact with Serum 

Proteins prior to DNA Addition 

Christine C. Conwell, Feng Liu, Leaf Huang 

204 

Substrate-Mediated Gene Delivery for Assessment of 

Signal Transduction Pathways in Cancer Cells 

Angela K. Pannier, Zain Bengali, Eric A. Ariazi, V. Craig 

Jordan, Lonnie D. Shea 

205 

Quantitative Analysis of Non-Viral Gene Therapy in a 

Three-Dimensional Liver Bioreactor 

Nathan C. Tedford, Jennifer Fang, Bonnie Huang, Karel 

Domansky, Linda G. Griffith, Douglas A. Lauffenburger 

206 

Intracellular Signal-Responsive Gene Carrier for Cell- 

Specific Gene Therapy 

Yoshiki Katayama, Jeong Hun Kang, Kota Kodama, Jun Oishi, 

Takuro Niidome 

207 

Anti-GAD Antibody Targeted Non-Viral Gene Delivery 

to Islet Beta Cells 

Ji Hoon Jeong, Minhyung Lee, Won Jong Kim, James W. 

Yockman, Tae Gwan Park, Yong Hee Kim, Sung Wan Kim 

208 

Physicochemical and Biological Characterization of 

Cyclodextrin-Based Polycation (CDP)/siRNA Composites 

Designed for Systemic Delivery 

Derek W. Bartlett, Jeremy D. Heidel, Mark E. Davis 

209 

Nuclear Localization Mediated by the Adenoviral-Core 

Peptide Mu Enhances Phage jðC31-Integrase Activity in 


Richard P. Harbottle, Laurence Vaysse, Ahad Rahim, Yvonne 

Kienast, Ourania Passa, Oleg Tolmachov, Charles Coutelle 

210 

Intracellular Disassembly and Transcription Efficiency of 

Polyplexes Delivered into Cells Using Novel Self- 

Assembling Gene Carriers 

Tetsuji Yamaoka, Tomoko Hashimoto, Tatsuya Kitagawa, Akira 

Murakami 

211 

Novel Fiber-Mediated Adenovirus Interactions in 

Primary Rabbit Lacrimal Acini 

Jiansong Xie, Altan Rentsendorj, Hasmik Agadjanian, Lali K. 

Medina-Kauwe, Sarah F. Hamm-Alvarez 

212 

Differential, Transient Transduction of Defined Human 

Stem Cell Subsets Using Nanoparticles in a Clinically 

Applicable Ex Vivo Protocol 

Jesper Bonde, Dustin J. Maxwell, David A. Hess, Ryan Lahey, 

David Piwnica-Worms, Jan A. Nolta


ABSTRACTS 


213 

PEG-Modified Cationic Gold Nanoparticles and Their 

Abilities of In Vivo Gene Delivery 

Takahito Kawano, Yasuro Niidome, Yoshiki Katayama, Takuro 

Niidome 

214 

HVJ-E/Importin β Hybrid for Efficient Gene Delivery 

Takeshi Nagasaki, Takeshi Kawazu, Seiji Shinkai 

215 

Investigating Urokinase Plasminogen Activator Receptor 

(CD87), as a Potential Target for Non-Viral Gene 

Transfer 

Maya Thanou, Thomas Kean 

216 

Enhancement of Gene Transfection Both In Vitro and In 

Vivo Using Polymer Conjugated HVJ-Envelope Vector 

Hidetoshi Mima, Ryuji Tomoshige, Yasuhiko Tabata, Seiji 

Yamamoto, Susumu Ito, Katsuto Tamai, Yasufumi Kaneda 

217 

Synthesis and Characterization of a Smart Triblock 

Copolymer Carrier for siRNA 

Tatiana Segura, Jeffrey A. Hubbell 

218 

Soluble Flt-1 Gene Delivery Using PEI-g-PEG-RGD 

Conjugate for Anti-Angiogenesis 

Won Jong Kim, James W. Yockman, Minhyung Lee, Ji Hoon 

Jeong, Yong-Hee Kim, Sung Wan Kim 

219 

Development of a Polyethylene Glycol Gene Delivery 

Vehicle 

Nicole M. Kohrt, Lauren E. Grabski, Shelly E. Sakiyama- 

Elbert 

220 

Long-Term Implantation of Photo-Crosslinked Alginate 

Microcapsules 

Feng Shen, Anna A. Li, Murray A. Potter, Patricia L. Chang 

221 

Spermine-Grafted Hyaluronic Acid as Multifunctional 

Coating of the Plasmid/Polycation Complex for Protection, 

Targeting, and Transcriptional Activation 

Tomoko Ito, Naoko Iida-Tanaka, Koji Kubo, Kenichi 

Yoshikawa, Yoshiyuki Koyama 

222 

Enhancement of Intracellular Gene Delivery by the S4 - 13 

PV Cell Penetrating Peptide 

Miguel Mano, Cristina Teodósio, Artur Paiva, Sérgio Simões, 

Maria C. Pedroso de Lima 

223 

In Vitro Gene Transfer from a DNA-PEI Loaded 

Polylactide-Glycolide Porous Sponge 

Garrett R. Rettig, Moses Oyeyumi, Yen-Chen Huang, Kevin G. 

Rice 

224 

Novel Receptor-Mediated Gene Transfection Systems 

Comprising Plasmid / Polycation / RGD-PEG-COOH 

Ternary Complexes 

Mitsuko Sakae, Tomoko Ito, Naoko Iida Tanaka, Yoshiyuki 

Koyama 

225 

Characterization and Evaluation of Branched and Linear 

Polyethyleneimine Conjugated PLGA Microparticles for 

Applications in DNA Vaccine Delivery 

Sudhir P. Kasturi, Karishma Sachabhipulkij, Krishnendu Roy 

226 

Transcription-Activating Effect of the Synthetic Water- 

Soluble Polyampholytes 

Misa Yamashita, Tomoko Ito, Naoko Iida-Tanaka, Yoshiyuki 

Koyama 

227 

Functionalized Multi-Block Co-Polymers of Short 

Length Linear Polyethylenimines for Safe and Efficient 


Gregory Slobodkin, Majed Matar, Angela Ramsey, Jason Fewell, 

Elaine Brunhoeber, Khursheed Anwer 

228 

Secretory PTD Fusion Proteins 

Paul A. Shaw, Ian R. Catchpole, William H. Colledge 

229 

Development of a Novel Drug Delivery System Based on 

Intrapericardial Administration of Plasmid DNA in a 

Thermosensitive Gel Formulation 

Caroline Roques, André Salmon, Armelle Serose, Elias Fattal, 

Yves Y. Fromes 

230 

The Study on Chitosan Nanoparticles as New Gene 

Delivery System 

Minli Sun, Hao Zhang, Baigen Zhang 

231 

Fluorescent Tracking of Non-Viral Gene Delivery 

Saeeda Jaffar, Angela Belcher 

Inborn Errors of Metabolism: Systemic and Storage 

Diseases 

232 

Gene Therapy for Deficient Cholesterol Synthesis in a 

Mouse Model of SLOS 

Madhavee Buddhikot, Josep Marcos, Dorothy Tabron, Forbes 

Porter, Cedric Shackleton, Gordon Watson

60 


ABSTRACTS 


233 

Towards Reversing Sandhoff Pathology by Lentiviral- 

Mediated Gene Therapy 

Audrey Arfi, Rivka Ziesling, Livia Poenaru, Anthony H. 

Futerman, Catherine Caillaud 

234 

Polyclonal Insertion Sites in Two Rhesus Macaques after 

Non-Myeloablative Transplantation with MFGS-gp91phox Transduced Autologous CD34+PBPC 

Sebastian Brenner, Martin F. Ryser, Uimook Choi, Narda 

Whiting-Theobald, Gilda Linton, Elizabeth Kang, Romy 

Lehmann, Anne M. Farese, Thomas J. MacVittie, Joachim 

Roesler Mitchell E. Horwitz, Harry L. Malech 

235 

The Route of Vector Delivery Impacts the Therapeutic 

Effects of AAV-Mediated Gene Therapy in Adult 

Mucopolysaccharidosis IIIB Mice 

Haiyan Fu, Lu Kang, Sheryl S. Moy, Jerillyn S. Jennings, 

Antonio Perez, Joseph Muenzer 

236 

Bone Marrow-Derived Cells Do Not Contribute to 

Endocrine Pancreas Regeneration in Diabetic RIP/IGF-I 

Transgenic Mice 

Eduard Ayuso, Veronica Jimenez, Judith Agudo, Virginia 

Haurigot, Jose Carlos Segovia, Juan Bueren, Fatima Bosch 

237 

Evaluation of Low-Level, Constitutive Expression of bðglucuronidase 

on the Clinical Manifestations of 

Mucopolysaccharidosis Type VII 

Anthony Donsante, Beth Levy, Carole Vogler, Mark Sands 

238 

Stem Cell Gene Delivery to Immunocompetent Mice 

Using a Non-Myeloablative Regimen Results in Long- 

Term Transgene Expression and Stable Mixed Chimerism 

Brian W. Bigger, Simon N. Waddington, Mamta Buch, Megha 

Nivsarkar, Nicholas Jones, Suzanne M. Watt, Kathryn Wood, 

Michael Themis 

239 

Immunomodulation Is Necessary to Achieve Stable 

Expression after Retroviral Vector-Mediated Liver- 

Directed Gene Therapy to Adult Mice with MPS I 

Xiucui Ma, Lingfei Xu, Yuli Liu, Bin Wang, Katherine P. Ponder 

240 

Intramuscular Administration of an AAV Vector Expressing 

4-Sulfatase (4S) to Newborn Cats With 

Mucopolysaccaridosis VI: Sulfatase Activity in Muscle, 

Serum, and Liver 

Mark Haskins, Thomas O’Malley, Ping Wang, Karyn Cullen, 

Anne Traas, Alessandra Tessitore, Monica Doria, Alberto 

Auricchio 

241 

Gene Expression in the Striatum of HPRT Knockout 

Mice 

Song Shaochun, Theodore Friedmann 

242 

The Use of AAV Serotype 1 Vector for Expression of að- 

Galactosidase A in Fabry Knockout Mice 

Koichi Ogawa, Yukihiko Hirai, Katsuko Zenri-Aihara, 

Yoshitaka Fukunaga, Takashi Shimada 

243 

Transplantation of Neonatal NOD/SCID/MPSVII Mice 

with Human Mesenchymal Stem Cells 

Phillip E. Herrbrich, Todd E. Meyerrose, Alex A. Hofling, 

Mark Sands, Jan A. Nolta 

244 

AAV Vector-Mediated Gene Therapy of 

Mucopolysaccharidosis Type I 

Lalitha Belur, Damaris Lorenzo, Elena Aronovich, Walter C. 

Low, Chester B. Whitley, R. Scott McIvor 

245 

AAV2/8-Mediated Efficacy and Immune Tolerance in a 

Pompe Disease Mouse Model 

S. D. Bercury, R. J. Ziegler, C. M. Barbon, D. Souza, D. 

Armentano, B. L. Thurberg, T. Taksir, H. Do, M. Trout, N. 

Raben S. H. Cheng 

246 

Comparison of rAAV2-Mediated Expression of Human 

and Murine β-Glucuronidase in Adult MPS VII Mice 

Kristin C. Greathouse, Chiou-Miin Wang, Thomas J. Sferra 

247 

Gene Therapy with a Retroviral Vector Expressing 

Canine β-Glucuronidase to Juvenile (7 Week Old ) Dogs 

Improves the Biochemical Manifestations of MPS VII 

Bin Wang, Ping Wang, Tom O’Malley, Lingfei Xu, Mark E. 

Haskins, Katherine P. Ponder 

248 

A Mouse Model of Type VII Glycogenosis to Assay Gene 

Therapy Approaches 

Fatima Bosch, Miquel Garcia, Anna Pujol, Albert Ruzo, Anna 

Arbos, Juan E. Feliu 

249 

Minimal Dose of AAV8/DC190-ASM Necessary for 

Therapeutic Efficacy in a Mouse Model of Niemann- 

Pick Disease 

R. J. Ziegler, C. M. Barbon, S. D. Bercury, M. L. Hawes, E. 

H. Schuchman, R. J. Desnick, S. H. Cheng 

250 

Correction of Hunter Syndrome in the Ids-Knock-Out 

Mouse Model by r-AVV Mediated Gene Therapy 

Monica Cardone, Vinicia Polito, Alessandra D’Azzo, Joseph 

Muenzer, Alberto Auricchio, Andrea Ballabio, Maria Pia Cosma


ABSTRACTS 




251 

Dystrophin in Vascular Smooth Muscle Is Important for 

Duchenne Muscular Dystrophy Therapy 

Kaori Ito, Shigemi Kimura, Gail D. Thomas, Shiro Ozasa, 

Makoto Matsukura, Makoto Ikezawa, Hiroe Ueno, Kowashi 

Yoshioka, Misao Suzuki, Takeshi Miwa Teruhisa Miike 

252 

A Novel Approach of Gentamicin Therapy for Duchenne 

Muscular Dystrophy Using Hybrid Liposome and 

Establishment of a System To Identify the Patients 

Eligible for the Treatment 

Shigemi Kimura, Kaori Ito, Shro Ozasa, Hiroe Kawano, 

Makoto Ikezawa, Makoto Matsukura, Ryuichi Ueoka, Yoko 

Matsumoto, Msafumi Matsuo, Yasuhiro Takeshima Teruhisa 

Miike 

253 

An AAV Vector-Mediated Gene Transfer into Canine 

Skeletal Muscle 

Madoka Ikemoto, Katsutoshi Yuasa, Madoka Yoshimura, Akiyo 

Nishiyama, Yuko Miyagoe-Suzuki, John McC Howell, 

Shin’ichi Takeda 

254 

Decorin Promotes Differentiation of Myoblasts into 

Myotubes That Express Slow MyHC In Vitro 

Yong Li, Juan Li, Ying Tang, Xiao Xiao, Johnny Huard 

255 

Embryonic Stem Cell-Mediated Regenerative Therapy 

for Duchenne Muscular Dystrophy 

Shiro Ozasa, Shigemi Kimura, Makoto Matsukura, Kaori Ito, 

Makoto Ikezawa, Hiroe Kawano, Teruhisa Miike, Kenichi 

Yamamura, Kimi Araki, Kuniya Abe Hitoshi Niwa 

256 

An Ideal Therapeutic Agent for Duchenne Muscular 

Dystrophy Involving a Gutted Adenovirus Expressing 

Full-Length Utrophin 

Jatinderpal R. Deol, Renald Gilbert, Mylene Bourget, Joon-Shik 

Moon, Josephine Nalbantoglu, George Karpati 

257 

Ex Vivo Gene Therapy for Duchenne Muscular Dystrophy: 

Lentiviral and PhiC31 Integrase Approaches 

Simon P. Quenneville, Pierre Chapdelaine, Joël Rousseau, 

Michele P. Calos, Jacques P. Tremblay 

258 

Identification of Differentially Expressed Genes in 

Duchenne Muscular Dystropy Utilising RNAi Technology: 

Possible Targets for Gene Therapy 

Mohammad Mahdi Ghahramani Seno, Ian R. Graham, Ken 

Laing, Marita Pohlschmidt, Takis Athanasopoulos, Mark 

Crompton, George J. Dickson 

259 

Uterine Fibroids Gene Therapy: Adenovirus-Mediated 

Herpes Simplex Virus Thymidine Kinase/Ganciclovir 

Treatment Inhibits Growth of Human and Rat Leiomyoma 


Ayman Al-Hendy, Marwa Kamel, Gregory Christman, Salama 

A. Salama 

260 

A Database for Managing Neuromuscular Disease Data 

in the Province of Quebec 

Charland Luc, Lavoie Jeremie, Puymirat Jack 

261 

Recombinant Adeno-Associated Viral (rAAV) 

Microdystrophin Vectors as Therapeutic Tools for 

Duchenne Muscular Dystrophy (DMD) 

Takis Athanasopoulos, Ian Graham, Helen Foster, Norma Perez, 

Adeline Vulin, Vanessa Hill, Stewart Fabb, Luis Garcia, Olivier 

Danos, George Dickson 

262 

Plasmid-Mediated Gene Transfer in mdx Mice Using 

Mini- and Micro-Dystrophin Constructs 

Leland E. Lim, Carmen Bertoni, Thomas A. Rando 

263 

Characterization and Gene Transfer in Mesenchymal 

Stem Cells Derived from Human Umbilical Cord Blood 

Feizhou Lu, Jianyuan Jiang, Masayuki Fujino, Xiao-Kang Li 

264 

Cell Therapy of Ovarian Failure: Intraovarian Transplanted 

Granulosa Cells Successfully Engraft and Repopulate 

Growing Follicles 

Ayman Al-Hendy, Hui Q. Wang, Salama A. Salama 

265 

Enhanced Plasmid-Mediated Dystrophin Expression in 

the mdx Mouse Model for Duchenne Muscular Dystrophy 

by a PhiC31 Integrase Plasmid System 

Carmen Bertoni, Sohail Jarrahian, Thurman M. Wheeler, 

Yining Li, Eric C. Olivares, Michele P. Calos, Thomas A. 

Rando 

266 

Development of rAAV-Based Skeletal and Cardiac 

Muscle Regulatory Cassettes for Gene Therapy of 

Duchenne Muscular Dystrophy 

Maja Z. Salva, Charis L. Himeda, Phillip Tai, Michael J. 

Blankinship, Paul Gregorevic, James M. Allen, Leonard A. 

Meuse, Jeffrey S. Chamberlain, Stephen D. Hauschka

62 


ABSTRACTS 


Cancer - Immunotherapy: Genetically Modified 

Antigen Presenting Cells and Adjuncts 

267 

Inflammatory Killing of Normal Cells To Treat Metastatic 

Tumors 

Luis Sanchez-Perez, Gregory Daniels, Timothy Kottke, Rosa 

Maria Diaz, Atique Ahmed, Jill Thompson, Heung Chong, 

Richard Vile 

268 

Primary Mouse Marrow Stromal Cells Behave as Antigen-Presenting 

Cells in Syngeneic Immune Responses 

John Stagg, Sandra Pommey, Jacques Galipeau 

269 

Stimulation of the Immune System Using Gene Therapy 

Is an Effective Approach for Treating Brain Tumors in 

Mouse and Rat Models 

James F. Curtin, Gwendalyn D. King, Weidong Xiong, 

Chunyan Liu, Pedro R. Lowenstein, Maria G. Castro 

270 

Vaccination of Recurrent Glioma Patients with Tumor 

Lysate-Pulsed Dendritic Cells Elicits Immune Responses: 

Results of a Clinical Phase I/II Trial 

Ryuya Yamanaka, Junpei Homma, Masuhiro Takahashi, 

Ryuichi Tanaka 

271 

Interactions between Replicating Virotherapy and Tumor 

Growth in the Presence of an Intact Immune System 

Michael Forshaw, Caroline Cole, Timothy Kottke, Jill Thompson, 

Jian Qiao, Peter Selby, Alan Melcher, Richard Vile 

272 

Induction of Anti-Tumoral Immune Response and IFN- 

Regulated Factors by Delivery of Control DNA 

Shulin Li, Miles Wilkinson, Xueqing Xia, Michael David, 

Lihong Xu 

273 

Effects of Pre-Existing Immunity on Gene Therapy 

Mediated by First Generation or High Capacity Adenoviral 

Vectors Expressing Flt3L in a Syngeneic Glioma 

Model 

Gwendalyn D. King, James F. Curtin, Weidong Xiong, 

Chunyan Liu, Donna Palmer, Philip Ng, Pedro R. Lowenstein, 

Maria G. Castro 

274 

Genetic Immunotherapy Targeted to Antigen Presenting 

Cells Using a CD40-Targeted Adenoviral Vector 

Disha A. Mody, Larry Smart, Yoshinobu Odaka, Xiao L. Li, 

Cigdem E. Yilmaz, Alexander V. Pereboev, J. Michael Mathis 

275 

RTVP-1 Protein Stimulates Markers of Maturation in 

Mouse and Human Dendritic Cells 

Hongyu Wang, El Moataz Abdelfattah, Salahaldin A. Tahir, 

Guang Yang, Alexei Goltsov, Weihua Tian, Chengzhen Ren, 

Chenghui Ren, Terry L. Timme, Timothy C. Thompson 

276 

Enhancement of Cytotoxic T-Lymphocyte Responses 

Against Carcinoembryonic antigen (CEA) Using Genetically 

Modified Dendritic Cells Expressing CEA and 

CD40 Ligand 

Nachimuthu Chinnasamy, James Neuenfeldt, James Shaffer, 

Dhanalakshmi Chinnasamy 

277 

Large-Scale Generation of Genetically Modified Human 

CD34+ Progenitor Cell-Derived Langerhans Cells 

Using a Closed Culture System Suitable for Clinical 

Immunotherapy 

Jianda Yuan, Ryan Kendle, Joanna Hodges, Michel Sadelain, 

James W. Young, Isabelle Riviere 

278 

Induction of Anti-Tumor Activity with Dendritic Cells 

Loaded with Tumor Cell Lysates or Adenoviral Vector- 

Encoding Tumor-Associated Antigens 

Jae-Gyun Jeong, Hwang-Jae Lee, Hae-Sook Ahn, Sung-Min 

Oh, Seong-Hyun Ho, Hongchan Cho, Seok Kang, Jong-Mook 

Kim, Sunyoung Kim, Sujeong Kim 

279 

A Novel Functionalized Polymeric Formulation for use 

in Delivering Therapeutic Genes for the Treatment of 

Solid Tumors 

Jason Fewell, Majed Matar, Jennifer Rice, Gregory Slobodkin, 

Bruce Hovanes, Khursheed Anwer 

280 

Effect of Polysaccharides from Medicinal Herbs as 

Danger Signals: Implication for Regulating Immune 

Responses 

Chih-Chien Shu, Jeng-Hwan Wang, Su-Jane Wang, Jih-Chun 

Wen, Wen-Chi Wei, Yi-Fu Yen, Kin-Yuen Cheng, Lie-Fen 

Shyur, Ning-Sun Yang 

281 

Evaluation of Plant Polysaccharides and Phyto-Compounds 

on Inflammation-Associated Tumorigenesis by 

Using Melanoma DNA Vaccine Model 

Su-Jane Wang, Jeng-Hwan Wang, Wen-Chi Wei, Chih-Chien 

Shu, Lie-Fen Shyur, Ning-Sun Yang 

282 

Effects of Fas Ligand on Inflammation and Tumor 

Survival Are Dependent on Its Expression Levels 

Masatoshi Tagawa, Akihiko Wada, Jiyang O-Wang, Kiyoko 

Kawamura, Osamu Shimozato, Yuji Tada, Yuichi Takiguchi, 

Koichiro Tatsumi, Takayuki Kuriyama


ABSTRACTS 


283 

Suppression of Inflammatory Pathways Selectively 

Induced by Oncolytic Viruses Enhances Viral Oncolysis 

Giulia Fulci, Laura M. Breymann, Elizabeth J. Fontana, 

Davide Gianni, Hiroaki Wakimoto, Ralph Weissleder, Robert 

Lee, Quing Liu, Antonio E. Chiocca 

284 

CD137L Gene Transfer Generates Effective and Long- 

Lasting Immunity in a Murine Plasmocytoma Model 

Young Kim, John W. Strehl, Marcus Gorschluter, Carsten Ziske, 

Christine Mayr, Ingo G. H. Schmidt-Wolf 

285 

Anti-Tumor Immune Responses Following Neoadjuvant 

Immunotherapy with a Recombinant Adenovirus 

Expressing HSP72 to Rodent Tumors 

James A. Krewet, Si-Yi Chen, Maulik R. Shah 


286 

Therapeutic Approach Using External Radiation and 

Suicide Gene Therapy Transduced by Chimeric Adenovirus 

Vector for Bladder Cancer 

Kazumasa Matsumoto, Norihiko Okuno, Bin S. Teh, Christian 

T. F. Freund, Julie X. Zhu, Maria T. Vlachaki, Alan R. 

Davis, Brian E. Butler, Seth P. Lerner 

287 

mda-7: A Remedy for Oncogene Addiction in Breast 

Cancer? 

Sunil Chada, Yanna Liu, Dora Bocangel, Ming Zheng, 

Rajagopal Ramesh, Raymond Meyn, Kelly Hunt 

288 

A System for GMP Expansion and Transduction of 

Human T Cells with High Functionality Proven by 

Consistent Induction of GvHD in a Mouse 

Xenotransplant Model 

Gerhard Bauer, Jon Walker, Bruno Nervi, Julie Ritchey, Jackie 

Hughes, Bill Eades, Mark Bonyhadi, Jan Nolta, John DiPersio 

289 

Highly Inducible and Prostate Specific Oncolytic Adenovirus 

with E1A-AR Chimera 

Ying Li, Chien-Lun Chen, David C. Johns, Ronald Rodriguez 

290 

Alpha-Tocopherol Succinate (VES) Enhances Ad-mda7- 

Mediated Killing of Ovarian Cancer Cells 

Manish Shanker, Sunil Chada, Rajagopal Ramesh 

291 

Comparison of Anti-Tumor Effects between Transmembrane 

and Secretory Tumor Necrosis Factor-alpha In Vitro 

and In Vivo 

Wenfang Shi, Qingfen Li, Zhuoya Li 

292 

Primary Chronic Lymphocytic Leukaemia Cells Are 

Resistant to the Oncolytic Effects of Attenuated Measles 

Virus 

Matthew A. Bates, R. G. Wickremasinghe, Adele K. Fielding 

293 

Optimizing the Efficacy of Spliceosome-Mediated RNA 

Trans-Splicing (SMaRT) for Suicide Gene Therapy of 

Cervical Cancer 

Michael Patrick, M. Puttaraju, Janet DiPasquale, Jesse Quintero, 

Yanping Yang, Mariano A. Garcia-Blanco, Lloyd G. Mitchell, 

Ed Otto, Carl C. Baker 

294 

Adenoviral-TRAIL and a Smac34-8K Fusion Peptide Act 

Synergistically to Induce Apoptosis in Low CAR- 

Expressing Human Glioma Cells 

Hiroyuki Mushiake, Moira R. Hitchens, Jeffrey C. Mai, Dai- 

Wu Seol, Paul D. Robbins 

295 

Adeno-Associated Virus-Mediated Transfer of the Genes 

Encoding Cryptic Kringle Fragments of Apolipoprotein 

(A) Suppresses Tumor Growth and Metastasis In Vivo 

Kyuhyun Lee, Sung-Tae Yun, Eui-Cheol Jo 

296 

Effective Suppression of Breast Cancer by 

Downregulation of VEGF Signaling 

Yi Lu, Jun Zhang, Derrick J. Beech, Lisa K. Jennings 

297 

Radiovirotherapy in Ovarian Cancer Using Recombinant 

Measles Expressing Sodium Iodide Symporter 

Kosei Hasegawa, Hooi-Tin Ong, David Dingli, Takafumi 

Nakamura, Stephen J. Russell, Kah Whye Peng 

298 

Gene Therapy Against Prostate Cancer Using the HSVtk/Prodrug 

System Followed by Prostatectomy 

Augusto Rojas-Martinez, Laura K. Aguilar, Rocio Ortiz- 

Lopez, Jacinto Esteban-Maria, Juan F. Gonzalez-Guerrero, 

Raquel Garza-Guajardo, Juan P. Flores-Gutierrez, E. Brian 

Butler, Hugo A. Barrera-Saldana, Estuardo Aguilar-Cordova 

299 

Increasing Gene Transfer and Oncolytic Potency of 

Adenoviruses in Orthotopic Models of Gastric Cancer 

Lotta Kangasniemi, Anna Kanerva, Mari Raki, Tuuli Ranki, 

Merja Sarkioja, Tuula Kiviluoto, Ulf-Hakan Stenman, Henrik 

Alfthan, Hongju Wu, David T. Curiel Akseli Hemminki 

300 

Adenoviral Delivery of the Gene Encoding Secretable 

Trimeric TRAIL Induces Apoptosis and Suppresses 

Human Malignant Glioma In Vivo 

Moonsup Jeong, In-Ho Kwon, Chae-Young Kim, Dai-Wu 

Seol, Paul D. Robbins, Byong-Moon Kim

64 


ABSTRACTS 


301 

Down-Regulation of bcl-xl Gene Expression with bcl-xl 

siRNA Vectors In Vitro and In Vivo 

Xiaobo X. Cao, Philip Rascoe, Jonathan Daniel, Charles 

Rodarte, W. Roy Smythe 

302 

Herpes Simplex Virus Thymidine Kinase/Ganciclovir 

Therapy Mediated by Transferrin-Associated Lipoplexes 

in a Murine Model for Oral Cancer 

Silvia S. Neves, Sergio Simões, Maria C. Pedroso de Lima 

303 

Validating CD46 as a Possible Mechanism Contributing 

to MV-Edm Oncolytic Specificity 

Hooi-Tin Ong, Stephen J. Russell, Kah-Whye Peng 

304 

Gene Therapy of Malignant Brain Tumor by a Novel 

Double-Stranded Adeno-Associated Viral (AAV) Vector 

Carrying the Decorin Gene 

Chi-Hsien Wang, Jun-Ming Han, Kai-Yun Chen, Shi-Yuan 

Cheng, Xiao Xiao, Hsin-I Ma 

305 

A Safe TRAIL to Lung Tumor Specificity 

Juan Shi, Dexian Zheng, Yanxin Liu, Mai Har Sham, Paul 

Tam, Farzin Farzaneh, Ruian Xu 

306 

A Novel Thymidine Kinase in Enhanced Gap Junction 

Mediated Suicide Gene Therapy 

Tomas J. Ekstrom, Zahidul Khan, Zoran Gojkovic, Mette 

Willer, Per M. Almqvist 

307 

The Tumor-Selective Viral Protein Apoptin Is a Potential 

Gene Therapeutic Agent for Prostate Cancer Therapy 

Xiang Liu, Ahmed El-Zawhry, David Holman, Saeed 

Elojeimy, Alicja Bielawaska, Jacek Bielawaska, Mahvash 

Tavassoli, James S. Norris 

308 

In Vivo Enhancement of HSV-TK/GCV Cancer Gene 

Therapy with Polyamine Biosynthesis Inhibition 

Tiina Wahlfors, Anne Karppinen, Juhani Jänne, Leena 

Alhonen, Jarmo Wahlfors 

309 

STAT5 Phosphorylation in Malignant Melanoma Is 

Mediated through SRC and JAK-1 Kinases and Exerts 

Anti-Apoptotic Effects 

Alireza Mirmohammadsadegh, Walter Bardenheuer, Mohamad 

Hassan, Annett Gustrau, Dennis Selimovic, Roya Doroudi, 

Nicola Schmittner, Sandeep Nambiar, Alessandra Marini, 

Thomas Ruzicka Ulrich R. Hengge 

Cancer - Targeted Gene Therapy: Targeting of Non- 

Viral Vectors 

310 

Discovered Peptide Mediates Specific Binding to Primary 

Medullary Thyroid Carcinoma after Systemic Injection 

Miriam Bockmann, Gero Hilken, Aaron N. Cranston, 

Matthias Drosten, Bruce A. J. Ponder, Anke Schmidt, Brigitte 

M. Putzer 

311 

Oncolytic Adenovirus CG5757 Preferentially Infects 

Primary Human Tumor Tissues and Shows Strong Anti- 

Tumor Activity in Tumor Models Following Systemic 


Yuanhao Li, Trini Arroyo, Stephen Thorne, Tony Reid, Natalie 

Nguyen, Neeraja Idamakanti, Melinda VanRoey, Gail Colbern, 

De-Chao Yu 

312 

Human Plasminogen Kringle 5-Engineered Murine 

Mammary Cancer Cells Arrest Tumor Growth and 

Promote Long-Term Survival 

Sabrina R. Perri, Moïra François, Jacques Galipeau 

313 

Efficacy of Group I Intron-Based Trans-Splicing 

Ribozyme in Tumor-Targeted Gene Therapy 

Byung-Su Kwon, Ju Hyun Kim, Jin-Sook Jeong, In-Hoo 

Kim, Seong-Wook Lee 

314 

The Epithelial Glycoprotein-2 Promoter for Highly 

Specific and Efficient Cancer Gene Therapy for a Broad 

Range of Tumors 

Willemijn M. Gommans, Gerben Duns, Pamela M. J. 

McLaughlin, Simone J. van Eert, Masato Yamamoto, David T. 

Curiel, Hidde J. Haisma, Marianne G. Rots 

315 

Highly Specific Expression of the Luciferase Gene in 

Lungs of Naïve Nude Mice Directed by Prostate Specific 

Antigen Promoter 

Hongwei Li, Jin Zhong Li, Gregory A. Helm, Dongfeng Pan 

316 

Dendritic Cell-Based Genetic Immunotherapy for 

Prostate Cancer 

Briana J. Williams, Nikolay Korokhov, Susan Boling, Linda 

Li, Michael Mathis, David T. Curiel 

317 

Raf-1 siRNA Inhibits Tumor Growth In Vivo 

Qixin Leng, A. James Mixson 

318 

Tumor Growth Inhibition from Tumor Targeted Delivery 

of Diphtheria Toxin Gene 

Puthupparampil V. Scaria, Qin Zhou, Raymond M. Schiffelers, 

Aslam Ansari, Martin C. Woodle


ABSTRACTS 


319 

ColoAd1, a Chimeric Ad11p/Ad3 Oncolytic Virus for the 

Treatment of Colon Cancer 

Irene Kuhn, Paul Harden, Maxine Bauzon, Terry Hermiston 

320 

Antitumor Effect of Intrahepatic Interleukin-12 Expression 

from a Regulable Plasmid Vector in Murine Models 

of Primary and Metastatic Liver Cancer 

Maider Zabala, Ruben Hernandez-Alcoceba, Margarita Ecay, 

Ignacio Melero, Christine Perret, Ivan Penuelas, Jesus Prieto, 

Gabriela M. Kramer 

321 

Splice-Switching of the Prostate Specific Membrane 

Antigen (PSMA) in LnCap Prostate Cancer Cells 

Tiffany L. Williams, Ryszard Kole 

322 

Tumor Specific Activation of Irinotecan by a 

Carboxylesterase Fused with Anti CEA scFv 

Junji Uchino, Koichi Takayama, Tomomichi Sone, Akiko 

Harada, Taishi Harada, David T. Curiel, Yoichi Nakanishi 

323 

Transcriptional Targeting of Viral Gene Transfer by 

Human Hexokinase II Promoter 

Ann-Marie Maatta, Sanna Korja, Tanja Hakkarainen, Jarmo 

Wahlfors, Kimmo Mäkinen, Esko Alhava, Riikka Pellinen 

324 

TRAIL Gene Therapy Is a Promising Therapy for 

Bladder Cancer 

Ahmed M. El-Zawahry, Christina Voelkel-Johnson 

325 

Identification of Genes Expressed Specifically in Esophageal 

Cancer Cells 

Fumiaki Sati, John M. Abraham, Jing Yin, Takatsugu Kan, 

Yuriko Mori, Stephen J. Meltzer 

326 

Gene Therapeutic Overexpression of MDR1 Affects 

Transcriptional Regulations of Proteins Involved in 

Apoptosis and Detoxification 

Patrick Maier, Li Li, Katharina Fleckenstein, Stephanie Laufs, 

Stefan Fruehauf, Jens Zeller, Frederik Wenz, Carsten Herskind 

327 

The Effect of NO on HIF-Mediated Gene Expression: 

Chemical Sources of NO Versus Genetic Regulation of 

iNOS 

Rana S. Al-Assah, Rachel L. Cowen, Edwin C. Chinje, Ian J. 

Stratford 


Session I to Poster Session III and will follow Abstract 

1029.) 

328 

An Orthotopic Murine Model of Advanced Breast 

Cancer for Imaging and Comparison of Targeting 

Moieties 

Tuuli Ranki, Anna Kanerva, Karl von Smitten, Merja Sarkioja, 

Lotta Kangasniemi, Mari Raki, Pirjo Laakkonen, Hongju Wu, 

David T. Curiel, Steven Goodison Akseli Hemminki 

329 

Use of Recombinant Non-Pathogenic Bacteria as Vectors 

for Hypoxia Targeted Gene Expression for Cancer Gene 


Asferd Mengesha, Ludwig Dobois, Brad G. Wouters, Phillipe 

Lambin, Jan Theys 

Hematologic - Disease Models 

330 

Lentiviral Vector Mediated Hematopoietic Stem Cell 

Gene Therapy Combined with Non-Lethal Conditioning 

Restores T Cell Function in the Murine Model of 

Wiskott-Aldrich Syndrome 

Francesco Marangoni, Loïc Dupré, Samantha Scaramuzza, 

Sara Trifari, Raisa Jofra Hernández, Adrian Thrasher, Anne 

Galy, Alessandro Aiuti, Luigi Naldini, Maria Grazia Roncarolo 

331 

Lentivirus-Mediated Ex Vivo Gene Therapy in ADA- 

Deficient SCID Mice 

Alessandra Mortellaro, Raisa Jofra Hernández, Matteo Guerrini, 

Antonella Tabucchi, Filippo Carlucci, Antonia Follenzi, Luigi 

Naldini, Claudio Bordignon, Maria Grazia Roncarolo, 

Alessandro Aiuti 

332 

Chronic Granulomatous Disease Gene Therapy Functionally 

Corrects the Phenotype of Polymorphonuclear 

Leukocytes (PMN) 

Ulrich Siler, Marion G. Ott, Stefan Stein, Elke Karaus, Maja 

Rutishauser, Corinne Wenk, Dieter Hoelzer, Manuel Grez, 

Reinhard Seger 

333 

Preferential Targeting of Transcriptional Start Sites after 

Retroviral-Mediated T-Cell Gene Therapy for Adenosine 

Deaminase Deficiency 

Daniele Moratto, Jayashree G. Jagadeesh, Akihiro Konno, 

Marita Bosticardo, Gregory E. Crawford, Ingeborg Holt, Linda 

M. Muul, Shepherd H. Shurman, Tyra Wolfsberg, Fabio 

Candotti 

334 

Treatment of Anemia in Mice with Chronic Renal Failure 

Utilizing Erythropoietin-Secreting Genetically-Engineered 

Murine Bone Marrow Stromal Cells 

Nicoletta Eliopoulos, Raymonde F. Gagnon, Moira Francois, 

Jacques Galipeau

66 


ABSTRACTS 


335 

Correction of a Murine Model of Von Willebrand 

Disease by Gene Transfer 

Robert G. Pergolizzi, Jin Guang, Diane Chan, Denisa Wagner, 

Peter Lenting, Ronald G. Crystal 

336 

Correction of Chronic Granulomatous Disease by Gene 


Manuel Grez, Marion G. Ott, Stefan Stein, Ulrich Siler, 

Ulrike Koehl, Hana Kunkel, Andrea Schilz, Klaus Kuehlcke, 

Dieter Hoelzer, Reinhard Seger 

337 

Towards Gene Correction Therapy for Wiskott-Aldrich 

Syndrome with Engineered Zinc Finger Nucleases 

Lei Zhang, Fyodor D. Urnov, Jeffrey C. Miller, Christian 

Beausejour, Ya-Li Lee, Jeremy Rock, Kenneth Kim, Michael C. 

Holmes, Philip D. Gregory 

338 

Non-Viral Gene Transfer into Hematopoietic Cells of 

Artemis Deficient ScidA Mice Using the Sleeping Beauty 

Transposon System 

Andy Wilber, Shannon Buckley, Uma Lakshmipathy, Joel 

Frandsen, Morton J. Cowan, R. Scott McIvor 

339 

Long-Term Protein Transduction Domain Mediated 

Intra-Cellular Delivery of Purine Nucleoside Phosphorylase 

(PNP) Corrects PNP Deficiency in Mice 

Ana Toro, Eyal Grunebaum 

340 

Drug-Selectable as Well as Therapeutic Gene Transfer 

into Hematopoietic Stem Cells May Be Essential in Gene 

Therapy for Inherited Bleeding Disorders of Large 

Animals 

David A. Wilcox, Juan Fang, Lily M. Du, Mary K. Boudreaux 

341 

Lentiviral Vector-Mediated Gene Therapy as Treatment 

for Wiskott-Aldrich Syndrome (WAS): Pre-Clinical 

Studies in Human Cell Lines and WASp -/- Mice 

S. Humblet-Baron, S. Anover, K. Kipp, Q. Zhu, P. Ye, W. 

Zhang, Y. Ovechkina, S. Khim, A. Astrakhan, T. Strom D. 

Kohn, F. Candotti, Y. Vyas, H. Ochs, C. Miao, D. Rawlings 

342 

Human Erythropoietin Gene Therapy for Patients with 

Chronic Renal Failure 

Yitzhak Lippin, Michal Dranitzki-Elhale, Einat Brill-Almon, 

Chava Mei–Zahav, Sarah Mizrachi, Yael Liberman, Adrian 

Iaina, Eli Kaplan, Eduardo Podjarny, Nicole Casadevall Noam 

Shani, Eithan Galun 

343 

Repair of Thalassemic bð-Globin pre-mRNA by 

Antisense AAV and Lentiviral Vectors in Cell Culture 

Thipparat Suwanmanee, Vivian W. Choi, Richard J. Samulski, 

Tal Kafri, Ryszard Kole 

344 

Application of a suicide Gene to X-SCID Gene Therapy 

Toru Uchiyama, Satoru Kumaki, Masahumi Onodera, Du Wei, 

Looi Chung Yeng, Yoichi Sasahara, Sigeru Tsuchiya 

345 

Gene Therapy for Wiskott-Aldrich Syndrome Using 

Lentiviral Vectors: Evidence for Efficacy and Safety after 

Transduction of Human T Cells and Hematopoietic Stem 


Loïc Dupré, Samantha Scaramuzza, Francesco Marangoni, 

Sara Trifari, Silvana Martino, Shigeru Tsuchiya, Adrian 

Thrasher, Anne Galy, Luigi Naldini, Alessandro Aiuti Maria 

Grazia Roncarolo 

346 

Gene Correction of X-Linked SCID Using Engineered 

Zinc Finger Nucleases and Integration Defective 

Lentiviral Delivery 

Angelo Lombardo, Christian Beausejour, Fyodor D. Urnov, 

Jeffrey C. Miller, Michael C. Holmes, Philip D. Gregory, Luigi 

Naldini 

347 

Long-Term Gene Expression and Phenotypic Correction 

of FANCC Deficient Lymphoblastoid Cells Using the 

Sleeping Beauty Transposon System 

Shannon A. Wadman, Karl J. Clark, Perry B. Hackett, R Scott 

McIvor, Jeffrey J. Essner 

348 

Integration Site Analysis of a Helper-Dependent Adenovirus 

Vector Containing AAV ITRs and 22 kb of the 

Human Globin LCR 

Hongjie Wang, Tobias Leege, Dmitry Shayakhmetov, Qiliang 

Li, Thalia Papayannopoulou, George Stamatoyannopoulos, 

Andre Lieber 

349 

Partial Correction of IL-12 Receptor beta-1 (IL-12Rb1) 

Deficiency in Mice upon Transplantation of Retrovirally 

Transduced Hematopoietic Stem Cells 

Marita Bosticardo, Charles Scanga, Francesco Novelli, Jean- 

Laurent Casanova, Fabio Candotti 


350 

Pre-Clinical Assays for Cystic Fibrosis Gene Therapy 

Uta Griesenbach, on behalf of the UK Cystic Fibrosis Gene 

Therapy Consortium


ABSTRACTS 


351 

Repeat Administration of Polyethylenimine (PEI) 

Aerosols of Plasmid DNA to the Murine Lung Is Associated 

with a Loss of Gene Transfer Efficiency 

Lee A. Davies, Stephen C. Hyde, Deborah R. Gill 

352 

Gene Transfer of Angiogenic Inhibitors Suppresses Lung 

Tumor Growth in Spontaneous Pulmonary Metastasis 

Mouse Model 

Kexia Cai, Mai Har Sham, Weidong Xiao, Paul Tam, Ruian 

Xu 

353 

Retrovirally-Marked Human Bone Marrow Derived 

Mesenchymal Stem Cells Attenuate Radiation Induced 

Pneumonitis in a Xenotransplant Model 

Andrew Hope, Todd E. Meyerrose, Jan A. Nolta 

354 

Correction of Respiratory Function by Recombinant 

AAV1 Mediated Gene Therapy in a Murine Model of 

Glycogen Storage Disease Type II 

Cathryn Mah, Kerry O. Cresawn, Lara R. DeRuisseau, David 

Fuller, Barry J. Byrne 

355 

Towards Cystic Fibrosis and Airway Gene Therapy: 

Evaluation of EGFP Gene Expression in Murine Nasal 

Airways Mediated by Simian Immunodeficiency Virus 

Vectors Pseudotyped with Sendai Virus Glycoproteins F 

and HN 

Katsuyuki Mitomo, Makoto Inoue, Yasuji Ueda, Mie Ikegami, 

Uta Griesenbach, Eric W. F. W. Alton, Mamoru Hasegawa 

356 

Sonoporation Increases Non-Viral Gene Transfer to the 

Murine Lung 

Stefania Xenariou, Haidong Liang, Uta Griesenbach, Ray 

Farley, Luci Somerton, Charanjit Singh, Po-Wah So, Amy 

Herlihy, Stefano Ferrari, Duncan Geddes Martin Blomley, Eric 

Alton 

357 

IL-17 Induces Migration of Adult Marrow Stromal Cells 

to Lung Epithelium 

Roberto Loi, Jennifer Jaskolka, Robert Prenovitz, Ying Jian You, 

Steven Brody, Daniel J. Weiss 

358 

Gene Transfer to the Airway Epithelium Mediated by a 

Third Generation HIV-1 Based Vector: Efficiency and 

Role of Heparan Sulfate 

Elena Copreni, Stefano Castellani, Lucia Palmieri, Salvatore 

Carrabino, Luigi Naldini, Massimo Conese 

359 

Specific Modulation of Tight Junctions to Enhance 

Airway Gene Transfer 

Ruth S. Everett, Miriam K. Vanhook, Larry G. Johnson 

360 

Disturbance of Airway Epithelium Is Reduced, Whilst 

Lentivirus-Mediated Gene Transfer Is Maintained, after 

Low Dose Lysophosphatidylcholine Pre-Treatment In 

Vivo 

Patricia Cmielewski, Alice G. Stocker, David W. Parsons, 

Donald S. Anson 

361 

Radiation-Induced Lung Apoptosis Is Mediated by TNFalpha 

Action 

Ming Zhang, Jun Qian, Ming Chen, Spring Kong, Theodore 

S. Lawrence 

362 

Using Real-Time (TaqMan ® ) PCR to Genotype Offspring 

of Transgenic CF-null Mice – A Core Facility of 

the UK Cystic Fibrosis Gene Therapy Consortium 

Rebecca L. Smith, Lee A. Davies, Hazel Painter, Anusha 

Varathalingam, Deborah R. Gill, Stephen C. Hyde 

363 

Gene Delivery to Human Sweat Glands: A Model for 

Cystic Fibrosis Gene Therapy 

Haeyul Lee, David R. Koehler, Cho Y. Pang, Ronald H. 

Levine, Philip Ng, Donna J. Palmer, Paul M. Quinton, Jim 

Hu 

364 

Prevention of Radiation Pneumonitis by Recombinant 

Adenovirus-Mediated Transfer of Soluble TGF-bðtype II 

Receptor Gene 

Koichi Takayama, Haiping Zhang, Junji Uchino, Akiko 

Harada, Taishi Harada, Yoichi Nakanishi 

365 

Aerosol Delivery of an Enhanced Helper-Dependent 

Adenovirus Formulation to Rabbit Lung Using an 

Intratracheal Catheter 

David R. Koehler, Helena Frndova, Kitty Leung, Emily Louca, 

Donna Palmer, Philip Ng, Colin McKerlie, Peter Cox, Allan L. 

Coates, Jim Hu 

366 

Development of Quantitative TaqMan RT-PCR for the 

Evaluation of Non-Viral Mediated Gene Transfer to the 

Airways 

Ian A. Pringle, Rebecca L. Smith, Bryony L. Jones, Deborah R. 

Gill, Stephen C. Hyde

68 


ABSTRACTS 


367 

Safety and Immunological Responses Following Intrapleural 

Administration of an AAV5 Vector Encoding 

Human α1-Antitrypsin to Non-Human Primates 

Ben-Gary Harvey, Bishnu P. De, John P. Ayala, Neil R. 

Hackett, Adriana Heguy, Ronald Crystal 

Gene Regulation: Gene Targeting and RNA 

368 

Differential Sensitivity of Normal and CML-Derived 

CD34+ Cells to Inhibition of SHP2, Gab2 and Stat5 

Gene Expression by RNA Interference (RNAi) 

Michaela Scherr, Karin Battmer, Anuhar Chaturvedi, Beate 

Schultheis, Arnold Ganser, Matthias Eder 

369 

Targeted Gene Modification Via Triple Helix-Forming 

Oligonucleotides 

Peter M. Glazer, Faye Rogers, Janice Lloyd 

370 

Multi-Copy, Multi-siRNA Vectors as Versatile Tools for 

Multiple Gene Knock-Down Applications 

Laura Poliseno, Monica Evangelista, Mauro Giacca, Giuseppe 

Rainaldi 

371 

Optimizing Custom Zinc-Finger Nucleases for Use in 

Human Cells 

Stephen Alwin, Maja B. Gere, Eva Guhl, Karin Effertz, Carlos 

F. Barbas III, David J. Segal, Matthew D. Weitzman, Toni 

Cathomen 

372 

Targeting of Non-Viral Vectors to Specific Subnuclear 

Domains 

Joshua Z. Gasiorowski, David A. Dean 

373 

Use of Lentiviral and Adeno-Associated Vectors for 

Targeted Gene Inactivation in Human Cancer Cells 

Sabrina Arena, Elisa Vigna, Luigi Naldini, Alberto Bardelli 

374 

Knockdown of Shp-2 Expression by siRNA in Mouse 

Embryonic Stem Cell Derived EB Cells Affects Their 

Hematopoietic Development 

Gang-Ming Zou, Rebecca J. Chan, W. Christopher Shelley, 

Mervin C. Yoder 

375 

Targeted Gene Repair Induces the DNA Damage 

Response Pathways through the Activation of ATM, and 

the Stalling of Replication Forks Leading to a Blockage of 

Inherited Correction 

Luciana Ferrara, Julia Engstrom, Erin Brachman, Miya Drury, 

Eric B. Kmiec 

376 

Various Aspects of the Single-Stranded DNA Fragment- 

Mediated Gene Correction 

Hiroyuki Tsuchiya, Hideyoshi Harashima, Hiroyuki Kamiya 

377 

Developing siRNAs to the E2Fs to Control Vascular 

Growth and Remodeling 

Paloma H. Giangrande, JianXin Zhang, Juliana Layzer, Otto 

Hagen, Joseph R. Nevins, Bruce A. Sullenger 

378 

Retargeting Mobile Group II Introns to Repair Mutant 

Genes 

Monique N. Kierlin, John P. Jones III, Rob G. Coon, Jiri 

Perutka, Alan M. Lambowitz, Bruce A. Sullenger 

379 

New Zinc Finger Protein Nuclease Architectures for 

More Efficient Gene Modification Therapies 

Jeffrey C. Miller, Lei Zhang, Fyodor D. Urnov, Sarah Patterson, 

Danny Xia, Jeremy Rock, Sheldon Augustus, Carl O. Pabo, 

Philip D. Gregory, Edward J. Rebar Michael C. Holmes 

380 

Studies on the Mechanism of Gene Repair Using 

Saccharomyces cerevisae as a Model System 

Katie Maguire, Hetal Parekh-Olmedo, Eric B. Kmiec 

381 

Methods for Optimizing Lentiviral Vector-Mediated 

Gene Suppression by RNA Interference 

Eddy Kizana, Genaro Ramirez, Eugenio Cingolani, Roselle 

Abraham, Eduardo Marban 

382 

Expanding the Repertoire of Heterospecific LoxP Sites 

for Cre-Mediated Site-Specific Recombination 

Stephen J. Langer, Jarred Keith, Leslie A. Leinwand 

383 

Zinc Finger Nuclease-Boosted Gene Targeting & 

Synergistic Transient Regenerative Stem Cell Gene 

Therapy: Toward Clinical Gene Repair/Alteration & 

Custom Site-Specific Integrative Gene Therapy 

Roger Bertolotti 

384 

Double-Stranded DNA Breaks Stimulate the Frequency 

of Targeted Gene Repair in Mammalian Cells 

Timothy Schwartz, Luciana Ferrara, Hetal Parekh-Olmedo, 

Eric B. Kmiec 

385 

Gene Correction in the Absence of DNA Repair: A 

Universal Model Involving Homologous Pairing and 

DNA Replication 

Hetal Parekh-Olmedo, Erin Brachman, Eric B. Kmiec


ABSTRACTS 



RNA Virus Vectors: Integration and Vector Safety 


386 2:00 PM 

Large Scale Analysis of Foamy Virus Vector Integration 

Sites in Human CD34+ Cells 

Grant Trobridge, Daniel G. Miller, Michael A. Jacobs, James M. 

Allen, Erik Olson, Hans-Peter Kiem, Rajinder Kaul, David W. 

Russell 

387 2:15 PM 

Myeloid Sarcoma Associated with Gene Marking of 

Hematopoietic Stem Cells in a Rhesus Macaque 

Ruth Seggewiss, Stefania Pittaluga, Rima Adler, Cole Ferguson, 

Elio F. Vanin, Patrick F. Kelly, Robert E. Donahue, Brian P. 

Sorrentino, Arthur W. Nienhuis, Cynthia E. Dunbar 

388 2:30 PM 

Use of the cHS4 Chromatin Insulator to Reduce 

Oncoretrovirus Vector-Mediated Genotoxicity 

David W. Emery, Changlong Li, George Stamatoyannopoulos 

389 2:45 PM 

Testing the Oncogenic Potential of Retroviral and 

Lentiviral Vector Integration 

Eugenio Montini, Daniela Cesana, Francesca Sanvito, Sergi 

Sergi Lucia, Benedicenti Fabrizio, Ponzoni Maurilio, Claudio 

Doglioni, Luigi Naldini 

390 3:00 PM 

Flanking a Retrovirus Vector with the cHS4 Chromatin 

Insulator Reduces the Frequency of Vector-Mediated 

Trans-Activation of Endogenous Genes 

Changlong Li, David W. Emery 

391 3:15 PM 

Effective Gene Therapy with Non-Integrating Lentivirus 

Vectors 

Rafael J. Yáñez-Muñoz, Kamaljit S. Balaggan, Angus 

MacNeil, Alexander J. Smith, Prateek Buch, Steven Howe, 

Yanaí Durán, Robert E. MacLaren, Patrick N. Anderson, 

Christine Kinnon Robin R. Ali, Adrian J. Thrasher 

392 3:30 PM 

Development of a New Lentiviral Vector for Site-Specific 

Integration into Mammalian Genomes 

Angelo Lombardo, Michelle Calos, Luigi Naldini 

393 3:45 PM 

Site-Directed Integration in Human Genome by 

Lentiviral Vectors Containing HIV-1 Integrase/Polydactyl 

Zinc Finger Fusion Proteins 

Wenjie Tan, Zheng Dong, Vivian Luo, Samson A. Chow 

394 4:00 PM 

Oncogenesis Following Delivery of a Non-Primate 

Lentiviral Gene Therapy Vector to Fetal Mice 

Michael Themis, Simon N. Waddington, Manfred Schmidt, 

Christof von Kalle, Yoahe Wang, Faisal Al-Allaf, Lisa Gregory, 

Megha Nivsarkar, Matthew Themis, Maxine Holder Suzanne 

M. K. Buckley, Niraja Dighe, Alaine Ruthe, Ajay Mistry, Brian 

Bigger, Adrian J. Thrasher, Charles Coutelle 

395 4:15 PM 

Safety of In Utero Gene Delivery of Lentiviral Vectors 

Susan M. Kingsman, James E. Miskin, Pippa A. Radcliffe, 

Caroline V. Hacker, Daniel Chipchase, Nicholas D. Mazarakis, 

Kyriacos A. Mitrophanous 




396 2:00 PM 

Identification and Characterization of Novel AAV Isolates 

Michael K. Schmidt, Emmanuelle Grott, Charles Buck, John A. 

Chiorini 

397 2:15 PM 

AAV-Mediated Gene Transfer to Endothelial Cells 

Mo-Ying Hsieh, Yi-Lin Liu, Alexander Schlachterman, 

Katherine A. High 

398 2:30 PM 

Adeno-Associated Virus Serotype 8 Efficiently Crosses 

Blood Vessel Barrier in Muscle and Heart for Whole- 

Body Gene Delivery 

Zhong Wang, Tong Zhu, Chunping Qiao, Liqiao Zhou, Bing 

Wang, Jian Zhang, Chunlian Chen, Juan Li, Xiao Xiao 

399 2:45 PM 

rAAV2 Movement Through the Late and Recycling 

Endosomes Is Influenced by Titer of Infection 

Liang Zhang, Wei Ding, John F. Engelhardt 

400 3:00 PM 

Successful Production of Pseudotyped Mosaic rAAV 

Vectors Using a Modified Baculovirus Expression System 

Erik Kohlbrenner, George Aslanidi, Kevin Nash, Stanislav 

Shklayev, Martha Campbell-Thompson, Barry J. Byrne, 

Richard O. Snyder, Nicholas Muzyczka, Kenneth H. 

Warrington, Sergei Zolotukhin 

401 3:15 PM 

Evaluation of Primitive Murine Hematopoietic Stem and 

Progenitor Cell Transduction In Vitro and In Vivo by 

Recombinant Adeno-Associated Virus Vectors Based on 

Serotypes 1 through 5 

Li Zhong, Weiming Li, Yanjun Li, Keyun Qing, Mervin C. 

Yoder, Arun Srivastava

70 


ABSTRACTS 


402 3:30 PM 

Comparison of Pseudotyped rAAV Vectors for Transduction 

of Primary Hematopoietic Stem and Progenitor 

Cells Assayed In Vitro and In Vivo 

Lijing Li, Sarah Hardy, Leslie Smith-Powell, Sai Srinivas, 

Stephen J. Forman, K. K. Wong, Saswati Chatterjee 

403 3:45 PM 

Defined Vector Substrates Used To Probe rAAV DNA 

Integration 

Douglas M. McCarty, Vivian Choi, Alda Fernandes, R. J. 

Samulski 

404 4:00 PM 

Roles of Inverted Terminal Repeats (ITRs) and Capsid 

Proteins from Novel NHP AAVs in rAAV Mediated 


Xiaoyang Zhou, Guangping Gao, Roland A. Owens, You Lu, 

Roberto Calcedo, Jim Miller, James M. Wilson 

405 4:15 PM 

Comparison Study of Structural Determinants for AAV 

Liver Transduction by Domain Swapping between AAV-2 

and AAV-8 Capsids 

Xuan Shen, Hui Xu, Zan Huang, Theresa A. Storm, Mark A. 

Kay 


Adenovirus Vectors: Innate Immunity 

Room: 120/124 

406 2:00 PM 

Role of MyD88 Signaling Pathway in the Host Immune 

Responses in Adenoviral Vector-Mediated Gene Therapy 

Zhe Zhang, Guang Ping Gao, Lee M. Wetzler, Yan Zhi, James 

M. Wilson 

407 2:15 PM 

Complement Blockade Prevents Several Innate Toxicities 

Rapidly Induced after Intravenous Adenovirus (Ad) 

Vector Administration 

Anne Kiang, Zachary Hartman, Jun-Ping Wei, Ruth Everett, 

Delila Serra, Haixiang Jiang, Michael M. Frank, Andrea 

Amalfitano 

408 2:30 PM 

The Kupffer Cell Scavenger Receptor Is Accountable for 

Hepatic Sequestration of Adenovirus 

Hidde J. Haisma, Anna Rita Bellu, Marianne G. Rots, Gera 

Kamps, Josee Plantinga, Jan Kamps 

409 2:45 PM 

Identifying Functional Adenovirus-Host Interactions 

Using Tandem Mass Spectrometry 

Anuj Gaggar, Martin Sadilek, Andre Lieber, Dmitry M. 

Shayakhmetov 

410 3:00 PM 

Adenovirus Activates Mouse Platelets and Induces 

Platelet Leucocyte Association 

Maha Othman, Andrea Labelle, David Lillicrap 

411 3:15 PM 

Trapping of Adenovirus Vectors in Blood 

Daniel Stone, Shaoheng Ni, Zong-Yi Li, Dmitry M. 

Shayakhmetov, Andre Lieber 

412 3:30 PM 

Renal Pathophysiology after Systemic Administration of a 

Recombinant Adenovirus: Changes in Drug Metabolism 

Based upon Vector Dose 

Michael P. Boquet, Hong T. Le, Erin A. Clark, Shellie M. 

Callahan, Maria A. Croyle 

413 3:45 PM 

Functional Differences of the Reticuloendothelial System 

in C57BL/6 and Balb/c Mice Mediate Differential Effects 

of Kuppfer Cell Blockade, Rag-Deficiency and Splenectomy 

on Transgene Expression after Adenoviral Transfer 

Jan Snoeys, Geertrui Mertens, Joke Lievens, Désiré Collen, Erik 

Biessen, Bart De Geest 

414 4:00 PM 

Vitamin B : A Key Component for Controlled Inactiva- 

2 

tion of Viruses Suitable for Biological Use 

Shellie M. Callahan, Piyanuch Wonganan, Linda J. Obenauer- 

Kutner, Suganto Sutjipto, Hong T. Le, Maria A. Croyle 

415 4:15 PM 

Evaluating Polyethylene Glycol (PEG) Modified Adenoviral 

Vectors for Cell Targeting and Detargeting and 

Reducing Vector Toxicity 

Hoyin Mok, Michael A. Barry 


Inborn Errors of Metabolism - Liver and Storage 

Disorders 

Room: 260/264 

416 2:00 PM 

Glucose-Dependent Insulin Production by Liver- 

Specific, Glucose-Regulatable Synthetic Promoters 

Results in the Cure of Diabetes 

Jaeseok Han, Brienne McLane, Hee-Sook Jun, Ji-Won Yoon 

417 2:15 PM 

Meganuclease-Mediated Chromosomal Surgery in Living 

Animals 

Agnes Gouble, Julianne Smith, Christophe Perez, Valerie 

Guyot, Jean-Pierre Cabaniols, Sophie Leduc, Laurence Fiette, 

Patrick Ave, Beatrice Micheaux, Philippe Duchateau Frederic 

Paques


ABSTRACTS 


418 2:30 PM 

rAAV-Mediated Expression of Adiponectin Receptor 

(AdipoR2) cDNA in Muscle Prevents the Development 

of Obesity in DIO Rats 

George Aslanidi, Stanislav Shklayev, Erik Kohlbrenner, Vadim 

Kroutov, Glenn Walter, Martha Campbell-Thompson, Sergei 

Zolotukhin 

419 2:45 PM 

Generation of Transgene Product-Specific Regulatory 

CD4+CD25+ T Cells by Hepatic AAV Gene Transfer 

O. Cao, E. Dobrzynski, B. L. Mingle, L. Wang, R. W. Herzog 

420 3:00 PM 

Tropism-Modified Adeno-Associated Virus Vector 

Mediates Targeting of Brain Vascular Endothelium In Vivo 

Yong Hong Chen, Beverly L. Davidson 

421 3:15 PM 

Liver Contains Sufficient Sulfatase Modifying Factor for 

4-Sulfatase and Correction of MPS VI with Neonatal 

Retroviral Gene Therapy in Cats 

Mark Haskins, Thomas O’Malley, Bin Wang, Jason Metgcalf, 

Ping Wang, John Hopwood, Katherine P. Ponder 

422 3:30 PM 

Dominant-Negative Interference in the Pah enu2 Mouse 

Model of PKU: Effectiveness of Vectors Expressing 

Either Modified Forms of Phenylalanine Hydroxylase 

(PAH) or Ribozymes Plus a Hardened PAH mRNA 

Catherine E. Charron, O. P. Perera, Stacy L. Porvasnik, Mandy 

Blackburn, Alfred S. Lewin, Philip J. Laipis 

423 3:45 PM 

Increased Incidence of Hepatocellular Change and 

Neoplastic Disease in AAV-WPRE Treated Pah enu2 Mice 

Jennifer E. Embury, Catherine E. Charron, Philip J. Laipis 

424 4:00 PM 

Intracranial AAV5 Synergizes with Non-Myeloablative 

Bone Marrow Transplantation in the Murine Model of 

Globoid-Cell Leukodystrophy 

Darshong Lin, Anthony Donsante, Shannon Macauley, Beth 

Levy, Carole Vogler, Mark Sands 

425 4:15 PM 

AAV2-Mediated Expression of Iduronate Sulfatase in 

Peripheral Tissues and the CNS in 

Mucopolysaccharidosis II Mouse Model by Combined 

Intravenous and Intracisternal Vector Delivery 

Lu Kang, Haiyan Fu, Jerillyn S. Jennings, Joseph Muenzer 


Neurologic Disorders 

Room: 265/266 

426 2:00 PM 

Cure of Established Disease and Direct Evidence of 

Cellular Cross-Correction in the Nervous System of 

Metachromatic Leukodystrophy Mice after HSC-Based 


Alessandra Biffi, Alessia Capotondo, Angelo Quattrini, Ubaldo 

Del Carro, Riccardo Brambilla, Stefania Fasano, Sergio 

Marchesini, Claudio Bordignon, Luigi Naldini 

427 2:15 PM 

AAV2- and AAV5-Mediated CNS Delivery of Human 

CLN2 Reduces Lysosomal Storage in a Mouse Model of 

Late Infantile Neuronal Ceroid Lipofuscinosis 

Marco A. Passini, Dolan Sondhi, James C. Dodge, Jie Bu, 

Wendy Yang, Neil R. Hackett, Stephen M. Kaminsky, 

Mukarram El-Banna, Qinwen Mao, Beverly L. Davidson 

David E. Sleat, Peter Lobel, Ronald G. Crystal, Gregory R. 

Stewart 

428 2:30 PM 

A Novel Approach for the Treatment of the Neurological 

Symptoms of MPS VII (Sly Disease) 

Brian J. Spencer, Inder M. Verma 

429 2:45 PM 

AAV5-Mediated Delivery of Human Aryl Sulfatase A 

(hARSA) Prevents Sufatide Storage and Neuropathological 

Phenotype in Metachromatic Leukodystrophy (MLD) 

Mice 

Nathalie Cartier, Caroline Sevin, Abdellatif Benraiss, Peter 

DeDeyn, Delphine Bonnin, Marie-T Vanier, Moullier Philippe, 

Volkmar Gieselmann, Patrick Aubourg 

430 3:00 PM 

Immune System Regulation of Transgene Expression 

from High-Capacity Gutless Adenoviral Vectors in the 

Mouse Brain: Very Long Term Expression Following Pre- 

Immunization 

Carlos Barcia, Jeffrey M. Zirger, Weidong Xiong, Philip Ng, 

Donna Palmer, Maria G. Castro, Pedro R. Lowenstein 

431 3:15 PM 

In Utero CNS Delivery of an Integrating HSV-1 

Amplicon Vector Leads to Prolonged, Neuron-Specific 

Gene Expression 

William J. Bowers, Michael A. Mastrangelo, Ann E. Casey, 

Hilary A. Southerland, Kathleen A. Maguire-Zeiss, Howard J. 

Federoff 

432 3:30 PM 

Molecular Chimerism Prevents Experimental Autoimmune 

Encephalomyelitis (EAE) 

Herena Eixarch, Carmen Espejo, Francisco Vidal, Silvia Garces, 

Mireia Castillo, Alex Bote, Elisabeth Kadar, Marta Rosal, 

Xavier Montalban, Jordi Barquinero

72 


ABSTRACTS 


433 3:45 PM 

Gene Therapy of Epilepsy by Adenovirus-Mediated 

Tetanus Toxin Light Chain Gene Transfer 

Jun Yang, Qingshan Teng, Mary E. Garrity-Moses, Thais 

Federici, Imad Najm, Stephan Chabardes, Michael Moffitt, 

Nicholas M. Boulis 

434 4:00 PM 

Long-Term Effects on Retinal Function and Structure 

Using AAV-Mediated Gene Therapy in Blind RPE65 

Null Mutation Dogs 

Kristina Narfstrom, Greg Tullis, Ragnheidur Bragadottir, 

Mathias Seeliger, Elizabeth P. Rakoczy, Martin L. Katz 

435 4:15 PM 

Functional and Morphological Rescue of the Type I 

Ocular Albinism Murine Retina Following AAV-Mediated 

Gene Transfer. 

Enrico M. Surace, Luciano Domenici, Katia Cortese, Consuelo 

Venturi, Gabriella Cotugno, Umberto Di Vicino, Alessandro 

Cellerino, Valeria Marigo, Carlo Tacchetti, Andrea Ballabio 

Alberto Auricchio 


Infectious Diseases and Vaccines: RNAi in Gene 

Therapy for Infections 

Room: 230/231 

436 2:00 PM 

shRNAs Target HIV-1 Vif Gene Coding for SOCS-Box 

Motif Prevent Virus Escape from RNA Interference- 

Mediated Inhibition 

Jianfei Ji, John J. Rossi 

437 2:15 PM 

Alternative Strategies To Deliver Multiple shRNAs in 

Order to Simultaneously Inhibit Multiple Targets 

Peter W. Roelvink, Michael W. Graham, David A. Suhy, Linda 

B. Couto, Sara M. Cunningham, Mark A. Kay, John J. Rossi, 

Alexander A. Kolykhalov 

438 2:30 PM 

Development of an siRNA Based Therapy for Hepatitis 

Virus Infection 

David V. Morrissey, Karin Blanchard, Lucinda Shaw, Kristi 

Jensen, Wendy Breen, Shawn Zinnen, Brent Dickinson, James 

A. McSwiggen, Chandra Vargeese, Keith Bowman Chris S. 

Shaffer, Lloyd Jeffs, Adam Judge, Barry Polisky, Jennifer A. 

Lockridge, Amy C. H. Lee, Ian MacLachlan 

439 2:45 PM 

CXCR4 and CCR5 shRNA Transgenic Macrophages Are 

Functionally Normal and Resist HIV-1 Infection 

Joseph Anderson, Ramesh Akkina 

440 3:00 PM 

A Non-Human Primate Model for Lentivirus-Mediated 

Anti-HIV RNAi Strategies 

Karen Beagles, Grant Trobridge, Brian Beard, John Rossi, Jiing- 

Kuan Yee, Shiu-Lok Hu, Hans-Peter Kiem 

441 3:15 PM 

Short Hairpin RNA Interference Expressed from a Gene- 

Deleted Adenoviral Vector Results in Reduction of 

Hepatits B Surface Antigen Levels In Vitro and in a Small 

Animal Model for Hepatitis B Infection 

Anja Ehrhardt, Hui Xu, Felix H. Salazar, Patricia L. Marion, 

Mark A. Kay 

442 3:30 PM 

Evaluation of siRNA for Induction of In Vitro Resistance 

in HIV-1 

Priscilla Yam, Jerry Wu, Ying Yu, John A. Zaia, Jiing-Kuan Yee 

443 3:45 PM 

Tissue Specific Expression of Short Hairpin RNAs for the 

Treatment of HCV Using Liver Specific RNA Pol II 

Expression Cassettes 

David A. Suhy, Alexander A. Kolykhalov, Linda B. Couto, 

Luz Maria Garcia, A. Rosanna Schroeder, Amy E. Parker, 

Gabriel Haniff, Sara M. Cunningham, Mark A. Kay, Peter W. 

Roelvink 


Targeting of Adeno Viral Vectors and Cancer Treatment 

Room: 123/127 

444 2:00 PM 

A New Vector Which Targets Breast Cancer and Its 

Vasculature 

Yanzheng Liu, Tao Ye, Jonathan Maynard, Hakan Akbulut, 

Albert Deisseroth 

445 2:15 PM 

Combinatorial Antiangiogenic Gene Therapy by 

Nonviral Gene Transfer Using the Sleeping Beauty 

Transposon Causes Tumor Regression and Improves 

Survival in Mice Bearing Intracranial Human Glioblastoma 

John R. Ohlfest, Zachary L. Demorest, Yasuhiko Motooka, 

Isabelita Vengco, Seunguk Oh, Eleanor Chen, Frank A. 

Scappaticci, Stephen C. Ekker, Walter C. Low, Andrew B. 

Freese David A. Largaespada 

446 2:30 PM 

Tie2 Expression Defines an Integrated System of Cell 

Types Specifically Involved in Angiogenesis, and Provides 

a Platform for Targeted Gene Delivery to Tumors 

Michele De Palma, Mary Anna Venneri, Rossella Galli, 

Nathalie Belmonte, Maurilio Sampaolesi, Lucia Sergi Sergi, 

Luigi Naldini


ABSTRACTS 


447 2:45 PM 

Mesenchymal Stem Cells as Delivery Systems for Cancer 

Therapy: Evaluation of Tropism and Efficacy 

Frank Marini, Jennifer Dembinski, Matus Studeny, Claudia 

Zompetta, Michael Andreeff 

448 3:00 PM 

A Genetic Strategy to Inhibit Complement Activation 

Arising from Adenoviral Vectors 

Kurt R. Zinn, Hongju Wu, Amanda Stargel, April J. Adams, 

Tandra R. Chaudhuri 

449 3:15 PM 

Tumor Targeted, Systemic Delivery of Therapeutic Viral 

Vectors Using Hitch Hiking on Antigen Specific T Cells 

Jian Qiao, Caroline Cole, Timothy Kottke, Rosa Maria Diaz, 

Luis Sanchez-Perez, Gregory Brunn, Jill Thompson, Richard 

Vile 

450 3:30 PM 

Combination of Fas Gene Therapy and Ceramide 

Analogues: A Double Edge Sword for the Treatment of 

Head and Neck Cancer 

Saeed N. Elojeimy, John C. McKillop, Ahmed M. El-Zawahry, 

David A. Schwartz, David H. Holman, Xiang Liu, Terry Day, 

Alicja Bielawska, Yusuf A. Hannun, James S. Norris 

451 3:45 PM 

Matrix Metalloproteinase-Targeted Oncolytic Sendai 

Virus Vector “Armed” with a Sucide Gene “Yeast Cytosine 

Deaminase”; Remarkable Combinational Effects 

Hiroaki Kinoh, Makoto Inoue, Kentaro Washizawa, Eiji Akiba, 

Mamoru Hasegawa 

452 4:00 PM 

The Enhancement of Anticancer Effect of Chemotherapy 

by the Delivery of Rad51 siRNA 

Makoto Ito, Seiji Yamamoto, Keisuke Nimura, Katsuto Tamai, 

Yasufumi Kaneda 

453 4:15 PM 

The Histone Deacetylase Inhibitor FK228 Can Increase 

Adenovirus Transgene Protein Expression in Human 

LOX IMVI Melanoma Xenografts 

Merrill E. Goldsmith, Alian Aguila, Michael C. Alley, William 

R. Waud, Susan Bates, Tito Fojo 


Advances in Hematologic Gene Therapy 

Room: 263/267 

454 2:00 PM 

Mouse Models for the Analysis of Genetic Risk Factors 

for Hematopoietic Transformation in XSCID Gene 


Yan Shou, Lilia Stepanova, Brian P. Sorrentino 

455 2:15 PM 

Drug Resistance Gene Therapy to Induce Donor-Specific 

Tolerance after Nonmyeloablative Allogeneic Stem Cell 

Transplantation in Dogs 

Sabine Gerull, Karen E. Beagles, Brian C. Beard, Laura J. 

Peterson, Hans-Peter Kiem 

456 2:30 PM 

HOXB4 Overexpression Expands Short-Term Repopulating 

Cells in Nonhuman Primates and Dogs 

Xiao-Bing Zhang, Laura J. Peterson, Alixandra A. Knapp, 

Brian C. Beard, R. Keith Humphries, Hans-Peter Kiem 

457 2:45 PM 

The Benefits of CH-296 during Ex Vivo Gene Transfer 

Go beyond the Ability to Co-Localize the Retroviral 

Particle and Target Cell 

Mo A. Dao, Jan A. Nolta 

458 3:00 PM 

Reversal of Canine Leukocyte Adhesion Deficiency by 

Retroviral-Vector Mediated Gene Therapy with Non- 

Myeloablative Conditioning 

Thomas R. Bauer, Jr., Laura M. Tuschong, Mehreen Hai, Yuchen 

Gu, Robert A. Sokolic, Tanya Burkholder, John D. Bacher, 

Dennis D. Hickstein 

459 3:15 PM 

Long-Term Treatment of Canine Cyclic Neutropenia by 

G-CSF-Lentivirus 

William Osborne, Margaret Brzezinski, Ofer Yanay, Lanaya 

Waldon, Jeffrey Christensen, Denny Liggitt, David Dale 

460 3:30 PM 

Transplantation of Human Aldehyde Dehydrogenase 

Expressing Cells Leads to Widespread Tissue Distribution 

of Donor Cells in the Pancreas and Liver of NOD/SCID/ 

MPSVII Mice 

David A. Hess, Timothy P. Craft, Louisa Wirthlin, Phillip E. 

Herrbrich, Alex A. Hofling, Mark S. Sands, Jan A. Nolta 

461 3:45 PM 

Qualitative and Quantitative Luciferase Expression In 

Vivo of Transduced Hematopoietic Cell populations 

Using Bioluminescent Imaging (BLI) 

Henk Rozemuller, Ingrid F. H. Koggel, Robbert Spaapen, Anton 

Hagenbeek, Anton C. M. Martens 

462 4:00 PM 

Busulfan Dose Escalation to Increase Gene Marking of 

Hematopoietic Stem Cells by Lentiviral Vectors in Infant 

Rhesus Monkeys 

Christoph A. Kahl, Alice F. Tarantal, Chang I. Lee, Daniel F. 

Jimenez, Christopher Choi, Karen Pepper, Denise Peterson, 

Misty D. Fletcher, Alyssa C. Leapley, Donald B. Kohn

74 


ABSTRACTS 


463 4:15 PM 

Efficient Lentiviral Gene Transfer and Expression in 

Human Embryonic Stem Cells 

Carolyn Lutzko, XiaoJin Yu, Dinithi Senadheera, Donald B. 

Kohn 


Gene Regulation: Regulated Systems and Cell 

Engineering 

Room: 261/262 

464 2:00 PM 

Development of a Novel Gene Regulation System 

Downstream of Promoter 

Hongzhe Duan, Chengwen Li, Richard Jude Samulski 

465 2:15 PM 

In Vivo Selection of Genetically Modified Primary 

Human Hematopoietic Cells Using a Cell Growth 

Switch 

Linlin Wang, Yasuo Nagasawa, Brent Wood, Ingrid Lintmaer, 

Thalia Papayannopoulou, Michael Harkey, Cynthia Nougirat, 

C. Anthony Blau 

466 2:30 PM 

Long-Term Rapamycin Control of Transgene Expression 

in Mouse Salivary Glands in a Single AAV Vector 

Jianghua Wang, Antonis Voutetakis, Milton Papa, Victor M. 

Rivera, Tim Clackson, Beatrijs M. Lodde, Fumi Mineshiba, 

Bruce J. Baum 

467 2:45 PM 

Allogeneic Bone-Marrow Transplantation in Mice 

Provides Tolerance and Promotes Massive In Vivo 

Selection of Subsequently Transplanted Hepatocytes 

Konrad L. Streetz, Regis Doyonnas, Denny D. Jenkins, Seppi 

Lin, Judith A. Shizuru, Helen Blau, Karl Sylvester, Mark A. 

Kay 

468 3:00 PM 

Cellular Therapy with Transgene Expressing APC 

Activates CD4+CD25+ Regulatory T Cells Which 

Modulate the Immune Response to Gene Therapy 

Derived Products in Immunocompetent Mice 

Andrea Annoni, Manuela Battaglia, Antonia Follenzi, Angelo 

Lombardo, Luigi Naldini, Maria Grazia Roncarolo 

469 3:15 PM 

Induction of Donor Specific Tolerance Using Gene 

Therapy and Sublethal Conditioning 

Daron Forman, Chaorui Tian, John Iacomini 

470 3:30 PM 

Foamy Viral Mediated Transduction of Recombinant 

FANCC in the Absence of Prestimulation Is Sufficient To 

Restore the Long Term Repopulating Activity of Fancc -/- 

Stem Cells and Prevent the Acquisition of Myeloid 

Malignancies 

Yue Si, Cordula Leurs, Edward F. Srour, Jin Yuan, Helmut 

Hanenberg, D. Wade Clapp 

471 3:45 PM 

Microvessel Injury and Irradiation Damage in Salivary 

Glands 

Ana Paola P. Cotrim, Jianghua Wang, Antonis Voutetakis, 

Anastasia L. Sowers, James B. Mitchell, Bruce J. Baum 

472 4:00 PM 

Hematopoietic Cell Differentiation from Common 

Marmoset (Callithrix jacchus) Embryonic Stem Cells by 

Their Genetic Manipulation Using the Third Generation 

Lentiviral Vector 

Tomoko Yokoo, Ryo Kurita, Erika Sasaki, Takashi Hiroyama, 

Yukoh Nakazaki, Kiyoko Izawa, Hajime Ishii, Yoshikuni 

Tanioka, Kisaburo Hanazawa, Yuan Son Bai Yasushi Soda, 

Kenzaburo Tani 

473 4:15 PM 

Definitive Identification and Isolation of Embryonic 

Stem Cell-Derived Target Cells by Adenoviral Conditional 

Targeting; Efficient Purification of Immature and 

Mature Cells in the Cardiac Lineage 

Ken-ichiro Kosai, Tomoyuki Takahashi 

Poster Session II 


RNA Virus Vectors: Gene Transfer, Vector Production 

474 

Transduction of Minimally Stimulated Hematopoietic 

Progenitor Cells with a Foamy Virus Vector That Expresses 

MGMTP140K Shanbao Cai, Jennifer R. Hartwell, Aaron G. Ernstberger, W. S. 

Goebel, Helmut Hanenberg, Karen E. Pollok 

475 

In Vivo Targeting of MLV(HIV) Pseudotype Vectors to T 

Lymphocytes in a Transgenic Mouse Model 

Silke Schuele, Stefanie Steidl, Sylvia Raupp, Cheick Coulibaly, 

Ulrich Kalinke, Klaus Cichutek, Matthias Schweizer 

476 

HIV-1 and HIV-2 Chimeric Lentiviral Vectors for Gene 

Transfer 

Geetanjali Sachdeva, Kritika Kachapati, Suresh Arya


ABSTRACTS 


477 

Development of SIV Based Vectors with Efficient 

Delivery for Vector Dynamic Studies in the Simian 

System 

Christopher Chen, Jiamo Lu, Tatiana Slepushkina, Andrew 

Worden, Vladimir Slepushkin, Laurent M. Humeau, 

Gwendolyn K. Binder, Xiaobin Lu 

478 

A HIV-1 Based Cross-Packaging System for FIV Vectors 

Adam S. Cockrell, Hong Ma, Thomas McCown, Kim D. 

Kluckman, Randy J. Thresher, Tal Kafri 

479 

Targeted Entry of Retroviral Vectors Depends on Characteristics 

of the Target Receptor and the Chimeric Envelope 

Protein 

Lorraine M. Albritton, Fang Li, Pankaj Kumar, Langzhu Tan 

480 

Examination of the Relationship between the Level of 

Gene Transfer and the Number of Envelope Proteins 

That Are Incorporated Per Retrovirus Particle When 

High Doses of Purified Virus Stocks Are Used to 

Transduce Cells 

Natalia Landázuri, Joseph M. Le Doux 

481 

Dynamics of Transduction of Human Cells by EIAV 

Vectors – The Importance of Cell Entry 

Daniel C. Farley, Fraser J. Wilkes, James E. Miskin, Susan M. 

Kingsman, Kyriacos A. Mitrophanous 

482 

Neuronal Gene Transfer with HIV-1-Based Lentiviral 

Vectors Pseudotyped with Lyssavirus Glycoproteins 

Qingshan Teng, Mary E Garrity-Moses, Thais Federici, Jun 

Yang, Robert Kutner, Noel Tordo, Jakob Reiser, Nicholas M. 

Boulis 

483 

Targeted Receptor Trafficking Affects the Efficiency of 

Retrovirus Transduction 

Delfi Krishna, Julia Raykin, Joseph Le Doux 

484 

Ultrasound-Mediated Microbubble Destruction and 

Retroviral Gene Delivery 

Sarah L. Taylor, Ahad Rahim, Nigel Bush, Jeff Bamber, Colin 

Porter 

485 

Lentivirus-Mediated Gene Transfer to Cultured Fetal 

Sheep Airway Epithelium and Lung Tissue Explants 

Ze-Yan Yu, Karen McKay, Peter Van Asperen, Maolin Zheng, 

Jane Fleming, Samantha Ginn, Eugenie Lumbers, Peter Rowe, 

Ian Alexander 

486 

Optimized Conditions for Retroviral Gene Transfer by 

RetroNectin ® Bound Virus Infection Method 

Hideto Chono, Hiromi Okuyama, Yasushi Katayama, Nobuto 

Koyama, Junichi Mineno, Kiyozo Asada, Ikunoshin Kato 

487 

Enhancement of Adeno- and Lentivirus Mediated Gene 

Transfer in Human Tumor Cell Lines by Cell Permeable 

Peptides and Polycations 

Saara Lehmusvaara, Outi Meriläinen, Tanja Hakkarainen, 

Jarmo Wahlfors 

488 

Engineering Enhanced Retroviral Vectors through the 

Insertion of a His Tag into VSV-G 

6 

Julie H. Yu, David V. Schaffer 

489 

Further Studies of the Most Efficient Vector Systems for 

Gene Transduction into Human Dendritic Cells Line 

(im-NMD) 

Eigo Sato, Masanari Kato, Yuko Hara, Hua Yan, Tohko 

Miyagi, Xiao-Kang Li, Wataru Sugiura, Naoki Yamamoto, 

Kenichi Teramoto, Shigeki Arii Hiromitsu Kimura 

490 

Lentiviral-Mediated Gene Transfer to Cells of the 

Vasculature 

Ciara A. O’Shea, Robert J. Nelson, Padraig Strappe, Timothy 

O’Brien 

491 

Development of Fast and Efficient Methods for the 

Purification and Concentration of Lentiviral Vectors 

Rachel M. Koldej, Donald S. Anson 

492 

Harvest Timing and Media Composition Effects on 

Lentiviral Vector Production 

Christopher Ballas, Terry Johnson, Kenneth Cornetta 

493 

Concentration and Purification of Lentivirus Pseudotypes 

Bearing Alternative Envelope Glycoproteins 

Robert H. Kutner, Jakob Reiser 

494 

Assessing Lentiviral Vector Quality Using Transmission 

Electron Microscopy 

Lakshmi Sastry, Carole Miller, Terry Johnson, Aparna Jasti, 

Vince Gattone, Kenneth Cornetta 

495 

Upscaling of Retroviral Vector Production under GMP 

Guidelines to Generate High Titer Preparations for 

Clinical Use 

Sandra Jung, Peter Leyendecker, Frank Kogelberg, Christian 

Wolf, Andrea Schilz, Klaus Kuehlcke, Sonja Naundorf

76 


ABSTRACTS 


496 

Stability of Oncolytic Measles Viruses during Storage and 

Clinical Preparation 

Troy R. Wegman, Kirsten K. Langfield, Henry J. Walker, Guy 

E. Griesmann, Julie A. Sauer, Sharon A. Stephan, Mark J. 

Federspiel 


497 

Sex and Estrous Cycle Stage Influence the Efficiency of 

AAV-Mediated Gene Transfer in the Rodent Brain 

James C. Dodge, Jennifer Clarke, Marco A. Passini, Anthony 

Song, Catherine R. O’Riordan, Seng H. Cheng, Gregory R. 

Stewart 

498 

Adeno-Associated Virus Type-1 Vectors Efficiently 

Transduce Human Polarized Airway Epithelia without 

Polarity Bias 

Ziying Yan, Xiaoming Liu, Chi-Man Lei, Meihui Luo, John 

F. Engelhardt 

499 

Species-Specific Differences in the Polarity of rAAV5 and 

rAAV2 Transduction of Mouse and Human Airway 

Epithelia 

Xiaoming Liu, Ziying Yan, Meihui Luo, John F. Engelhardt 

500 

Biology of Novel AAV Serotype Vectors in the Liver 

Directed Gene Transfer to Nonhuman Primates 

Guangping Gao, You Lu, Roberto Calcedo, Rebecca Grant, 

Julio Sanmiguel, Lili Wang, Julie Johnston, Joanita Figueredo, 

Martin Lock, James M. Wilson 

501 

AAV6 Pseudotyped Double-Stranded-AAV Vectors Allow 

Adenoviral-Like Transduction and Brute-Force Integration 

in the Growing Mouse 

Ognjen Petras, Timothy J. Davern, Marcus O. Muench, 

Xiaodong Han 

502 

GFP-Tagged Adeno-Associated Viral (AAV) Particles 

Allow the Study of Cytosolic and Nuclear Trafficking 

Kerstin Lux, Stefanie Schlemminger, Luca Perabo, Daniela 

Goldnau, Jan Endell, Michael Hallek, Hildegard Buening 

503 

Mutational Strategy To Enhance or Rescue Functionality 

of Existing AAV Vectors 

Luk H. Vandenberghe, Michael H. Yang, Julie Johnston, 

Guangping Gao, James M. Wilson 

504 

In Vivo Characterization of AAV Serotype 9 Vectors in 

Mice 

Katsuya Inagaki, Theresa A. Storm, Sally Fuess, Mark A. Kay, 

Hiroyuki Nakai 

505 

Analysis of AAV Genome Conversion and Stabilization 

Bernd Hauck, Jing Xie, Weidong Xiao 

506 

Transductional and Genome Biodistribution of Novel 

AAV Serotype Vectors after Systemic Delivery and Their 

Liver Specific Dose Response Profiles in Mice 

You Lu, Guangping Gao, Julio Sanmiguel, Roberto Calcedo, 

Peter Bell, Nelson Wivel, James M. Wilson 

507 

Modeling Adeno-Associated Virus 2 Capsid for the 

Identification of Targeting Ligands for Enhanced Gene 

Transfer to Polarized Human Airway Epithelial Cells 

April F. White, Marina Mazur, Eric J. Sorscher, Selvarangan 

Ponnazhagan 

508 

Impact of Sex on Serum að1-Antitrypsin Levels Following 

Intrapleural and Intravenous Administration of 

AAVrh.10 Vectors to Mice 

Bishnu P. De, Adriana Heguy, Neil R. Hackett, Guangping 

Gao, James M. Wilson, Ronald G. Crystal 

509 

Endogenous Estradiol Augments RGD Modified AAV2- 

Mediated Gene Transfer in Human Ovarian Epithelial 

Cancer 

Wenfang Shi, Jeffery Bartlett 

510 

Neonatal Gene Transfer with Non-Human Primate AAV 

Vector Serotypes Results in Widespread Transduction and 

Gene Activity in the Mouse Brain 

Brian A. Karolewski, Hennessy Howell, John H. Wolfe 

511 

Effect of Hepatocyte Division on Molecular Fate of rAAV 

DNA 

Young-Kook Choi, Yuanqing Lu, Sihong Song 

512 

The Cellular TATA-Binding Protein Is Required for 

Replication of a Minimal Adeno-Associated Virus Type 2 

(AAV-2) p5 Promoter Element 

Achille François, Mickael Guilbaud, Rafi Awedikian, Gilliane 

Chadeuf, Philippe Moullier, Anna Salvetti 

513 

Novel Characteristics of Heparin Binding by Adeno- 

Associated Virus Serotype 6 

James M. Allen, Eric E. Finn, Jeffrey S. Chamberlain


ABSTRACTS 


514 

Effect of DNA-PKcs on AAV Replication 

Young-Kook Choi, Irene Zolotukhin, Barry J. Byrne, Sihong 

Song 

515 

Overlapping Adeno-Associated Viral (AAV) Vector 

Mediated Gene Transfer Is Dependent on Viral Serotype 

and the Transgene Sequence in Skeletal Muscle 

Arkasubhra Ghosh, Yongping Yue, Dongsheng Duan 

516 

Analysis of AAV Serotype 8 Vector Integration in Normal 

and DNA-PKcs-Deficient Scid Mice by a Novel Strategy 

Katsuya Inagaki, Xiaolin Wu, Sally Fuess, Theresa A. Storm, 

Mark A. Kay, Hiroyuki Nakai 

517 

Type 1 Rep52 Is Superior to Authentic Rep52 for Producing 

Recombinant Adeno-Associated Virus Type 5 in 

Insect Cells 

Masashi Urabe, Takayo Nakakura, Hiroaki Mizukami, 

Akihiro Kume, Robert M. Kotin, Keiya Ozawa 

518 

Production of Recombinant Type 2 Adeno-Associated 

Virus in Bioreactors 

Yves Durocher, Phuong Lan Pham, Gilles St-Laurent, Danielle 

Jacob, Brian Cass, Josephine Nalbantoglu, Amine Kamen 

519 

Selective Inactivation of Helper Adenovirus with High 

Hydrostatic Pressure for AAV-2 Vector Production 

Joshua N. Leonard, David V. Schaffer, Peter Ferstl, Antonio 

Delgado 

520 

The Effects of Transgene and Cassette Size on Recombinant 

AAV2 Production and Expression 

Corinne M. Goldsmith, Antonis Voutetakis, Bruce J. Baum 

521 

Spliceosome-Mediated RNA Trans-Splicing with rAAV 

Partially Restores CFTR Function to Polarized Human 

CF Airway Epithelial Cells 

Xiaoming Liu, Meihui Luo, Liang N. Zhang, Ziying Yan, Wei 

Ding, Roman Zak, Gary S. Mansfield, Lloyd G. Mitchell, John 

F. Engelhardt 

522 

Enhanced Repair of Articular Cartilage Defects in Rabbits 

by Overexpression of Human FGF-2 Via rAAV-Mediated 


Magali Cucchiarini, Henning Madry, Chunyan Ma, Tanja 

Thurn, David Zurakowski, Dieter Kohn, Michael D. Menger, 

Stephen B. Trippel, Ernest F. Terwilliger 

523 

AAV Transcytosis through Barrier Epithelia and Endothelium 

Giovanni Di Pasquale, John A. Chiorini 

Adenovirus Vectors: Applications 

524 

Adenovirus-Mediated Gene Delivery of Decoy Death 

Receptors Protects Mice from Hepatitis 

Delphyne Descamps, Frédéric Vigant, Stéphanie Esselin, 

Elisabeth Connault, Paule Opolon, Michel Perricaudet, Karim 

Benihoud 

525 

Optimized Gene Transfer into Mesenchymal Stem Cells 

and Embryonic Stem Cells by Modified Adenovirus 

Vectors 

Hiroyuki Mizuguchi, Kenji Kawabata, Fuminori Sakurai, 

Tomomi Sasaki, Teruhide Yamaguchi, Takao Hayakawa 

526 

Conditionally Replicative Adenoviruses Designed to 

Restore Defects in the p53 Pathway for Effective 

Virotherapy of Cancer 

Victor W. van Beusechem, Danielle A. M. Heideman, Jan E. 

Carette, Martin Scheffner, Ramon Alemany, Winald R. 

Gerritsen 

527 

Reductions in Tissue Transduction and Toxicity after 

Systemic Administration of Adenovirus Vectors Containing 

Fiber-Shaft Exchange in Combination with both 

CAR- and av Integrin-Binding Ablation 

Naoya Koizumi, Hiroyuki Mizuguchi, Kenji Kawabata, 

Fuminori Sakurai, Yoshiteru Watanabe, Takao Hayakawa 

528 

Infectivity Enhancement of a Genetic Fiber Mosaic 

Adenovirus Vector in Ovarian Cancer Cells 

Yuko Tsuruta, Larisa Pereboeva, Joel N. Glasgow, Yosuke 

Kawakami, David T. Curiel 

529 

Oncolytic Virotherapy Employing flt-1 Driven mda-7/IL- 

24 Gene Delivery 

Sergey A. Kaliberov, Lyudmila N. Kaliberova, Amy S. Petersen, 

Valentina Krendelchtchikova, Victor Krasnykh, Donald J. 

Buchsbaum 

530 

Adenoviral and Hydrodynamic Gene Transfer of Replicating 

Episomes Result in Stable Transgene Expression 

after Partial Hepatectomy in Mice 

Jan Snoeys, Désiré Collen, Bart De Geest

78 


ABSTRACTS 


531 

Balloon Catheter-Mediated Hepatic Vein Delivery of a 

Viral Vector Mitigates Neutralization by Anti-Viral 

Antibodies and Results in Efficient Transduction of 

Rabbit Liver 

Bradley L. Hodges, Kristin M. Taylor, Qiuming Chu, 

Samantha E. Scull, Rebecca G. Serriello, Scott C. Anderson, 

Ronald K. Scheule 

532 

Effects of a New YB-1 Dependent Adenovirus (XVir) in 

Human Prostate Cancer Cell Lines and in a Xenograft 

Mouse Model 

Klaus Mantwill, Nadia Koehler-Vargas, Alexandra 

Bernshausen, Uwe Treiber, Rudolf Hartung, Bernd Gansbacher, 

Per Sonne Holm 

533 

Enhanced Transduction of Dendritic Cells by FCgRI- 

Targeted Adenovirus Vectorsgð 

Ramil E. Sapinoro, Angela P. H. Burgess, Stephen Dewhurst 

534 

Optimising Expression Cassettes for Vascular Gene 

Expression 

Stephen J. White, Melanie A. Roberts, Jeremy R. Sanford, Javier 

F. Cáceres, Andrew C. Newby 

535 

Long-Term Correction of Hyperbilirubinemia in a Rat 

Model of Crigler-Najjar Syndrome Type 1 

Viraj P. Mane, Gabriele Toietta, Wilma Norona, Milton 

Finegold, Phil Ng, Arthur L. Beaudet, Anthony F. McDonagh, 

Brendan Lee 

536 

Adenoviral Vector-Mediated Gene Therapy for 

Adenocortical Carcinoma: Therapeutic Strategies and 

Safety Considerations 

Luisa Barzon, Urska Trost, Monia Pacenti, Anna-Lisa Stefani, 

Marta Trevisan, Giulia Masi, Karina Fincati, Giorgio Palù 

537 

Transcriptionally Targeting the Survivin Promoter to 

Human Gliomas 

Zeng B. Zhu, Winan J. Houdt, Yosef S. Haviv, Baogen Lu, 

Minghui Wang, Angel A. Rivera, Ilya V. Ulasov, Maciej S. 

Lesniak, Gene P. Siegal, Clemens M. F. Dirven David T. 

Curiel 

538 

Fiber Chimeric Oncolytic Adenoviral Vectors Mediate 

Efficient Gene Transfer and Oncolysis of Head and Neck 

Cancer and Melanoma Cells In Vitro and In Vivo 

Shanthi Ganesh, Yuanhao Li, Peter Working, P. Seshidhar 

Reddy, De-Chao Yu 

539 

Antitumor Activity of an hTERT-Specific, GFP Expressing 

Oncolytic Adenovirus 

John J. Davis, Li Wang, Lidong Zhang, Wei Guo, Fuminori 

Teraishi, Kai Xu, Lin Ji, Bingliang Fang 

540 

Re-Targeting of Adenoviral Vector-Mediated Interferongð 

Gene Transfer to Colon Cancer Cells That Express the 

Human Carcinoembryonic Antigen 

Maaike Everts, Jing Li, Krishnakant Pondugula, Angel A. 

Rivera, David T. Curiel, J. Michael Mathis 

541 

Oncolytic Effects of Conditionally Replicating 

Adenoviruses (CRAds) with Mutations in E1A and E1B 

Regions 

Hyunhee Kim, Jaesung Kim, Kyung-Ju Choi, Pyung Hwan 

Kim, Tae Young Koo, Ahmed M. Al-Abd, Hye Jin Choi, Joo- 

Hang Kim, Chae-Ok Yun 

542 

Targeting EGFR with Metabolically Biotinylated Fiber- 

Mosaic Adenovirus 

Larisa Pereboeva, Svetlana Komarova, Selvarangan 

Ponnazhagan, David T. Curiel 

543 

Enhanced Glia-Specific Expression Using GFAP- 

’Super’Promoters in an Adenoviral Context In Vitro and 

In Vivo 

Bertie de Leeuw, Maarten ter Horst, Mark Rodijk, Rob C. 

Hoeben, Michael Brenner, Peter A. E. Sillevis Smitt 

544 

Interferon alpha-2b Gene Delivery for Bladder Cancer 

Demonstrates Feasibility of Repeated Intravesical Dosing 

with an Adenovirus Vector 

Heidrun Engler, Robert J. Connor, Jennifer M. Anderson, Drake 

LaFace, Daniel C. Maneval 

545 

Adenovirus Mediated Gene Transfer and Oncolysis In 

Vitro in Head and Neck Cancer and Melanoma Cells 

Enhanced by Polycations 

Lian Mo, Gary Lee, Shanthi Ganesh, Yuanhao Li, Peter 

Working, P. Seshidhar Reddy, De-Chao Yu 

546 

Gene Therapy of Ovarian Cancer by IAI.3B Promoter 

Driven Oncolytic Adenovirus 

Katsuyuki Hamada, Toshiro Shirakawa, Akinobu Gotoh 

547 

Improvement of Liver Gene Transfer by First and Third 

Generation Adenoviral Vectors 

Julien S. Crettaz, Pedro Berraondo, Itsaso Mauleon, Laura 

Ochoa, Veijay Shankar, Miguel Barajas, Maider Zabala, Nico 

van Rooijen, Stefan Kochanek, Cheng Qian Jesus Prieto, Ruben 

Hernandez-Alcoceba, Gloria Gonzalez-Aseguinolaza


ABSTRACTS 


548 

Ethanol Increases Transgene Expression in Parenchymal 

Liver Cells after Adenoviral Transfer in Rabbits 

Jan Snoeys, Joke Lievens, Yingmei Feng, Peter Frederik, Désiré 

Collen, Eddie Wisse, Bart De Geest 

549 

Peripheral Gene Delivery of Glial Cell Line-Derived 

Neurotrophic Factor Alleviated Neurological Deficits in 

Diabetic Rats 

Guei-Sheung Liu, Chiou-Lian Lai, Sheng-Long Howng, 

Ming-Hong Tai 

550 

Adenoviral-Mediated Antioxidant Overexpression: 

Possible Protective Role Against Hyperglycemic Conditions 

in Human Aortic Endothelial Cells 

Aoife Duffy, Aaron Liew, Afshin Samali, Timothy O’Brien 

551 

Enhanced Cell Killing Effect of E1 Modified 

Adenoviruses Expressing HSV-TK Suicide Gene 

Yoon-A Kang, Dong Hyun Ko, Jaesung Kim Kim, Young-Sook 

Lee, Oh-Joon Kwon, Joo Hyuk Sohn, Chae-Ok Yun, Joo- 

Hang Kim 

552 

Gene Therapy of Uterine Fibroids: Adenovirus-Mediated 

Expression of Dominant Negative Estrogen Receptor 

Inhibits ERE-Reporter Transactivation in Rat and Human 

Leiomyoma Cells 

Ayman Al-Hendy 

553 

A Potent Replicative Delta Adenoviral Vector for HPV 

Tumors 

Ivan Delgado-Enciso, Irma A. Martinez-Davila, Rocio Ortiz- 

Lopez, Ramon Alemany-Bonastre, Christian I. Silva-Platas, 

Angel Lugo-Trampe, Hugo A. Barrera-Saldana, Augusto Rojas- 

Martinez 

554 

Evaluation of Viral Vector Gene Delivery Systems in 

Endothelial Cells In Vitro and Ex Vivo 

Yanjuan Lin, Anming Xiong, Maria T. Millan 

555 

An Oncolytic Adenovirus Controlled by a Modified 

Telomerase Promoter Is Attenuated in Telomerase- 

Negative Cells, but Shows Reduced Activity in Cancer 


Sergia Bortolanza, Qian Cheng, Gabriela M. Kramer, Celia 

Gomar, Fabio Farinati, Jesus Prieto, Ruben Hernandez-Alcoceba 

Naked DNA Gene Transfer: Optimizing Expression, 

Animal Models 

556 

Another Hepatic Control Region for Sustained Expression 

of Human Factor IX in the Mouse Liver 

Ji Eun Lee, Hong Sung Kim, Keun Sik Kim, Eun Jung Kim, 

Jong Chul Kim, In Ho Song, Yong Serk Park 

557 

Unraveling the Molecular Mechanisms Responsible for 

Episomal Transgene Silencing In Vivo 

Zhi-Ying Chen, Cheng-Yi He, Hui Xu, Mark A. Kay 

558 

cGMP Production of Plasmid DNA for Phase I Clinical 

Studies in an Academic Center 

Mark Przybylowski, Shirley M. Bartido, Michel Sadelain, 

Isabelle Riviere 

559 

Potentiation of DNA Vaccination Using an Integrated 

Application System for Electroporation-Mediated 

Intramuscular Delivery 

Alain Luxembourg, Drew Hannaman, Steve Masterson, Barry 

Ellefsen, Gerald Nakamura, Robert Bernard 

560 

Optimization of E. coli Fermentation for Plasmid DNA 

Production 

Aaron Carnes, Williams James, Hodgson Clague 

561 

Analytical Characterization of Supercoiled Plasmids 

Anthony P. Green, Irina Tumanova, Joseph Burzynski, Dana 

Rosencrance, Julie Brunbeck, Jason Scheidel, S. Fernando Ausar, 

C. Russell Middaugh 

562 

Study on the Inhibitory Effect of Antisense ET R A 

Oligodeoxynucleotides for the Proliferation of Vascular 

Smooth Cells 

Lan Zhang, Baigen Zhang, Jiwei Zhang 

563 

Differential Histone Modifications in Transcriptional 

Active Versus Silenced Episomal Vectors In Vivo 

Efren Riu, Zhi-Ying Chen, Mark A. Kay 

564 

Delivery of Episomal Vectors Carrying Large Genomic 

Regions for Gene Therapy 

Wing T. Cheung, Catherine Grillot-Courvalin, Clare Huxley 

565 

A Ready-to-Use Process for Large-Scale Plasmid DNA 

Manufacturing 

Astrid Breul, Wayne Tvrdik, Markus Müller, Joachim Schorr

80 


ABSTRACTS 


566 

Silencing of Plasmid DNA after Hydrodynamics-Based 

Transfection in Mice 

Hiroshi Ochiai, Hideyoshi Harashima, Hiroyuki Kamiya 

567 

Prevention of Bilirubin Toxicity in the Gunn Rat by 

Long-Term Correction of Hyperbilirubinemia with 

Liver-Specific Plasmid-Based Gene Transfer 

Istvan Danko, Zhen Jia 

568 

In Vitro SFHR-Mediated Modification of SMA Locus in 

Different Human Trophoblast Cells 

Federica Sangiuolo, Antonio Filareto, Paola Spitalieri, Lucia 

Scaldaferri, Emanuela Bruscia, Ruggiero Mango, Citro 

Gennaro, Massimo De Felici, Giuseppe Novelli 

569 

Therapeutic Ultrasound for Anti-Angiogenic Gene 

Delivery: Mechanism Studies and Application for 

Prostate Tumor 

Maayan Duvshani-Eshet, Marcelle Machluf 

570 

The Effects of Cyclic Stretch on the Cytoskeleton and Its 

Role in Gene Transfer to the Alveolar Epithelium 

R. Christopher Geiger, David A. Dean 

571 

Development of a Quantitative Potency Assay for Quality 

Control Testing for pVGI.1(VEGF2), a Naked DNA 

Vector 

Li-chun Huang, Qing Wang, Jiemin Zhou, Hsing-yi Jan, 

Yawen L. Chiang 

572 

Alveolar Epithelial Cell-Specific Plasmid Nuclear Import 

James V. DeGiulio, David A. Dean 

573 

Investigation into the Frequency of Post-Integrative Gene 

Silencing Using a Non-Selective Sleeping Beauty Transposition 

System 

Brian S. Garrison, Stephen R. Yant, Jacob G. Mikkelsen, Mark 

A. Kay 

574 

Early Stage Product Development to Scale-Up: Use of 

Mini-Fermentation System for Screening and Process 

Development of Plasmid DNA Products 

Sriram Ramakrishnan, Justin Miller, Jeffrey Darnell, Henry Hebel 

575 

In Vivo Evaluation of Supercoiled Plasmids 

Anthony P. Green, Irina Tumanova, Joseph Burzynski, Dana 

Rosencrance, Julie White, Jason Scheidel, David Weiner, Jean 

Boyer 


System 

576 

Hydrophobic Portion of Cationic Amphiphiles Are More 

Important Than We Think for Transfection Efficiency 

Li Wang, Yaeko Hiyama, Harsh Parikh, Robert C. MacDonald 

577 

Distribution, Time Course, In-Vivo Imaging, Expression 

and Inflammatory Response after Non-Viral Gene 

Delivery to Rat and Primate CNS 

James G. Hecker, Ellen S. Hauck, Michael H. Nantz, Alice F. 

Tarantal 

578 

Quantitative Evaluation of the Relationship between 

Efficiency of Nuclear Transfer of Plasmid DNA and 

Trans-Gene Expression in Individual Cell: Consideration 

of Mechanism Underlying in the Heterogeneity of the 

Trans-Gene Expression Via Artificial Vector 

Hidetaka Akita, Rie Ito, Hiroyuki Kamiya, Kentaro Kogure, 

Hideyoshi Harashima 

579 

In-Vivo Time Course of Reporter Gene Expression after 

Nucleic Acid Delivery to the Lateral Ventricle in Rat 

Brain 

Ellen S. Hauck, Shaomin Zou, Michael H. Nantz, James G. 

Hecker 

580 

Vitreous - A Barrier to Non-Viral Ocular Gene Therapy 

Liesbeth Peeters, Niek N. Sanders, Kevin Braeckmans, Stefaan 

C. De Smedt, Joseph Demeester 

581 

Development of a Non-Viral DNA Vector Multifunctional 

Envelop-type Nano Device Modified with 

Octaarginine Peptide 

Kentaro Kogure, Ikramy A. Khalil, Rumiko Moriguchi, Shiroh 

Futaki, Hideyoshi Harashima 

582 

Synthetic siRNA Can Activate the Mammalian Innate 

Immune Response in a Sequence Dependent Manner 

Adam D. Judge, Janet R. Shaw, Vandana Sood, Kevin 

McClintock, Ian MacLachlan 

583 

The Transgene Expression in the Lung after Systemic 

Injection of DNA Formulated into Nanoparticles with 

Linear PEI Depends of Bolus Effect and the Presence of 

Free PEI 

Jean Luc Coll, Olivier Freund, Alain Cuzange, Jean Serge 

Remy, Jean Paul Behr, Patrick Erbacher 

584 

Gene Delivery to Primary Neuronal Cells 

Shaomin Zou, Ellen S. Hauck, Michael H. Nantz, James G. 

Hecker


ABSTRACTS 


585 

A Water-Soluble Lipoploymer Gene Carrier to Deliver a 

Bicistronic Plasmid Conveying IL-2 and Soluble Flk-1 

for the Treatment of Renal Cell Carcinoma Tumors 

James W. Yockman, Won Jong Kim, Sung Wan Kim 

586 

Novel Lipidic Vectors for Targeted Delivery of Functional 

Oligonucleotides to Pulmonary Endothelium 

Annette S. Wilson, Stephen J. Kennel, Leaf Huang, Bruce R. 

Pitt, Song Li 

587 

Artificial Viral-Like Gene Delivery System Multifunctional 

Envelope-type Nano Device Equipped with 

Transferrin and a pH-Sensitive Fusogenic Peptide 

Shinji Chaki, Kentaro Sasaki, Kentaro Kogure, Hirofumi 

Hamada, Masaharu Ueno, Shiroh Futaki, Hideyoshi 

Harashima 

588 

Structure-Activity Relationships in Two Series of 

Pyridinium Gemini Surfactants for Gene Delivery 

Marc A. Ilies, Betty H. Johnson, William A. Seitz, Ed L. Ezell, 

Aaron Miller, E. Brad Thompson, Alexandru T. Balaban 

589 

Construction and Antiproliferative Effect of mc-gðIFN 

Jiangxue Wu, Bijun Huang, Benling Xu, Gang Xue 

590 

Efficient, Serum-Resistant Transfection of Murine 

Squamous Cell Carcinoma Cells by Metafectene and 

GeneJammer: Application to HSV-tk/Ganciclovir Gene 


Krystyna Konopka, Nathan Overlid, Nejat Düzgünes 

591 

Transfection Efficiency of Monomolecular DNA 

Nanoparticles Formulated with New Perfluorinated 

Polycationic Dimerizable Detergents 

Karine Fabio, Christophe Di Giorgio, Pierre Vierling 

592 

A Multifunctional Envelope-Type Nano Device for the 

Novel Gene Delivery of siRNA Plasmids: Optimization 

of Condensing Polycations 

Rumiko Moriguchi, Kentaro Kogure, Hidetaka Akita, Siroh 

Futaki, Makoto Miyagishi, Kazunari Taira, Hideyoshi 

Harashima 

593 

Progress towards DNA-Based and siRNA Therapeutics 

Michael R. Jorgensen, Michael Keller, Eric Perouzel, Andrew D. 

Miller 

594 

Enhancement of Cellular and Nuclear Uptake of Macromolecules, 

Therapeutic Genes and Lipoplexes by Using 

NLS and PTD Containing Viral Peptides 

Roosmarijn Vandenbroucke, Niek N. Sanders, Stefaan De 

Smedt, Joseph Demeester 

595 

Synthesis of Novel Polyhydroxylalkyleneamine for 

Nonviral Gene Deliver 

Li Qun, Guisheng Zhang, Dexi Li 

596 

Incorporation of a Hepatocyte-Specific Compound in 

Polycationic Liposomes Enhances Gene Transfection in 

Hep G2 Cells 

Michelle R. McCombs, Gideon O. Berger, Michael H. Nantz, 

Jian Wu 

Inborn Errors of Metabolism: Hemophilia 

597 

Liver Tissue Engineering at Extra-Hepatic Site: A Novel 

Therapeutic Approach toward Hemophilia 

Kazuo Ohashi, Mark A. Kay, Takashi Yokoyama, Hiroyuki 

Kuge, Hiromichi Kanehiro, Michiyoshi Hisanaga, Saiho Ko, 

Hiroyuki Naka, Akira Yoshioka, Yoshiyuki Nakajima 

598 

In-Vivo Test of Coagulation Factor VIII with Improved 

Properties for Hemophilia A Gene Therapy by Helper- 

Dependent Adenoviral Vectors 

Vincenzo Cerullo, Michael McCormack, Racel Garcia, Christian 

Clarke, Steven W. Pipe, Brendan Lee 

599 

Development of EIAV-Based Vectors for the Treatment of 

Haemophilia A 

Emily J. Custard, Fraser J. Wilkes, Susan M. Kingsman, 

Kyriacos A. Mitrophanous, Pippa A. Radcliffe 

600 

A Novel Gene-Deleted Adenoviral Vector for Somatic 

Integration and Long-Term Phenotypic Correction of 

Murine and Canine Hemophilia B 

Anja Ehrhardt, Hui Xu, Stephen R. Yant, Jeffrey A. Engler, 

Mark A. Kay 

601 

Persistent Expression of Factor VIII In Vivo Following 

FIV Lentiviral Gene Transfer 

Yubin Kang, Litao Xie, Diane Thi Tran, Colleen S. Stein, 

Melissa Hickey, Beverly L. Davidson, Paul B. McCray 

602 

Induction of Tolerance to Human Factor VIII after 

Neonatal Gene Transfer of a Retroviral Vector Is Dose- 

Dependent in Mice and Is Not Effective in Cats 

Lingfei Xu, Manxue Mei, Mark E. Haskins, Karyn Cullen, 

Katherine P. Ponder

82 


ABSTRACTS 


603 

Correction of Hemophilia B Phenotype by Novel 

Method of Regional Intravenous Delivery of AAV Vector 

to Skeletal Muscle of Hemophilia B Dogs 

Valder Arruda, Hansell Stedman, Haiyan Jian, Glenn Pierce, 

Joerg Schuettrumpf, Christian Furlan Freguia, Stefano Baila, 

Kapil Gopal, Timothy Nichols, Katherine High 

604 

Long-Term, Erythroid-Specific Expression of Human 

Factor IX in Hemophilic Mice Engrafted with Lentiviral 

Vector-Transduced Hematopoietic Stem Cells 

Alex H. Chang, Matthias Stephan, Michel Sadelain 

605 

Efficient AAV 1 and AAV2 Hybrid Vector for Gene 

Therapy of Hemophilia 

Bernd Hauck, Ruian Xu, Jing Xie, Qiulan Ding, Wenman Wu, 

Matthew Sipler, Hongli Wang, Katherine High, Weidong Xiao 

606 

Molecular Characterization of Hereditary Factor VII 

Deficiency in Beagles 

Majed N. Aljamali, Mary Beth Callan, Eleanor S. Pollak, Urs 

Giger, Petra Werner, Monika Griot-Wenk, Katherine A. High 

607 

Long-Term Phenotypic Correction of Canine Hemophilia 

B Following Systemic Administration of Helper 

Dependent Adenoviral Vector 

Nicola Brunetti-Pierri, Timothy Nichols, Donna Palmer, 

Stephanie McCorquodale, Elizabeth Merricks, Arthur Beaudet, 

Philip Ng 

608 

Cytotoxic T Lymphocyte Response to AAV2- and AAV8- 

Mediated Human Factor IX Gene Therapy in Mice 

Jian Chen, Hui-Chen Hsu, Alex Schlachterman, Katherine A. 

High, John D. Mountz 

609 

Long Term Persistence of Canine Factor IX-Specific Th1 

Lymphocytes in Regional Lymph Nodes after AAV1- 

Mediated Intramuscular Gene Transfer in a Hemophilia 

B Dog 

Federico Mingozzi, Denise Sabatino, Timothy Nichols, 

Stephanie McCroquodale, Valder Arruda, Katherine High 

610 

Evaluation of Three Reticuloendothelial System Blocking 

Modalities on the Outcome of Adenoviral Mediated 

Gene Therapy in Hemophilic Mice 

Maha Othman, Andrea Labelle, Christine Hough, Alan H. 

Lazarus, David Lillicrap 

611 

Nonviral Gene Transfer of Factor VII as Alternative 

Treatment for Hemophilia A 

Steven C. Chen, Daniel Greenberg, Peiqing Ye, Earl W. Davie, 

Carol H. Miao 

612 

Determination of Specific Th1 and Th2 CD4 T Cell 

Responses in AAV2- and AAV8-Mediated Human Factor 

IX Gene Therapy in Mice 

Jian Chen, Hui-Chen Hsu, Alex Schlachterman, Katherine A. 

High, John D. Mountz 

613 

Dependence of Vector Dose and Age of Mice in the 

Induction of Immune Response Against Factor VIII 

Following Liver-Directed Nonviral Gene Transfer 

Peiqing Ye, Carol H. Miao 

614 

Frequency of T Cell Responses to AAV-2 Capsid in 

Peripheral Blood Mononuclear Cells of Normal Subjects 

Does Not Correlate with Anti-AAV2 Antibody Titers 

Denise Sabatino, Federico Mingozzi, Yi-lin Liu, Daniel Hui, 

Michael Tigges, Glenn Pierce, Katherine High 

615 

A Self-Limiting but Prolonged Inflammatory Response to 

AAV-Factor IX Gene Transfer to Skeletal Muscle 

L. Wang, R. W. Herzog 



616 

Protection of Monocrotaline-Induced Pulmonary 

Hypertension by Adeno-Associated Virus Vector- 

Mediated Interleukin-10 Expression 

Takayuki Ito, Takashi Okada, Hiroshi Miyashita, Tatsuya 

Nomoto, Yoshikazu Maeda, Mutsuko Sarukawa, Masahisa 

Shimpo, Toru Yoshioka, Takashi Matsushita, Hiroaki 

Mizukami Akihiro Kume, Keiji Yamamoto, Uichi Ikeda, 

Kazuyuki Shimada, Keiya Ozawa 

617 

A Synthetic Promoter Drives Macrophage Expression of 

ApoE and Ameliorates Atherosclerosis 

Senlin Li, Weijing He, Mei Qiang, Robert A. Reddick, Gregory 

L. Freeman 

618 

A Novel Gene Therapy Strategy for Vascular Ehlers- 

Danlos Syndrome by the Combination with RNAi 

Mediated Inhibition of a Mutant Allele and Transcriptional 

Activation of a Normal Allele 

Atsushi Watanabe, Takahito Wada, Kumiko Tei, Ryuichiro 

Hata, Yoshimitsu Fukushima, Takashi Shimada


ABSTRACTS 


619 

Co-Expression of AAV Mediated Angiopoietin-1 and 

VEGF in Ischemic Hearts Yield Leakage-Resistant Blood 

Vessels and Better Therapeutic Effect Than VEGF Alone 

Hua Su, Shuji Joho, Yu Huang, Alicia Barcena, Janice 

Arakawa-Hoyt, William Grossman, Yuet W. Kan 

620 

SMC-Specific Transcription Elements for Improved 

Adenoviral-Mediated Transgene Expression in Saphenous 

Veins 

Angelika B. Kritz, Song Wan, Anthony P. C. Yim, Paul A. 

Kingston, Andrew H. Baker 

621 

Angiogenesis and Arteriogenesis in Ischemic Skeletal 

Muscles Following VEGF Gene Transfer by Adeno- 

Associated Virus Serotype 1 Vectors 

Hua Yan, Xiaobing Wu, Lingyun Zu, Yanhong Guo, Peng 

Zhang, Wei Gao 

622 

PhiC31 Integrase as a Tool: Gene Therapy for Angiogenesis 

Joylette L. Portlock, Sohail Jarrahian, Carmen Bertoni, Thomas 

A. Rando, Michele P. Calos 

623 

Receptor-Independent Cell Entry of Adenovirus- 

Polylactide Nanoparticle Composites Leads to an 

Increased Gene Expression in Endothelial and Smooth 

Muscle Cells 

Michael Chorny, Ilia Fishbein, Ivan Alferiev, Origene 

Nyanguile, Robert J. Levy 

624 

Development of a Novel Ribbon-type NF-kðB Decoy 

Oligodeoxynucleotides to Treat Cardiovascular Diseases 

Mariana K. Osako, Naruya Tomita, Yasuo Kunugiza, Keishi 

Sekiguchi, Keiko Izawa, Ryuichi Morishita 

625 

Adenoviral-Mediated Gene Delivery of a Constitutively 

Active Form of Early Growth Response Factor-1 Increases 

Tissue Perfusion in a Murine Model of Hindlimb 

Ischemia 

Jonghoe Byun, Young-Sam Lee, Hyung-Suk Jang, Jeong-Min 

Kim, Jung-Sun Lee, Jae-Young Lee, Koung Li Kim, In-Soon 

Shin, Wonhee Suh, Jin-Ho Choi Eun-Seok Jeon, Duk-Kyung 

Kim 

626 

Ultrasound-Triggered/Enhanced Delivery of Oligonucleotides 

into Aorta 

Shao-LIng Huang, David D. McPherson, Robert C. 

MacDonald 

627 

Adenoviral-Mediated Systemic Transduction of the 

Angiotensin II (Ang II) Type 2 Receptor (AT2R) Enhances 

the Hypotensive Action of Losartan 

Hongwei Li, Yongxin Gao, Justin Grobe, Mohan Raizada, 

Michael Katovich, Colin Sumners 

628 

Adenovirus-Mediated Expression of bð-Adrenergic 

Receptor Kinase C-Terminus Reduces Intimal Hyperplasia 

and Luminal Stenosis in a Pig Model of Arteriovenous 

Polytetrafluoroethylene Graft Failure 

Zhengyu Luo, Geoffrey Y. Akita, Taro Date, Chris Treleaven, 

Karen A. Vincent, Denise Woodcock, Seng H. Cheng, Richard J. 

Grogery, Canwen Jiang 

629 

Successful Gene Therapy Against Atherosclerosis by AAV 

Delivery of IL-10 and TGFbðACT Jay L. Mehta, Yong Liu, Jaiwei Chen, Dayuan Li, Paul L. 

Hermonat 

630 

In Vivo h- VEGF Gene Transfer Improves Early 

165 

Endothelialisation and Patency in Synthetic Vascular 

Grafts 

Mika Lahtinen, Vitas Zemgulis, Pontus Blomberg, Giedrius 

Baliulis, Fredrik Carlsson, Harry Khamis, David Bergqvist 

631 

Novel Therapeutic Approach Using siRNA to Reduce 

Plasma High Concentration of Lp(a) 

Naruya Tomita, Keiko Izawa, Yasuo Kunugiza, Mariana K. 

Osako, Ryuichi Morishita 

632 

Adenoviral Mediated Nuclear Factor kappa B Super 

Repressor Gene Transfer to Rat Insulinoma Cells Protects 

Against Cytokine Induced Oxidative Damage 

Cillian T. McCabe, Timothy O’Brien 

633 

Synergistic Effects of Naked DNA Vectors Encoding 

VEGF and bFGF on Angiogenesis and Blood Flow 

Recovery in Ischemic Hindlimb 

Jung-Sun Lee†, Jonghoe Byun†, Jeong-Min Kim, Chae-Young 

Kim, Byong-Moon Kim, Ji Hyung Chung, Yangsoo Jang, 

Duk-Kyung Kim 

634 

Proliferation and Angiogenesis in Vascular Cells 

Overexpressing NOS Isoforms 

Ronan J. Cooney, Timothy O’Brien 

635 

Experimental Study on Changes of the Revascularization 

and Expression of VEGF and Its Receptors in Ischemic 

Skeletal Muscle 

Wei Liang, Baigen Zhang, Hao Zhang, Jiwei Zhang, 

Zhaoxiong Zhou

84 


ABSTRACTS 


636 

Long Term Elevation of HDL Cholesterol Induced by 

Rabbit apo A-I or Rabbit LCAT Gene Transfer Inhibits 

Media Thinning in a Rabbit Model of Advanced Atherosclerosis 

Joke Lievens, Jan Snoeys, Yingmei Feng, Désiré Collen, Bart De 

Geest 

637 

Recombinant Adeno-Associated Viral Vector 2-Mediated 

Gene Transfer of VEGF Induces Neovascular Forma- 

165 

tion and Improves Blood Flow in Chronic Ischemic 

Swine Myocardium 

Xiaoli Deng, Xiaobing Wu, Jie Jiang, Yanhong Guo, Lingyun 

Zu, Wei Gao 

638 

Combined LDL Receptor and ApoA-I Gene Therapy for 

Advanced Atherosclerosis in a Mouse Model of Familial 

Hypercholesterolemia 

Hsu Chao, Kerry Ko, Lawrence Chan, Kazuhiro Oka 

639 

VEGF Gene Expression in Patients’ Ischemic Skeletal 

Muscle with Diabetic Foot 

Zheng Wang, Jiwei Zhang, Baigen Zhang 


Session III to Poster Session II and will follow Abstract 

639. See page 101 for abstract title and authors.) 

Neurologic: Pain 

640 

A Novel Herpes Simplex Virus Vector Expressing 

Endomorphin-2: Characterization and Antinociceptive 

Properties 

Darren Wolfe, Shuanglin Hao, Jian Hu, Marina Mata, Joseph 

Glorioso, David Fink 

641 

Development of a Selectable HSV Replication System 

Based on Calcium Influx Cytotoxicity Mediated by 

Expression and Activation of TRPV1 

Darren Wolfe, Rahul Srinivasan, Shaohua Huang, David 

Krisky, Peter Friedman, Joseph Glorioso 

642 

Gene Therapy of Diabetic Neuropathy Using HSV- 

Mediated Transfer of Erythropoietin to Dorsal Root 

Ganglion In Vivo 

Munmun Chattopadhyay, Darren P. Wolfe, David M. Krisky, 

Claire P. Walter, Joseph C. Glorioso, Marina Mata, David J. 

Fink 

643 

RNA Interference Targeting EP4 Prostanoid Receptor 

Attenuates Inflammatory Pain in Rats 

Chung-Ren Lin, Haibin Wang, Clifford J. Woolf, Fumimasa 

Amaya 

644 

Gene Delivery for Chronic Pain Control: 

Micrcroencapsulated Plasmid DNA Encoding the Anti- 

Inflammatory Cytokine Gene, Interleukin-10 (IL-10) 

Erin D. Milligan, Melissa J. Mahoney, Stephen J. Langer, 

Travis Hughes, Evan M. Sloane, John Mahoney, Brian Jekich, 

Pedro E. Cruz, Terence R. Flotte, Steve F. Maier Kirk W. 

Johnson, Leslie A. Leinwand, Raymond A. Chavez, Linda R. 

Watkins 

645 

ITRs Are Necessary for IL-10 Encoding Naked Plasmid 

Induced Reversal of Chronic Pain State in Rat 

Travis Hughes, Stephen J. Langer, Evan M. Sloane, Erin D. 

Milligan, John Mahoney, Brian Jekich, Kirk W. Johnson, Ray 

A. Chavez, Steven F. Maier, Linda R. Watkins Leslie A. 

Leinwand 

646 

Zinc Finger Protein Transcription Factors as Potential 

Therapeutic Agents for the Treatment of Neuropathic 

Pain 

Siyuan Tan, Andrew McNamara, Yann Jouvenot, Dave Krisky, 

Darren Wolfe, Bradley Compos, Xiaohong Zhong, Joe Glorioso, 

H. Steven Zhang, Philip D. Gregory 

Neurologic: Spinal Cord Injury and Motor Neuron 

Disease 

647 

Expression of Neurotrophin-3 Promotes Axonal Plasticity 

in the Acute but Not Chronic Injured Spinal Cord 

Qin Chen, Lijun Zhou, H. David Shine 

648 

Lentiviral Vector Expressing Retinoic Acid Receptor bð2 

Promotes Regeneration in a Rat Spinal Cord Injury 

Model 

Liang-Fong Wong, Ping K. Yip, Anna Battaglia, John Grist, 

Martin Jones, Malcolm Maden, Susan M. Kingsman, Alan J. 

Kingsman, Stephen B. McMahon, Nicholas D. Mazarakis 

649 

Effects of GIT1on Human Neuronal (NT2N) Cells 

Ann-Shung Lieu, Jin Zhong Li, Donna J. Webb, Gerald R. 

Hankins, Gregory A. Helm 

650 

AAV-IGF1 and Exercise for Synergistic Therapies in ALS 

Brian K. Kaspar, Lindsey Christian, Lindsay Frost, Jeffrey D. 

Rothstein, Fred H. Gage


ABSTRACTS 


651 

Oligonucleotide-Mediated Gene Repair Restores Full 

Length SMN mRNA Expression in Mutant-SMN 

Murine Fibroblasts 

Emanuela Bruscia, Rafal Ochalski, Michael Rice, Giuseppe 

Novelli, Federica Sangiuolo, Diane Krause 

652 

Adeno-Associated Vector Delivery of Gutamate Transporters 

in an ALS Mouse Model 

Christine Haenggeli, Brian Kaspar, Jeffrey Rothstein 

653 

Injection of AAV Directly into the Spinal Cord Increases 

Levels of Gene Expression 

Christine Haenggeli, Jeffrey D. Rothstein, Brian K. Kaspar 

654 

Quantifying GDNF Levels in the Spinal Cord after 

Intracerebral Delivery of rAAV5 GDNF 

Kevin D. Foust, Paul J. Reier, Ronald J. Mandel, Terence R. 

Flotte 

Neurologic: Advances in Vectors, Delivery and Imaging 

655 

Compacted DNA Nanoparticles Effectively Transfect 

Brain Cells In Vitro and In Vivo 

David M. Yurek, Anita Fletcher-Turner, Mark J. Cooper 

656 

Development of Viral Vectors To Mediate Neuron- 

Specific Gene Silencing 

Colin P. Glover, Alison S. Bienemann, Beverly L. Davidson, 

James B. Uney 

657 

A Nucleic Acid-Based Transgene Reporter for In Vivo 

MRI 

Eric T. Ahrens, Clinton S. Robison, William F. Goins 

658 

Development and Evaluation of an Infusion Device for 

AAV-Mediated Gene Delivery to the Brain 

Jurg M. Sommer, Laura M. Sanftner, Haihwa Kao, Peter H. 

Smith, Brian M. Suzuki, Joseph Vargas, John R. Forsayeth, 

Janet Cunningham, Krys S. Bankiewicz, Jan Bernal Ruth 

Lessard, Glenn Pierce, Kirk W. Johnson 

659 

The Plasticity of Hematopoietic Stem Cells (HSC): Rat 

HSC Transduced In Situ by rSV40 Vectors Differentiate 

into Multiple Lineages of CNS Cells 

Jean-Pierre Louboutin, Bianling Liu, Beverley S. Reyes, 

Elizabeth J. Van Bockstaele, David S. Strayer 

660 

Quantitative Comparison of AAV Serotypes AAV2, 

AAV5, and AAVrh.10 Efficiency for CNS Gene Therapy 

Following Intracranial Gene Delivery 

Dolan Sondhi, Daniel A. Peterson, Elizabeth Vassallo, Christine 

T. Sanders, Jamie A. Stratton, Neil R. Hackett, Guangping 

Gao, James M. Wilson, Ronald G. Crystal 

661 

Efficient SV40-Mediated Gene Transfer to the Brain 

Jean-Pierre Louboutin, Beverley S. Reyes, Elizabeth J. Van 

Bockstaele, David S. Strayer 

662 

The Tracking of Exogenous Cells in the Injured Central 

Nervous System: Viral Vector Labeling Versus an Intrinsic 

Genetic Marker 

Bas Blits, Brandon M. Kitay, Andre R. Sanchez, Christian M. 

Andrade, Damien D. Pearse 

Neurologic: Sensory 

663 

Protection Against Aminoglycoside-Induced Ototoxicity 

by AAV Vector-Mediated GDNF Gene Transfer into the 

Cochlea 

Yuhe Liu, Takashi Okada, Kuniko Shimazaki, Tatsuya 

Nomoto, Kianoush Sheykholeslami, Shin-Ichi Muramatsu, 

Hiroaki Mizukami, Akihiro Kume, Shuifang Xiao, Keiichi 

Ichimura Keiya Ozawa 

664 

Regeneration of Sensory Cells in the Inner Ear of 

Deafened Mature Guinea Pigs 

Donald L. Swiderski, Masahiko Izumikawa, Douglas E. 

Brough, Yehoash Raphael 

665 

Experiences with Delivery of Nanoparticles to the Mouse 

Cochlea 

Mark Praetorius, Kim Baker, Bernhard Schick, Peter K. 

Plinkert, Hinrich Staecker 

666 

LV with Different Promoters Mediated Gene Expression 

in the Inner Ear 

Maoli Duan, Anders Fridberger, Dongguang Wei, Cecilia 

Lundberg 

667 

Lentiviral Gene Transfer of RPE65 cDNA in Knock-Out 

Mouse Models of Leber Congenital Amaurosis 

Corinne Kostic, Alexis-Pierre Bemelmans, Muriel Jaquet, Dana 

Hornfeld, William W. Hauswirth, Janis Lem, Zhongyan Wang, 

Daniel F. Schorderet, Francis L. Munier, Andreas Wenzel Yvan 

Arsenijevic

86 


ABSTRACTS 


668 

Non-Viral Gene Delivery for Ocular Diseases with 

Compacted DNA Nanoparticles 

Rafal Farjo, Jeff Skaggs, Alexander B. Quiambao, Mark J. 

Cooper, Muna I. Naash 

669 

Sonic Hedgehog (SHH) Is Required for Retinal Angiogenesis 

and Its Inhibition Prevents Retinal 

Neovascularization 

Enrico M. Surace, Alessandra Tessitore, Gabriella Cotugno, 

Aniello Vitale, Alberto Auricchio 

670 

Mutation Independent Silencing of Genes Involved in 

Retinal Degeneration by RNA Interference (RNAi) and 

Trans Complementation by a Codon Exchanged RNAi- 

Resistant Transgene 

Siobhan Cashman, Erin Binkley, Rajendra Kumar-Singh 

671 

Nerve Growth Factor and Neurotrophin-3 Delivered by 

HSV Gene Transfer Reverses Diabetic Neuropathy 

James R. Goss, Munmun Chattopadhyay, William F. Goins, 

Darren Wolfe, Marina Mata, Joseph C. Glorioso, David J. Fink 

Gene Therapy for Connective Tissue 

672 

The Effect of Serine Protease Inhibitor 6 (SPI-6) Gene 

Transfer on a Mouse Model of Sjögren’s Syndrome 

Fumi Mineshiba, Beatrijs M. Lodde, Jianghua Wang, Livia A. 

Casciola-Rosen, Antony Rosen, Bruce J. Baum 

673 

Plasmid-Based GHRH Supplementation Delivered by 

Electroporation as a Treatment of Arthritis/Laminitis in 

Horses 

Patricia A. Brown, David M. Hood, Ruxandra Draghia-Akli 

674 

Molecular, Biochemical and Biomechanical Analysis of 

Articular Cartilage Repaired with Genetically Modified 

Chondrocytes Expressing Insulin-Like Growth Factor-I 

Laurie R. Goodrich, Eric Strauss, Chris T. Chen, Chisa 

Hidaka, Paul D. Robbins, Chris H. Evans, Alan J. Nixon 

675 

Enhanced Survival of Chondrocytes over Expressing 

Insulin like Growth Factor I in Equine Cartilage Repair 

Laurie R. Goodrich, Chisa Hidaka, Mary L. Strassheim, Paul 

D. Robbins, Chris H. Evans, Alan J. Nixon 

676 

Local Stimulation of Articular Cartilage Repair by 

Transplantation of Encapsulated Chondrocytes 

Overexpressing Human FGF-2 In Vivo 

Gunter Kaul, Magali Cucchiarini, David Zurakowski, Stephen 

B. Trippel, Dieter Kohn, Henning Madry 

677 

Gene Delivery of TNFR:Fc by Adeno-Associated Virus 

Vector Blocks Progression of Periodontitis 

Mario Taba Jr, Heather H. Huffer, Charles E. Shelburne, 

Jaclynn M. Kriegl, Steven A. Goldstein, Kurt H. Lustig, Haim 

Burstein, William V. Giannobile 

678 

Inflammatory Cytokines Regulate Transgene Expression 

from Rheumatoid Synoviocytes Infected with Double 

Stranded Adeno-Associated Virus Vectors 

Russell S. Traister, Sylvie Fabre, Xiao Xiao, Raphael Hirsch 

679 

Establishment of New IL-6 Receptor Inhibitor Applicable 

to the Gene Therapy 

Yasuo Adachi, Naoko Yoshio, Chieko Aoki, Alexander Pereboev, 

David T. Curiel, Masato Yamamoto, Norihiro Nishimoto 

680 

Application of Gene Therapy to Tendon Healing: 

Modification of Tenocytes with Exogenous PDGF and 

VEGF Genes to Promote Collagen Production 

Jin Bo Tang, Xiao Tian Wang, Paul Y. Liu 

681 

Hydrogel Encapsulation of Adenovirus-Transduced Cells 

Expressing BMP2 for Local Osteoinduction 

Malavosklish Bikram, Christine Fouletier-Dilling, Andre M. 

Gobin, Alan R. Davis, Elizabeth A. Olmsted-Davis, Jennifer L. 

West 

682 

Over-Expression of Follistatin-Like Protein Exacerbates 

Collagen Induced Arthritis 

Takako Miyamae, Anthony D. Marinov, David C. Wilson, 

Raphael Hirsch 

683 

Adenoviral Vector-Mediated Expression of Bone Morphogenetic 

Protein-4 in Mesenchymal Stem Cells 

Induces Differentiation into the Osteoblastic Lineage and 

Bone Production In Vivo 

Barbara Lombardo, Rosa Di Noto, Maria Teresa Esposito, 

Sabrina Battista, Gabriela Ciapetti, Ombretta Capitani, Luigi 

Nicolais, Paolo Netti, Francesco Salvatore, Lucio Pastore 

684 

Viral Gene Therapy in Tendon Healing: Adeno-Associated 

Virus-2 but Not Other Serotypes Effectively 

Transduces Intrasynovial Tenocytes with Persistent 

Expression of the Transgene 

Xiao Tian Wang, Paul Y. Liu, Jin Bo Tang


ABSTRACTS 


685 

Inhibitory Effect of Ribbon-type NF-kB Decoy 

Oligodeoxynucleotides on Osteoclast Induction and 

Activity 

Yasuo Kunugiza, Tetsuya Tomita, Naruya Tomita, Mariana 

Kiomy Osako, Takuji Kizawa, Keishi Sekiguchi, Ryuichi 

Morishita, Hideki Yoshikawa 

686 

Bone Formation Inhibitors in Animal Sera 

Jin Zhong Li, Ann-Shung Lieu, Gerald R. Hankins, Gregory 

A. Helm 

687 

Tracking Transgene Product-Specific T Cells In Vivo 

Lixin Wang, E. M. Lord, Roland Herzog 

688 

Adno-Associated Virus (AAV)-Mediated Catalase 

Overexpression Protects the Extensor Digitorum 

Longous Muscle from Contraction-Induced Injury 

Mingju Liu, Yongping Yue, Dongsheng Duan 

689 

Therapeutic Angiogenesis for Prefabrication of Ischemic 

Flaps with Multiple Growth Factor Gene Transfers: A 

Study in a Rat Model 

Paul Y. Liu, Kan Liu, Xiao Tian Wang, Kimberly M. Rieger- 

Christ, Jin Bo Tang, Ian C. Summerhayes 

690 

Real Time Imaging of Myoblast Transplantation Using 

the Human Sodium Iodide Symporter (hNIS) as Reporter 

Gene 

Manaf Bouchentouf, Basma F. Benabdallah, Joel Rousseau, 

Marcel Dumont, Jacques P. Tremblay 

Cancer - Immunotherapy: Cytokine Transgenes and 

Vaccinations 

691 

Ad-mda7/IL-24 Induces Systemic Anticancer Immunity 

In Vivo 

Ryo Miyahara, Sanjeev Banerjee, Kouichiro Kawano, Clay 

Efferson, Naotake Tsuda, Yasuko Miyahara, Constantine G. 

Ioannides, Sunil Chada, Rajagopal Ramesh 

692 

Bone Marrow Derived Neural Stem-Like Cells Expressing 

IL-27 Exhibit Antitumor Activity in Intracranial 

Gliomas 

Xiangpeng Yuan, Jinwei Hu, Takayuki Yoshimoto, Keith L. 

Black, John S. Yu 

693 

Regression of Established Subcutaneous B16 Melanoma 

Tumors after Intratumoral Delivery of a IL-15 Expressing 

Plasmid Followed by In Vivo Electroporation 

Kenneth E. Ugen, Michelle Kutzler, Jeffrey Westover, Barbara 

Marerro, Chuanhai Cao, David B. Weiner, Richard Heller 

694 

Breast Cancer Specificity, Anti-Tumor Activity, and 

Reduced Toxicity of an Adenoviral Vector Encoding 

Interleukin-2 under Control of the Human 

Mammaglobin Promoter 

Paggy Hew, Changxin Shi, Melissa Paiva, Jen Robertson, 

Chuyan Ying, Frank L. Graham, Christina Addison, Jack 

Gauldie, Mary M. Hitt 

695 

Cooperative Effects of Adenoviral Vector-Mediated 

Interleukin 12 Gene Therapy and Radiotherapy in a 

Preclinical Model of Metastatic Prostate Cancer 

Tetsuo Fujita, Terry L. Timme, Koji Naruishi, Nobuyuki 

Kusaka, Julie X. Zhu, Guang Yang, Alexei Goltsov, Chenghui 

Ren, Chengzhen Ren, Maria T. Vlachaki Bin S. Teh, E. Brian 

Butler, Timothy C. Thompson 

696 

Tumor Vaccine Combined with Cytokines and Suicide 

Gene Therapy for Canine Spontaneous Melanoma 

Liliana M. E. Finocchiaro, Marie E. Maminska, Pablo J. J. 

Castillo, Armando L. Karara, Gabriel L. Fiszman, María D. 

Riveros, Gerardo C. Glikin 

697 

Intratumoral Injection of Cytokine-Secreting Syngeneic/ 

Allogeneic Fibroblasts Transfected with Tumor DNA 

Prolongs the Survival of Mice with an Intracerebral Tumor 

Terry Lichtor, Roberta P. Glick, InSug O-Sullivan, Edward P. 

Cohen 

698 

IL-23 Transduced Dendritic Cells Produce a Potent 

Tumor-Specific Immunity Against Intracranial Gliomas 

Jinwei Hu, Xiangpeng Yuan, John M. Ong, Christopher J. 

Wheeler, Maria L. Belladonna, John S. Yu, Keith L. Black 

699 

Evaluation of Potential Toxicity of Electroporation 

Mediated Deliver of a Plasmid Encoding for IL-12 in a 

Mouse Melanoma Model 

Richard Heller, Adil Daud, Jeffrey Westover, Kathleen Merkler, 

Yolmari Cruz, Richard Gilbert, Loree Heller 

700 

Regression of Squamous Cell Carcinoma by Single 

Administration of IL-12 and B7.1 Genes Via 

Electroporation 

Jiaguo Liu, Marina Torrero, Shulin Li

88 


ABSTRACTS 


701 

Enhanced Anti-Tumor Effect of Interferon Gene Transfer 

by Sustained Transgene Expression Using CpG-Reduced 

Plasmid DNA 

Hiroki Kawano, Makiya Nishikawa, Masaru Mitsui, 

Yoshinobu Takakura 

702 

Concurrent Delivery of GM-CSF and B7-1 Using an 

Oncolytic Adenovirus Elicits Potent Anti-Tumor Effect 

on the Growth Established B16-F10 Tumor Model 

Kyung-Ju Choi, Jaesung Kim, Jing Hwa Huang, Minjung 

Kim, Beum-Seok Suh, Hogeun Kim, Byoung Chul Cho, Joo- 

Hang Kim, Chae-Ok Yun 

703 

Combination Therapy with Bleomycin and IL-12 Gene 

Induces Regression of Breast Cancer 

Marina N. Torrero, Shulin Li 

704 

Tumor Cell Surface Display of the Immunoglobulin 

Heavy Chain F by Gene Transfer as a Means to Mimic 

c 

Antibody Therapy 

Susanne Lang, David Riddle, Laura Sanz, Heung Chong, Jill 

Thompson, Richard Vile 

705 

A New Strategy for Cancer Therapy: Application of 

Adenovirus-Mediated Complete-Length Antibody Gene 

Chen Jie, Jiang Minghong, Guo Minggao, Su Changqing, 

Wang Xinghua, Cui Zhenfu, Liu Xinyuan, Wu Mengchao, 

Qian Qijun 

706 

DNA Vaccine Strategy Against Chronic B-Cell Lymphoma: 

Anti-Idiotypic CDR3 Vaccination 

Monica Rinaldi, Daniela Fioretti, Sandra Iurescia, Emanuela 

Signori, Pasquale Pierimarchi, Giancarlo Tonon, Vito Michele 

Fazio 

707 

Transduction and Expansion of T Lymphocytes Genetically 

Engineered To Target the CD19 Antigen for the 

Treatment of CLL Using Xcyte Dynabeads® 

Jolanta Stefanski, Renier J. Brentjens, Mark L. Bonyhadi, 

Michel Sadelain, Isabelle Rivière 

708 

Combination of Allogeneic Hematopoietic Stem Cell 

Transplantation and Allogeneic MHC Gene Transfer 

Against Solid Cancers 

Kazunori Aoki, Masaki Ohashi, Miwa Kushida, Kimiko 

Yoshida, Masaki Mandai, Gary J. Nabel, Teruhiko Yoshida 

709 

Comparison of the GVHD-Inducing Potential and GCV 

Sensitivity of Naïve and Transduced and Selected CD34- 

TK-Expressing Murine T Cells after Allogeneic Bone 

Marrow Transplantation 

Michael P. Rettig, Julie K. Ritchey, Bruno Nervi, Mark L. 

Bonyhadi, John F. DiPersio 

Cancer - Targeted Gene Therapy: Targeting of Non- 


710 

Tissue-Specific Gene Therapy by Replication-Competent 

Retroviral Vector for Prostate Cancer with Fludarabine 

Phosphate 

Choichiro Ozu, Eiji Kikuchi, Silvia Menendez, Carlos C. 

Cardo, Christopher R. Logg, Noriyuki Kasahara, Bernard H. 

Bochner 

711 

Treatment of Acute Myeloid Leukemia by rAAV 8 Vector 

Mediated Human Interferon-bð Gene Transfer 

Reuben Benjamin, Andrew M. Davidoff, Pizzey Arnold, Nalini 

Singh, Asim Khwaja, Jenny McIntosh, Cathy C. Y. Ng, Tony 

Meager, Meenu Wadhwa, Amit C. Nathwani 

712 

Treatment of Human and Rat Hepatocellular Carcinomas 

by Long Term Expression of rAAV-Mediated Transfer of 

Human TERTC27 Polypeptide 

Marie C. Lin, Samuel S. Ng, Yi Gao, ChingTung Lum, 

ZhenFan Yang, JunJian Huang, George K. Lau, SheungTat 

Fan, Hsiang-fu Kung 

713 

Growth Suppression of Hepatocellular Carcinoma by 

AAV-Mediated Angiogenic Inhibitors 

Lai-Yin Tse, Ruian Xu 

714 

Adenoassociate Virus Mediated Long Term Expression of 

Human TERTC27 Polypeptide Inhibited Glioblastoma 

Carcinogenesis by Multiple Mechanisms 

Samuel S. Ng, Yi Gao, JunJian Huang, Cui-fen Huang, 

Hsiang-fu Kung, Marie C. Lin 

715 

Noninvasive Bioluminescence Imaging of Gene Expression 

Following Intracerebral Gene Delivery of AAV and 

Adenovirus into the Mouse Brain 

Perry Liu, Hideyuki Migita, Heather Gibson, Harald Petry, 

Linda Cashion, Terry Hermiston, Katalin Kauser, Rick 

Harkins, Gabor M. Rubanyi, Ron Vergona Hu Sheng Qian 

716 

Transducing Efficiency Screening for rAAV2, rAAV1, and 

rAAV5 in 56 Human Tumor Cell Lines 

Lina Li, Linda Yang, Dominic A. Scudiero, Robert H. Shoemaker, 

Robert M. Kotin


ABSTRACTS 


717 

Cancer Gene Therapeutic Approaches Using an AAV- 

Mediated Gene Delivery System Containing Antisense 

VEGF-A cDNA 

Keerang Park, Mee-Young Ahn, Seock-Yeon Hwang, Young- 

Hwa Cho, Bong-Su Kang, Youn-Ju Kim, Sung-Ha Cho, 

Young-Min Kim, So-Yong Yi, Sun-Kyung Kim Eun-Jung 

Park, Kang-Eun Lee 

718 

Development of AAV and Adenovirus Cocktail Vector 

System for Efficient Cancer Gene Therapy by Human 

TERTC27 Polypeptide 

Yi Gao, Samuel S. Ng, Chen Yang, David Chau, JunJian 

Huang, Cui-fen Huang, Hsiang-fu Kung, Marie C. Lin 

719 

Neutralization of Free but Not Cell Associated Oncolytic 

Measles Vurses by Antibody and Complement 

Ianko Iankov, Stephen J. Russell 

720 

EGFRvIII Retargeted Oncolytic Measles Virus Strains 

Have Significant Antitumor Activity Against Gliomas 

Cory Allen, Sompong Vongpunsawad, Takafumi Nakamura, C. 

David James, Mark Schroeder, Roberto Cattaneo, Stephen J. 

Russell, Evanthia Galanis 

721 

Measles Virus for Cancer Gene Therapy of Primary Liver 

Tumors 

Boris R. A. Blechacz, Patrick Splinter, Greiner Suzanne, Mark 

J. Federspiel, Kah-Whye Peng, Nicholas F. LaRusso, Stephen J. 

Russell 

722 

Antitumor Effect of IL-24 Mediated by Tumor Targeting 

Vector ZD55 Is Much Better Than That Mediated by 

Replication Deficient Adenovirus 

Li-li Zhao, Jin-fa Gu, Ai-wen Dong, Yan-hong Zhang, Lingfeng 

He, Jin-he Zhang, Yi-gang Wang, Zi-lai Zhang, Qi-jun 

Qian, Cheng Qian Xin-yuan Liu 

723 

VSV-G Pseudotyped, Semi-Replication Competent MLV 

Retroviruses for Cancer Gene Therapy 

Jian Qiao, Jesus Moreno, Timothy Kottke, Michael Forshaw, 

Rosa Maria Diaz, Richard Vile 

724 

Herpes Simplex Virus 1 Amplicon Vector-Mediated 

siRNA Targeting Epidermal Growth Factor Receptor 

Inhibits Growth of Human Glioma Cells in Culture and 

In Vivo 

Okay Saydam, Daniel Glauser, Irma Heid, Gulen Turkeri, 

Andreas Jacobs, Mathias Ackermann, Cornel Fraefel 

725 

Seneca Valley Virus, a Novel Systemically Deliverable 

Oncolytic Virus for the Treatment of Small Cell Lung 

Cancer and Other Neuroendocrine Cancers 

Paul L. Hallenbeck, Seshidhar P. Reddy, Shanthi Ganesh 

726 

Effect of G-CSF-Mediated Mobilization on Tumor 

Homing of Genetically Modified, Bone Marrow Derived 


Zong-Yi Li, Shaoheng Ni, Hans-Peter Kiem, Andre Lieber 

727 

Gene-Modified Mesenchymal Stem Cells Selectively 

Engraft in Stroma of Ovarian Carcinomas and Control 

Tumor Growth 

Jennifer Dembinski, Matus Studeny, Claudia Zompetta, 

Michael Andreeff, Frank Marini 

728 

Anti-TAG-72 Immunoliposomes for Efficient Tumor- 

Targeted Gene Therapy 

Keun-Sik Kim, Hong Sung Kim, Eun Jung Kim, Jong Chul 

Kim, In-Ho Song, Hyo Jeong Hong, Yong Serk Park 

729 

C-Met Antisense Plasmid Liposome Complex Increases 

Radiation Sensitivity of Lung Tumor Cells In Vivo or In 

Vitro 

Yunyun Niu, Laura Stabile, Michael W. Epperly, Jill Siegfried, 

Joel S. Greenberger 

730 

Cancer Specific Promoters for Gene Therapy of Small 

Cell Lung Cancer 

Nina Pedersen, Mikkel W. Pedersen, Michael S. Lan, Thomas 

T. Poulsen, Hans S. Poulsen 

731 

Completely Elimination of Xenograft SW620 Cancer by 

Combined Tumor Targeting MnSOD and Trail Genes 

Yan-hong Zhang, Jin-fa Gu, Li-li Zhao, Yi-gang Wang, Jinhui 

Wang, Ling-feng He, Wei-guo Zou, Qi-Jun Qian, Cheng 

Qian, Xin-yuan Liu 

732 

Defining Tropism of Oncolytic Vectors by Protease 

Availability: Measles Viruses Selectively Fusing Matrix- 

Metalloproteinase Expressing Cells 

Christoph Springfeld, Veronika von Messling, Christian 

Buchholz, Roberto Cattaneo 

Gene Regulation: Overcoming Obstacles to Gene 

Transfer and Expression 

733 

The Human XIST Gene Promoter Prevents Silencing of 

an Integrated Reporter Gene 

Michael R. Greene, Christopher H. Lowrey

90 


ABSTRACTS 


734 

Association of DNA Polymerase mð with Recombinant 

Adeno-Associated Viral Vectors 

David C. Brooks, Fayaz R. Khazi, Katherine A. High 

735 

Femtosecond Laser- A New Intradermal DNA Delivery 

Method for Gene Expression and Vaccination 

Evelyne Zeira, Alexandra Manevitch, Zakharia Manevitch, 

Menahem Harati, Hagit Yotvat, Michal Gropp, Orit Papo, Nili 

Daudi, Eli Kedar, Aaron Lewis Eithan Galun 

736 

Novel In Vitro and In Vivo Transcriptome Analysis 

Identifies New Adenovirus Responsive, Innate Gene 

Networks 

Zachary Hartman, Anne Kiang, Ruth Everett, Joe Nevins, 

Esther Black, Andy Amalfiatano 

737 

Induction of Molecular Chimerism in Rhesus Macaques 

Reconstituted with Autologous CD34+ Cells Transduced 

with the Gene Encoding the að(1- 

3)galactosyltranferase (aðGT) 

Lorenzo Benatuil, Robert Donahue, Aylin Bonifacino, Cynthia 

Dunbar, John Iacomini 

738 

Influence of the Major Histocompatibility Complex on 

Immunological Rejection of Skin Grafts Expressing a 

Gene Therapy Reporter Gene 

Julian D. Down, Christian LeGuern 

739 

Impact of Activation, Transduction/Selection Steps on the 

Gene Expression Profile of Ex-Vivo Retrovirally Transduced 

CD8+ T Cells, Using High Density DNA 

Microarrays 

Marina Deschamps, Jean Marie Certoux, John de Vos, Nicolas 

Montcuquet, Thierry Reme, Mark Bonyhadi, Eric Robinet, 

Pierre Tiberghien, Christophe Ferrand 

740 

In Vitro Correction of 47 bp Deletion in Casp-3 Gene in 

Human MCF7 Cells Using Histone-Mediated Delivery 

of Wild Type Gene Fragment Covering the Deletion 

Region 

Leonid A. Yakubov, Robert J. Levy 

741 

AAV Delivered Ribozymes to Recapitulate the Early 

Stages of Age Related Macular Degeneration 

Verline Justilien, Jijing Pang, William W. Hauswirth, Alfred S. 

Lewin 

742 

Human Artificial Chromosome (HAC) Vector Provides 

Long-Term Therapeutic Transgene Expression in Normal 

Human Primary Fibroblasts 

Minoru Kakeda, Masaharu Hiratsuka, Keiko Nagata, Akiko 

Sano, Kanako Osawa, Motonobu Katoh, Mitsuo Oshimura, 

Kazuma Tomizuka 

743 

Lentiviral Vector Gene Transfer in Monkeys: In Vivo 

Detection of Gene Expression Longitudinally Using 

MicroPET and Optical Imaging 

Alice F. Tarantal, Chang I. Lee, Daniel F. Jimenez, Sanjiv S. 

Gambhir, Donald B. Kohn, John J. Rossi, Simon R. Cherry 

744 

Enhanced Expression of Adenovirus Transgenes in the 

Mouse Liver and Human Tumors Following 

Topoisomerase I Inhibition 

Gideon Zamir, Evelyne Zeira, Eithan Galun 

745 

CD4-T Cell Responses Mediated by IFNgð and Perforin 

Eliminate Adenoviral-Mediated Transgene Expression 

from the CNS of Mice by Cytolytic and Non-Cytolytic 

Mechanisms 

Jeffrey M. Zirger, Carlos Barcia, Chunyan Liu, Maria Castro, 

Pedro Lowenstein 

746 

Evaluation of Nuclear Transfer and Transcription of 

Plasmid DNA Condensed with Protamine 

Tomoya Masuda, Hidetaka Akita, Hideyoshi Harashima 

747 

Identification and Characterization of Mutations within 

the Coxsackievirus and Adenovirus Receptor (CAR) 

Katherine J. D. A. Excoffon, Matthew Avenarius, Richard 

Smith, Joseph Zabner 

748 

Quantitative Comparison of the Intracellular Trafficking 

and Expression Pattern of the Exogenous Genes between 

the Adenovirus and Artificial Vector 

Susumu Hama, Hidetaka Akita, Rie Ito, Hiroyuki Mizuguchi, 

Takao Hayakawa, Hideyoshi Harashima 

749 

Characterization of Adenovirus-Mediated Gene Expression 

in Normal, Wounded and Wound Impaired Skin 

Danling Gu, David Looper, David W. Kang, Diane McAllister, 

Erlinda Quijano, Shu Fen Wen, Monica L. Zepeda 

750 

Expression Levels of the Amphotropic Receptor May 

Explain Gestational Age-Dependence of Tissue Transduction 

in Fetal Sheep 

Ferhat Ozturk, Paul Park, Joe Tellez, Wayne B. Anderson, 

Maribeth Eiden, Esmail D. Zanjani, Graca Almeida-Porada, 

Christopher D. Porada


ABSTRACTS 


751 

High Efficient Ex Vivo Gene Delivery into Human 

Corneal Endothelial Cells by Recombinant Adeno- 

Associated Virus 

Li-Ju Lai, Juan Li, Samuel Chao-Ming Huang, Ken-Kuo 

Lin, Xiao Xiao 

752 

Equine Infectious Anemia Virus Pseudotyped with the 

Vesicular Stomatitis Virus G-Protein, Preferentially 

Targets Neural Precursors in the Adult Mouse Brain 

Troy Ghashghaei, Manij Patel, John Olsen, Eva Anton 

753 

Efficient Gene Therapy by Intercellular Spread and 

Specific Internalization of the Transgene Product 

Antoine M. Beerens, Marianne G. Rots, Hidde J. Haisma 

754 

Different Regulation of Myocardial Atrial and Ventricular 

Calcium Pump Activity (Ca-ATP-ase) during Cardiac 

Arrest 

Ernoe Remsey-Semmelweis, John G. Kral, Richard B. Wait, M. 

A. Q. Siddiqui 



DNA Viruses: Vaccines, HSV, Baculovirus and SV40 


755 10:15 AM 

Vaccinia Deleted Multiple Anti-Apoptosis Viral Genes 

Enhances Its Specificity as an Oncolytic Virus 

Shuting Yang, Zongsheng Guo, Mark E. O’Malley, David L. 

Bartlett 


The Use of Immune Cell Trafficking to Deliver an 

Oncolytic Virus to Tumors 

Steve H. Thorne, Robert Negrin, Christopher H. Contag 


Replication Defective HSV-1 Immediate Early (IE) Gene 

Mutants: A Comparison of Vector Toxicity Versus 

Transgene Expression 

David M. Krisky, James Wechuck, Darren Wolfe, Shaohua 

Huang, Ali Ozuer, Joseph C. Glorioso 


A Multipurpose Vector System for the Simultaneous 

Screening of Libraries in Bacteria, Insect and Mammalian 

Cells and Expression In Vivo 

Olli H. Laitinen, Kari J. Airenne, Vesa P. Hytönen, Erik 

Peltomaa, Anssi J. Mähönen, Thomas Wirth, Miia M. Lind, 

Kari A. Mäkelä, Pyry I. Toivanen, Diana Schenkwein Tommi 

Heikura, Henri R. Nordlund, Markku S. Kulomaa, Seppo 

Ylä-Herttuala 


Liver Transduction with an SV40 Vector Encoding 

Insulin-Like Growth Factor I Reduces Hepatic Damage 

and Delays Cirrhosis Progression 

Maria Vera, Laura Martinez, Mikel Zaratiegui, Carlos 

Rodriguez, Jesus Prieto, Puri Fortes 


Development of an HSV-Based Model System to Identify 

Factors Acting in Embryonic Myogenesis 

April M. Sunyog, Darren P. Wolfe, David Krisky, James 

Wechuck, Ying Jiang, Joseph C. Glorioso 


Dominant-Negative FGF Receptor Expression by 

Oncolytic HSV Vector for the Treatment of Malignant 

Peripheral Nerve Sheath Tumors 

Ta-Chiang Liu, Tingguo Zhang, Hiroshi Fukuhara, Toshi 

Kuroda, Robert L. Martuza, Andreas Kurtz, Samuel D. 

Rabkin

92 


ABSTRACTS 


762 12:00 PM 

Cisplatin-Induced DNA Damage Repair Mechanism 

Potentiates the Synergistic Efficacy of Oncolytic Herpes 

Simplex Viral Therapy in the Treatment of Malignant 

Mesothelioma 

Prasad S. Adusumilli, Mei-Ki Chan, David P. Eisenberg, 

Zhenkun Yu, Karen Hendershott, Ting-Chao Chou, Valerie W. 

Rusch, Yuman Fong 


Naked DNA Gene Transfer: Optimizing Expression/ 

Animal Models 

Room: 260/264 


Optimization of Plasmid Components for Gene Therapeutic 

Purposes 

Ruxandra Draghia-Akli, Melissa A. Pope, Amir S. Khan 


RNA and Codon Optimized HIV Candidate Vaccines – 

from Bench to Clinical Trials 

Ralf Wagner, Petra Mooij, Sunita Balla, Jens Wild, Kurt Bieler, 

Hans Wolf, Frank Notka, Jonathan Heeney, Giuseppe Pantaleo 


Intravascular Delivery of Naked Plasmid DNA into 

Muscle for Long-Term Correction of Hyperbilirubinemia 

in the Gunn Rat 

Istvan Danko, Zhen Jia 


Bone Regeneration Induced by Combining In Vivo 

Electroporation of an Osteogenic Gene and Human 

Mesenchymal Stem Cells 

Gadi Pelled, A Lazarus, Yoram Zilberman, Eveline Zeira, 

Hagit Yotvat, Eithan Galun, Jin Zhong Li, Gregory A. Helm, 

Dan Gazit 


Role of the Cytoskeleton in Intracellular Plasmid Movement 

Erin E. Vaughan, David A. Dean 


Two Distinct Mechanisms, Silencing of RNA Expression 

and Loss of Vector DNA, Are Responsible for the Loss of 

Transgene Expression Following Delivery of Foreign 

Genes to Skeletal Muscle 

Alan R. Brooks, Peiyin Wang, Paul Szymanski, Perry Liu, 

Heather Gibson, Hu Sheng Qian, Rick N. Harkins, Gabor M. 

Rubanyi 


Tissue Engineering Scaffolds with Non-Viral Vectors: 

Sustained and Substrate-Mediated Delivery 

Jae-Hyung Jang, Christopher B. Rives, Zain Bengali, Lonnie 

D. Shea 

770 12:00 PM 

Novel Physical Methods for Nonviral DNA Transfer to 

Rabbit Retina 

Thomas W. Chalberg, Alexander Vankov, Philip Huie, Fanni E. 

Molnar, Michele P. Calos, Daniel V. Palanker 



Systems 

Room: 263/267 


Complexes of Nucleic Acids with Multivalent Cationic 

Lipids for the Study of Delivery Pathways and Mechanisms: 

From Plasmid DNA to siRNA 

Kai Ewert, Nathan F. Bouxsein, Christopher S. McAllister, 

Heather M. Evans, Charles E. Samuel, Cyrus R. Safinya 


Convective Flow Increases Lipoplex Delivery Rate to In 

Vitro Cellular Monolayers 

Steven S. Harris, Todd D. Giorgio 


Role of Lipid-Rafts in the Binding and Internalization of 

Dendriplexes and Lipoplexes 

Peng H. Tan, Maria Manunta, Andrew J. T. George 


Studies on the Intracellular Release of Genetic Drugs 

from Pharmaceutical Carriers 

Bart Lucas, Katrien Remaut, Stefaan C. De Smedt, Roosmarijn 

Vandenbroucke, Joseph Demeester 


Substrate-Mediated DNA Delivery for Neural Tissue 

Engineering 

Tiffany L. Houchin, Jae-Hyung Jang, Lonnie D. Shea 


A Novel Dual Functioning Non-Viral Vector - Delivering 

Genes While Targeting Nuclear Receptor Signaling 

Feng Liu, Christine Conwell, Lisa Scollenbergen, Leaf Huang 


Polymer Library Approaches to Gene Therapy 

Daniel G. Anderson, Andreas Zumbuehl, Weidan Peng, Janet 

A. Sawicki, Robert Langer 

778 12:00 PM 

Nocodazole Enhances CMV Promoter Activity in a 

Human Pancreatic Cancer (Panc 1) Cell through Activation 

of c-Jun 

Robert Arpke, Pi-Wan Cheng


ABSTRACTS 



Molecular Conjugates: Therapeutic Applications 

Room: 120/124 


Oral Delivery of Non-Viral DNA Nanoparticles for 

Hemophilia A 

Katherine Bowman, Rita Sarkar, Sanj Raut, Neelima Shah, 

Xuli Wang, Hai-Quan Mao, Kam W. Leong 


A Novel Multi-Component Molecular Conjugate Vector 

for Highly Efficient p53-Mediated Tumor-Specific Gene 


Stanley Saamoah-Moffatt, Sandra Wiehle, Richard J. Cristiano 


The Effect of Overexpression of Lysyl Oxidase on 

Dermal Wound Healing 

Ying-Ka I. Lau, Jennifer L. West 


Poly(glycoamidoamine)s Deliver DNA with High 

Efficacy to Cardiomyoblast Cells 

Yemin Liu, Suiwen He, W. Keith Jones, Theresa M. Reineke 


Transient Transduction of Repopulating Human CD34+ 

Cells Using Nanoparticles in a Clinically Applicable Ex 

Vivo Protocol 

Jesper Bonde, Dustin J. Maxwell, David A. Hess, Ryan Lahey, 

David Piwnica-Worms, Jan A. Nolta 


Promoter-Dependence of Plasmid-Pluronics Targeted 

alpha-Galactosidase A Expression in Skeletal Muscle of 

Fabry Mice 

Matthieu D. Lavigne, Marita Pohlschmidt, Javier F. Novo, 

Valery Alakhov, Dariusz C. Gorecki 


Cell-Permeable Peptides Mediated Gene Delivery in 

Hepatocytes In Vitro and In Vivo 

Zhenping Shen, Carol H. Miao 

786 12:00 PM 

Synthesis of Cationic Biodegradable Polymers for 

Targeted Gene Delivery to Hepatocytes 

Gregory T. Zugates, Daniel Anderson, Ingrid Lawhorn, Robert 

Langer 




Room: 230/231 


Lentivirus Mediated Dystrophin Expression in mdx 

Muscles 

En Kimura, Sheng Li, Brent Fall, Miki Haraguchi, Leonard 

Meuse, Jeffery S. Chamberlain 


Antisense Therapy for Duchenne Muscular Dystrophy: A 

Realistic Possibility 

Qi L. Lu, Adam Rabinowitz, HaiFang Yin, Julia Alter, George 

Bou-Gharios, Terrence Partridge 


Stable Genome Alteration of the Dystrophin Gene for 

the Treatment of Duchenne Muscular Dystrophy 

(DMD) Due to Frame-Shift Mutations Using Oligonucleotide-Mediated 

Gene Editing 

Carmen Bertoni, Thomas A. Rando 


Evaluation of Immune Responses to Dystrophin in 

Humanized mdx HLA-A*0201 Dystrophic Mice: 

Application in Duchenne Muscular Dystrophy Gene 

Therapy after U7 snRNA-Mediated Exon Skipping 

Florent Ginhoux, Sabrina Turbant, Marylène Leboeuf, David 

A. Gross, François Lemonnier, Luis Garcia, Olivier Danos, Jean 

Davoust 


Immunity to AAV-Mediated Gene Therapy in a Random- 

Bred Canine Model of Duchenne Muscular Dystrophy 

Zejing Wang, Michael J. Blankinship, Paul Gregorevic, Marie- 

Terese Little, Rainer J. Storb, James M. Allen, Stephen J. 

Tapscott, Jeffrey S. Chamberlain, Christian S. Kuhr 


Canine Mini-Dystrophin Gene Transfer by AAV1 in mdx 

Mice Ameliorates Dystrophic Pathology and Protects 

Membrane Integrity 

Bing Wang, Juan Li, Liqiao Zhou, Chunping Qiao, Mengnan 

Tian, Tong Zhu, Janet Bogan, Joseph Kornegay, Xiao Xiao 


Efficient Expression of the 6 kb Mini-Dystrophin Gene 

by Trans-Splicing Adeno-Associated Viral (AAV) Vector 

Restores the Entire Dystrophin-Associated Glycoprotein 

Complex and Reduces Contraction-Induced Damage in 

the Mdx Mouse Model of Duchenne Muscular Dystrophy 

Yi Lai, Yongping Yue, Mingju Liu, Arka Ghosh, John F. 

Engelhardt, Jeffrey S. Chamberlain, Dongsheng Duan

94 


ABSTRACTS 


794 12:00 PM 

Evaluation of Gene Transfer Efficacy Mediated by AAV1 

and AAV6 Vectors in Skeletal Muscle of Adult Mice 

Following Different Routes of Administration 

Christel Rivière, Karine Poulard, Gabor Veres, Olivier Danos, 

Anne M. Douar 


Cellular Immunotherapy of Cancer Using Genetically 

Modified Effectors 



The Use of Autologous LMP2-Specific Cytotoxic T 

Lymphocytes for the Treatment of Relapsed EBV +ve 

Hodgkin Disease and Non-Hodgkin Lymphoma 

Catherine M. Bollard, Stephen Gottschalk, Elizabeth Buza, 

Helen Huls, Vicky Gresik, George Carrum, Malcolm K. 

Brenner, Cliona M. Rooney, Helen E Heslop 


In Vivo Elimination of Human Leukaemia Cells by WT1 

Specific TCR Transduced T Cells 

Shao-An Xue, Liquan Gao, Daniel Hart, Waseem Qasim, 

Adrian Thrasher, Jane Apperley, Wolfgang Uckert, Emma 

Morris, Hans Stauss 


Targeted Elimination of Prostate Cancer by Genetically 

Directed Human T Lymphocytes 

Matthias Stephan, Terence P. F. Gade, Waleed Hassen, Elmer 

Santos, Gertrude Gunset 


Epstein Barr Virus-Specific Cytotoxic T Lymphocytes 

(EBV-CTL) Expressing an Anti-CD30 Chimeric T-Cell 

Receptor (cTcR) for the Treatment of Hodgkin’s Disease 

(HD) 

Barbara Savoldo, Cliona Rooney, Helen Heslop, Hinrich 

Abken, Andreas Hombach, Lan Zhang, Martin Pule, 

Gianpietro Dotti, Malcolm Brenner 


In Vitro and In Vivo Characterization of “Second-Generation” 

Co-Stimulatory Chimeric Antigen Receptors 

(CARs) Targeting the CD19 Antigen Present on B Cell 

Malignancies 

Renier J. Brentjens, Yan Nikhamin, Miko Matsushita, Michel 

Sadelain 


Adenoviral Transduced Monocytes: Efficient Antigen 

Presenting Cells for the Activation of Antigen-Specific T 


Ann M. Leen, Maheshika K. Ratnayake, Aaron E. Foster, 

Cliona M. Rooney, Stephen Gottschalk 


Fas Knockdown Mediated by siRNA in EBV-Specific 

Cytotoxic T-Lymphocytes (CTL) Reduces Their Sensitivity 

to Fas/FasL-Induced Apoptosis 

Gianpietro Dotti, Barbara Savoldo, Martin Pule, Karin C. 

Straathof, Ettore Biagi, Eric Yvon, Sthephane Vigouroux, 

Cliona M. Rooney, Malcolm K. Brenner 

802 12:00 PM 

Use of Chemokine Expression at a Tumor Site to 

Enhance Immune Cell Trafficking for Melanoma 


Uma Thanarajasingam, Laura Sanz, Rosa Maria Diaz, Jill 

Thompson, Michael Gough, Richard Vile 



Room: 123/127 


The Effects of Intratumoral Injection of a Replicating 

Morbillivirus in a Canine Model of Naturally Occurring 

Lymphoma 

Michael S. Henson, Steven E. Suter, Veronika A. von Messling, 

Roberto Cattaneo, Adele K. Fielding 


HSVtk Mediated Immunostimulatory Tumor Cell Killing 

Enhances Adoptive T Cell Therapy in a B16 Melanoma 

Model 

Luis Sanchez-Perez, Michael Gough, Jian Qiao, Uma 

Thanarajasingam, Atique Ahmed, Rosa Maria Diaz, Richard 

Vile 


Effective Suicide Gene Therapy of Leukemia in a Novel 

Model of Retroviral Insertion 

Martina Blumenthal, Dianne Skelton, Denise A. Carbonaro, 

Karen A. Pepper, Donald B. Kohn 


Phase I/II Trial of Intravenous NDV-HUJ (OV001) 

Oncolytic Virus in Recurrent Glioblastoma Multiforme 

Arnold I. Freeman, Zichria Zakay-Rones, John M. Gomori, 

Eduard Linetsky, Linda Rasooly, Amos Panet, Eugene Libson, 

Charles S. Irving, Tali Siegal, Eithan Galun 


Addition of HU to GCV Induces Bystander Cytotoxicity 

in Co-Cultures of HSVTK-Expressing and Non- 

Expressing HeLa Cells 

Brian G. Gentry, Paul D. Boucher, Donna S. Shewach


ABSTRACTS 



Monitoring of Transgene Expression in Cancer Patients 

with Positron Emission Tomography (PET) 

Bruno Sangro, Ivan Penuelas, Guillermo Mazzolini, Jose F. 

Boan, Josep M. Marti-Climent, Maria Ruiz, Cheng Qian, 

Nagichettiar Satyamurthy, Jorge R. Barrio, Michael E. Phelps 

Jose A. Richter, Sam Gambhir, Jesus Prieto 


mda-7 Kills Pancreatic Cancer Cells by Inhibition of Wnt/ 

PI3K Signaling: Identification of IL-20 Receptor- 

Mediated Bystander Activity Against Pancreatic Cancer 

Sunil Chada, Dora Bocangel, Ramesh Rajagopal, Elizabeth 

Grimm, Abner M. Mhashilkar, Mingzhong Zheng 

810 12:00 PM 

Ex-Vivo Activation and Genetic Manipulation of Human 

T Cells Results in Consistent In-Vivo Expansion and 

Lethal GvHD in a Novel Murine Xenotransplant Model 

Bruno Nervi, Michael P. Rettig, Julie K. Ritchey, Gerhard 

Bauer, Jon Walker, Phillip E. Herrbrich, Todd E. Meyerrose, 

Mark Bonyhadi, Jan A. Nolta, John F. DiPersio 



Room: 261/262 


ACE-Targeted eNOS and BMPR2 Gene Therapy 

Attenuates Pulmonary Hypertension in a Chronic 

Hypoxia Rat Model 

Ann M. Reynolds, Mark D. Holmes, David T. Curiel, Andrew 

H. Baker, Paul N. Reynolds 


Non-Human Primate Serotype rh.10 AAV Directed 

Therapeutic Serum Levels of að1-antitrypsin Following 

Intrapleural Administration in Mice with Pre-Existing 

Immunity to AAV 

Bishnu P. De, Adriana Heguy, Neil R. Hackett, John Lee, 

Guangping Gao, James M. Wilson, Ronald G. Crystal 


Distribution of Transgene Expressing Cells Following 

Intramarrow Injection to Transduce Hematopoietic Stem 

Cells In Situ Using rSV40 Vectors 

Jean-Pierre Louboutin, Bianling Liu, David S. Strayer 


Adenovirus-Mediated Gene Transfer Demonstrates That 

Pirin, a Transcription Factor Up-Regulated in the 

Bronchial Epithelium by Cigarette Smoke, Mediates 

Bronchial Epithelial Cell Apoptosis 

Brian D. Gelbman, Adriana Heguy, Timothy P. O’Connor, 

Joseph Zabner, Ronald G. Crystal 


Aerosol Delivery of Helper-Dependent Adenoviral 

Vector into Nonhuman Primate Lungs Results in High 

Efficiency Pulmonary Transduction with Minimal 

Toxicity 

Peter Hiatt, Nicola Brunetti-Pierri, David Koehler, Ruth 

McConnell, Julie Katkin, Donna Palmer, David Dimmock, Jim 

Hu, Milton Finegold, Arthur Beaudet Dee Carey, Karen Rice, 

Philip Ng 


RNAi Applications for Respiratory Epithelia: Delivery 

and Efficacy 

Anthony J. Fischer, Christine L. Wohlford-Lenane, Patrick L. 

Sinn, Hong Peng Jia, Steven M. Varga, Beverly L. Davidson, 

Paul B. McCray 


Protein Transduction Domain Mediated DFRD Correction 

of CFTR Function in Airway Cells from DF508 

Homozygous CF Patients 

Zhibao Mi, Fei Sun, Xiaoli Lu, Steve Condliffe, Xiaoyan 

Gong, Joseph Pilewski, Raymond A. Frizzell, Paul D. Robbins 

818 12:00 PM 

Recombinant AAV1-Mediated Treatment of CNS 

Glycogen Accumulation in Mouse Model of Glycogen 

Storage Disease Type II 

Lara R. DeRuisseau, Cathryn S. Mah, Thomas J. Fraites, Jr., 

David D. Fuller, Barry J. Byrne 

Presidential Symposium 430 


819 3:20 PM 

Molecular Follow-Up of Patients Treated with Allogeneic 

Hematopoietic Stem Cell Transplantation and Donor 

Lymphocytes Transduced with a Retroviral Vector 

Expressing HSV-TK and ∆LNGFR 

Alessandra Recchia, Zulma Magnani, Fabrizia Urbinati, 

Daniela Sartori, Sara Muraro, Maria T. Lupo Stanghellini, 

Massimo Bernardi, Alessandra Pescarollo, Marco Bregni, Chiara 

Bonini Claudio Bordignon, Fabio Ciceri, Fulvio Mavilio 

820 3:38 PM 

Highly Efficient Exon-Skipping and Sustained Correction 

of Muscular Dystrophy Using an Adeno-Associated 

Viral Vector 

Aurelie Goyenvalle, Adeline Vulin, Jean Claude Kaplan, France 

Leturcq, Olivier Danos, Luis Garcia 

821 3:56 PM 

Development of a Site-Specific Integrating Gene-Deleted 

Adenoviral Vector for Sustained Transgene Expression 

Levels In Vivo 

Anja Ehrhardt, Stephen R. Yant, Giering A. Jeff, Hui Xu, 

Michele P. Calos, Mark A. Kay

96 


ABSTRACTS 


Poster Session III 


RNA Virus Vectors: Safety, Therapeutics 


Session III to Poster Session I and will follow Abstract 

109.) 

822 

A Rev/RRE Dependent Packaging System for MLV Based 

Vectors Raises Biosafety Concerns 

Hong Ma, Adam Cockrell, Ryan Bash, Teryy VanDyke, Tal 

Kafri 

823 

Dissecting Overlapping Functions of Transcriptional 

Enhancer and Polyadenylation in the Lentiviral U3 

Qing Yang, Wayne Chou, Lung-Ji Chang 

824 

Oncoretroviral Vector Integration Biases in Mouse 

Hematopoietic Progenitors 

Mari Aker, Julie Tubb, George Stamatoyannopoulos, David W. 

Emery 

825 

Product Enhanced Reverse Transcriptase (PERT) Assay 

for RCL/RCR Detection 

Lakshmi Sastry, Yi Xu, Lisa Duffy, Sue Koop, Aparna Jasti, 

Holger Roehl, Doug Jolly, Kenneth Cornetta 

826 

The Risk of Integration: Characterization and Testing of 

Cell Lines Engineered for the Study of Insertional Gene 

Activation 

R. Scott McIvor, Nikunj V. Somia, Andrea D. Converse 

827 

Integration and Biodistribution Studies of Retroviral 

Vector in Mouse Gonad Tissues 

Gyu Seek Rhee, Ji hyun Seok, Soon Sun Kim, Seung Jun 

Kwack, Bo Ra Kim, Rhee Da Lee, Soo Yeong Chae, Young 

Hyuk Won, Seung Shin Yu, Dae Hyun Cho 

828 

Optimizing the Woodchuck Hepatitis Virus Post- 

Transcriptional Regulatory Element (WPRE) for Safety 

and Function in Lentiviral Vectors 

Manij Patel, John C. Olsen 

829 

Design of a Safe and Efficient Vector for Bone Marrow 

Chemoprotection by O6-Methyl-Guanine-DNA- MethylTransferase 

Axel Schambach, Jens Bohne, Saurabh Chandra, Elke Will, 

Lars Mueller, Geoffrey Margison, David A. Williams, Christopher 

Baum 

830 

Determination of the Profile and Site Preference of 

Lentiviral Vector Integration 

Caroline V. Hacker, Susan M. Kingsman, Kyriacous A. 

Mitrophanous, James E. Miskin 

831 

Evaluation of Lentiviral Vectors for Gene Therapy of 

Wiskott-Aldrich Syndrome 

Marguerite V. Evans-Galea, Matthew M. Wielgosz, Hideki 

Hanawa, Arthur W. Nienhuis 

832 

Suppression of HIV-1 Replication by Delievery of 

multiple siRNA Genes Against p24, Tat/ Rev and CCR5 

Hsin-Lung Lo, Jerry Wu, Tan Chang, Priscilla Yam, John 

Zaia, Jiing-Kuan Yee 

833 

A Preclinical Safety Study of Simian Immunodeficiency 

Virus (SIV)-Based Lentivirus Vector for Retinal Gene 

Transfer in Non-Human Primates 

Yasuhiro Ikeda, Yoshikazu Yonemitsu, Masanori Miyazaki, 

Riichiro Kono, Toshinori Murata, Yoshinobu Goto, Toshiaki 

Tabata, Yasuji Ueda, Naohide Ageyama, Keiji Terao Mamoru 

Hasegawa, Tatsuro Ishibashi, Katsuo Sueishi 

834 

Highly Efficient and Tumor-Selective Gene Delivery in 

Metastatic Colon Cancer Models Using a Replication- 

Competent Retrovirus Vector 

Kei Hiraoka, Takahiro Kimura, Christopher Logg, Satoshi 

Kondo, Noriyuki Kasahara 

835 

Gene Therapy for Arthritis: Long-Term Expression in a 

Sub-Population of Synovial Fibroblasts 

Elvire Gouze, Jean-Noel Gouze, Glyn D. Palmer, Carmencita 

Pilapil, Christopher H. Evans, Steven C. Ghivizzani 

836 

Suitability of Anti-Retroviral Genes for Gene Therapy of 

AIDS 

Matthias Schweizer, Sabine Fengler, Silke Schuele, Sylvia 

Raupp, Carsten Muenk, Dorothee M. von Laer, Klaus Cichutek 

837 

Prolonged Gene Expression in Murine Salivary Glands 

Following FIV-Based Lentiviral Vector Transduction 

Ela Shai, Reba Condiotti, Michael A. Curran, Garry P. Nolan, 

Eithan Galun 

838 

Construction and Use of Retroviral Vectors Encoding the 

Toxic Gene Barnase 

Sumit Agarwal, Bryan Nikolai, Tomoyuki Yamaguchi, Nikunj 

V. Somia


ABSTRACTS 


839 

Lentiviral Vectors Encoding Connexin43 Mutants 

Reduce Gap Junction Function: Toward Molecular 

Ablation of Re-Entrant Cardiac Arrhythmias 

Eddy Kizana, Samantha L. Ginn, Christine Smyth, Anita 

Boyd, Stuart P. Thomas, David G. Allen, David L. Ross, Ian 

E. Alexander 

840 

Recombinant Sendai Virus Vectors with Matrix Protein 

Gene Deletion Restore Dendritic Cell Allostimulatory 

Activity 

Sorin Armeanu, Irina Smirnow, Sascha Bossow, Wolfgang J. 

Neubert, Ulrich M. Lauer, Peter Brossart, Michael Bitzer 

841 

A Cytoplasmic Gene Therapy with Sendai Virus Vectors: 

Reduction of Cytopathic and Immunogenic Reaction of 

New Class Cytoplasmic RNA Vector 

Makoto Inoue, Hitoshi Iwasaki, Mariko Yoshizaki, Akihiro 

Tagawa, Kentaro Washizawa, Takashi Hironaka, Mamoru 

Hasegawa 

842 

Repeated Administration of Sendai Virus into Lung 

Uta Griesenbach, Gerry McLachlan, Luci Somerton, David 

Collie, Makoto Inoue, Takashi Hironaka, Toshiyuki Owaki, 

Duncan M. Geddes, Mamoru Hasegawa, Eric W. F. W. Alton 

843 

The Tupaia Paramyxovirus Envelope as Vector Delivery 

Module: Novel Fusion Protein Cleavage-Activation 

Sequence and Attachment Protein Retargeting 

Christoph Springfeld, Veronika von Messling, Christian A. 

Tidona, Gholamreza Darai, Roberto Cattaneo 

844 

Determinants of Viral Tropism: A Morbillivirus V Protein 

Exerts Host Defense Evasion Function in T Lymphocytes 

Veronika von Messling, Roberto Cattaneo 

845 

Properties of Sindbis Virus Vectors Produced with a 

Chimeric Split Helper System 

Anna Ketola, Sondra Schlesinger, Jarmo Wahlfors 

846 

In Vivo and In Vitro Cell Targeting with Library-Selected 

Protease-Activatable Replication-Competent 

Retroviruses 

Klaus Cichutek, Richard M. Schneider, Astrid Schwantes, 

Yuansheng Sun, Christian J. Buchholz, Irene Hartl, Stephen J. 

Russell 


847 

Adeno-Associated Virus Capsids from Divergent AAV 

Clades and Subgroup C Adenovirus Capsids Share a 

Common Microtubule-Binding Mechanism Relevant to 

Virus Trafficking 

Samir A. Kelkar, Bishnu P. De, Guangping Gao, James M. 

Wilson, Ronald G. Crystal, Philip L. Leopold 

848 

Development of AAV Vectors Targeted to Vascular 

Endothelium 

Matthew Stachler, Jennette Crumine, Jeffrey Bartlett 

849 

Transduction of Mouse Dendritic Cells Is Enhanced by 

Repetitive Infection with Self-Complementary Adeno- 

Associated Virus 6 Combined with Immunostimulatory 

Ligands 

Wayne A. Aldrich, Changchun Ren, April F. White, Shang- 

Zhen Zhou, Sanjay Kumar, Connie B. Jenkins, Denise R. 

Shaw, Theresa V. Strong, Pierre L. Triozzi, Selvarangan 

Ponnazhagan 

850 

Efficiency of Six AAV Serotypes in Transducing Rat 

Myocardium In Vivo 

Julieta Palomeque, Jennifer A. Wellman, Shangzhen Zhou, Peter 

Colosi, Manuel Ramos-Kuri, Federica del Monte, Roger J. 

Hajjar 

851 

Development of Organ Homing AAV-2 Vectors 

Lorraine M. Work, Stuart A. Nicklin, Nicola Britton, Michael 

Hallek, Hildegard Buning, Andrew H. Baker 

852 

Human ANP32B Protein Binds AAV-2 Rep and Increases 

Viral Replication and rAAV Production 

Gianluca Pegoraro, Alessandro Marcello, Michael P. Myers, 

Mauro Giacca 

853 

Persistent Expression of Single Chain Antibodies Mediated 

by AAV5 and AAVrh.10 Vectors 

Julie L. Boyer, Jeffrey Howard, Neil R. Hackett, Robert G. 

Pergolizzi, James M. Wilson, Ronald G. Crystal 

854 

Optimization of AAV Serotype and Promoter for Increased 

Distribution of Expression in Both Somatic and 

Central Nervous Systems of Mice 

Haiyan Fu, Joseph Muenzer, Lu Kang, Jerillyn S. Jennings, 

Richard J. Samulski, Douglas M. McCarty

98 


ABSTRACTS 


855 

In Vivo Spinal Cord Uptake and Neuronal Binding 

Properties of Tet1: A Potential Means for Enhanced 

Spinal Cord AAV Delivery 

Thais Federici, James K. Liu, Qingshan Teng, Mary Garrity- 

Moses, Jun Yang, Nicholas M. Boulis 

856 

Transduction Patterns, Enzyme Distribution, and Vector 

and Enzyme Transport Resulting from Unilateral Injections 

of AAV 7, 8, 9, or Rh10 Expressing β-Glucuronidase 

into Select Brain Regions of the C3H Mouse 

Cassia N. Cearley, John H. Wolfe 

857 

A Versatile and Efficient Viral Vector Platform for Stable 

In Vivo Expression of Short Hairpin RNA 

Dirk Grimm, Kusum Pandey, Konrad L. Streetz, Mark A. Kay 

858 

Adeno-Associated Virus Vector Serotype 8 (AAV8) 

Achieves a Higher Level of Gene Transfer to the Central 

Nervous System (CNS) upon Intraventricular Injection 

in Neonatal Mice Compared to AAV1 and AAV2 

Marike Broekman, Fabricio Costa, Comer Laryssa, LeRoy Stan, 

Sena Esteves Miquel 

859 

Enhancing Neurotropism with Multiple Mutations in the 

AAV-2 Capsid 

Jianfeng Xu, Caroline Bass, Chunyan Ma, Ernest F. Terwilliger 

860 

Tissue Distribution of Expression Using AAV8-Based 

Vectors after Intramuscular Injection and Other Routes of 

Delivery 

Tsuyoshi Ogura, Hiroaki Mizukami, YuanYuan Zhang, 

Takashi Okada, Akihiro Kume, Seiji Madoiwa, Jun Mimuro, 

Yoichi Sakata, Keiya Ozawa 

861 

AAV Gene Delivery for the Cornea 

Jia Liu, Sonal S. Tuli, David C. Bloom, Gregory S. Schultz, 

William W. Hauswirth, Alfred S. Lewin 

862 

Adipose Tissue Transduction Using AAV8-Based Vectors: 

Inadvertent Gene Transfer into Liver 

YuanYuan Zhang, Tsuyoshi Ogura, Jun Mimuro, Takashi 

Okada, Akihiro Kume, Yoichi Sakata, Keiya Ozawa, Hiroaki 

Mizukami 

863 

Directed Evolution of Adeno-Associated Virus (AAV) 

Vectors for Human Gene Therapy 

Luca Perabo, Jan Endell, Susan King, Daniela Goldnau, 

Kerstin Lux, Michael Hallek, Hildegard Buening 

864 

Adsorption of Adeno-Associated Virus to Commonly 

Used Catheter Materials 

Daisy P. Cross, Erica TenBroek, Cygni (Howie) Chan, Laura 

Christoferson, Orhan Soykan, Daniel C. Sigg 

865 

Focus on Specificity: Creating Efficient CML-Specific 

Recombinant Adeno-Associated Viral (AAV) Vectors 

Using a Random Peptide Library 

Marius Stiefelhagen, Marlon R. Veldwijk, Stephanie Laufs, 

Jurgen A. Kleinschmidt, Frederik Wenz, W. Jens Zeller, Stefan 

Fruehauf 

866 

Recombinant AAV 1, 2, 5, 8 and Chimeric 1/2-1/8 

Serotypes Display Differential Transduction Efficiency 

after Delivery to Mouse Brain 

Fabricio F. Costa, Marike Broekman, Stanley G. LeRoy, 

Laryssa A. Comer, Miguel Sena-Esteves 

867 

Use of AAV Display Libraries to Generate Myotropic 

Vectors 

Yu-Hung Kuo, Justin Fraser, Michael G. Kaplitt 

868 

Effective Purification of Adeno-Associated Virus Vectors 

by Disposable Ion Exchange Membranes 

Takashi Okada, Mutsuko Nonaka-Sarukawa, Takayuki Ito, 

Takashi Matsushita, Hiroaki Mizukami, Akihiro Kume, Keiya 

Ozawa 

869 

Infectious Titer Assay for Recombinant Adeno-Associated 

Virus Vectors Using Direct Cell Lysis and Endpoint 

Taqman PCR 

Simon M. Grainger, Richard Peluso, Christopher J. Gerard 

870 

Transfection of Mammalian Cells Using Linear 

Polyethylenimine Is a Simple and Effective Means of 

Producing Recombinant AAV 

Sharon E. Reed, Elizabeth M. Staley, David J. Pintel, Gregory 

E. Tullis 

871 

rAAV2-Mediated Gene Expression in Primary Cortical 

Neural Cells Following Inhibition of DNA Synthesis 

and/or EGFR Tyrosine Kinase 

Young Ran Nam, Sung Jin Kim, Chul-Hyen Joo, Yoo Kyum 

Kim, Heuiran Lee 

Adenovirus Vectors: Vector Technologies 

872 

Chimpanzee Derived Adenoviral Vectors Transduce and 

Activate Human Dendritic Cells 

Sury Somanathan, Soumitra Roy, Guangping Gao, Arbans 

Sandhu, James M. Wilson


ABSTRACTS 


873 

Adenovirus Polypeptide IIIa as a Novel Locale for 

Incorporation of Heterologous Peptides 

Joel N. Glasgow, Elena Kashentseva, Igor P. Dmitriev, David T. 

Curiel 

874 

Long-Term Transgene Expression In Vivo from a Helper 

Dependent Adenovirus—Epstein-Barr Virus Hybrid 

Vector 

Sean D. Gallaher, Jose S. Gil, Oliver Dorigo, Arnold J. Berk 

875 

Adenoviral Vector Targeting – New Targets for Old 

Capsids 

Douglas E. Brough, Hinrich Staecker, Masaki Akiyama, Chi 

Hsu, Selva R. Murugesan, David A. Einfeld, Jason G. Gall, 

Lisa L. Wei, Melissa Hamilton, Duncan McVey C. Richter 

King 

876 

Development of a Novel Adenovirus-Alphavirus Hybrid 

Vector Providing High Efficacy and Safety in the Treatment 

of Liver Cancer 

Min Guan, Cristian Smerdou, Juan R. Rodriguez-Madoz, M. 

Gabriela Kramer, Stefan Kochanek, Jesus Prieto, Cheng Qian 

877 

Long-Term Transgene Expression with a Second Generation 

Hybrid AdenoRetroviral Vector 

Changyu Zheng, Weitan Zhang, Joseph M. Vitolo, Bruce J. 

Baum 

878 

Toward ‘Pseudotyping’ Adenovirus Vectors: Efficient 

Generation of pIX-Expressing Helper Cell Lines 

Rob C. Hoeben, Taco G. Uil, Jeroen De Vrij, Leif Lindholm, 

Jort Vellinga 

879 

Transduction Enhancement by the Adenovirus/Polymer 

Complexes Derivatized with Protein Transduction 

Domain Peptides 

Ilia Fishbein, Ivan S. Alferiev, Richard Gaster, Michael Chorny, 

Origene Nyanguile, Robert J. Levy 

880 

AAV-Rep 78 Mediated Rescue of a 27kb Transgene 

Cassette from a Helper-Dependent Ad Vector Genome 

Hongjie Wang, Dmitry Shayakhmetov, Andre Lieber 

881 

Development of Novel E1-Complementary Cells for 

Adenoviral Production Free of Replication Competent 

Adenovirus 

Deborah Farson, Lucy Tao, Derek Ko, Thomas Harding, 

Dominic Brignetti, De-Chao Yu, Yuanhao Li 

882 

Development of a Transposon-Based Approach to 

Identification of Novel Transgene Insertion Sites within 

Replicating Viral Genomes 

Peter Kretschmer, Fang Jin, Cecile Chartier, Paul Harden, Terry 

Hermiston 

883 

Gold Nanoparticles as an Amplifying Payload Strategy for 

Adenoviral Cancer Gene Therapy 

Maaike Everts, Jennifer L. Leddon, Robbert J. Kok, Meredith A. 

Preuss, C. Leigh Millican, David E. Nikles, Duane T. Johnson, 

David T. Curiel 

884 

Integration of a Helper-Dependent Adenovirus Mediated 

by the Phage FðC31 Integrase 

Rénald Gilbert, Laurie Desfossé, Émilie Mairey, Benoît 

Raymond, Michele P. Calos, Jacques P. Tremblay, Bernard 

Massie 

885 

Calcium Phosphate Coprecipitation Enhances Adenoviral 

Vector-Based Gene Transfer to Murine Bone Marrow 

Derived Dendritic Cells 

Michael P. Seiler, Vincenzo Cerullo, Philip Ng, Donna Palmer, 

Brendan Lee 

886 

An Adenovirus Vector with a Chimeric Fiber Derived 

from Ovine Adenovirus for Novel Tropism Discovery 

Masaharu Nakayama, Joel N. Glasgow, Gerald W. Both, 

Massimo Palmarini, Yuko Tsuruta, Kenzaburo Tani, Joanne T. 

Douglas, David T. Curiel 

887 

Reducing the Incidence of Replication Competent 

Adenovector by Engineering the Vector Genome 

G. Campbell Kaynor, Jennifer L. Brown, Rhonda Brown, 

Xinzhong Wang 

888 

Optimization of Adenovirus Serotype 35 Vectors for 

Efficient Transduction in Human Hematopoietic Progenitors; 

Comparison of Promoter Activities 

Fuminori Sakurai, Hiroyuki Mizuguchi, Kenji Kawabata, 

Teruhide Yamaguchi, Takao Hayakawa 

889 

Lanthanum Enhances Adenovirus-Mediated Gene 

Transfer 

Oliver B. Betz, Glyn D. Palmer, Volker M. Betz, Keith 

Crawford, Christopher Niyibizi, Mark S. Vrahas, Christopher 

H. Evans

100 


ABSTRACTS 


890 

Development of a Single Vector System for Gene 

Delivery Via Hybrid Helper Dependent Adenovirus - 

Epstein-Barr Virus Vector for Long Term Persistance 

Jose S. Gil, Sean D. Gallaher, Oliver Dorigo, Arnold J. Berk 

891 

A Flexible System for Ad35-Based Vector Generation 

Wentao Gao, Angela Montecalvo, Huijie Sun, Paul D. Robbins, 

Andrea Gambotto 

892 

GeneJammer® Enhances Cellular Transduction with 

Recombinant Adenovirus 

Christine M. Dilling, Pablo Bosh, Alan R. Davis, Jessica A. 

Shafer, Steven Stice, Malavosklish Bikram, Jennifer L. West, 

Elizabeth A. Olmsted-Davis 

893 

Using QPCR and Automation to Assign Infectious 

Potencies to Adenovirus Based Vaccines and Vectors for 


Alan C. Puddy, Jenny Xu, Amy M. Bowman, Yuhua Zhang, 

Charles Y. Tan, Timothy L. Schofield, Peter A. DePhillips, John 

A. Lewis, Jayanthi J. Wolf 

Inborn Errors of Metabolism: Liver and Pancreatic 

Diseases 

894 

A Novel Mechanism between Type II Diabetes Mellitus 

and Procalcitonin Gene Expression 

Mehment Ali Soylemez, Oktay Seyment, Gunnur Yigit 

895 

Ex Vivo Gene Therapy of Metabolic Liver Disorders 

Using Uncultured and Lentivirally Transduced Hepatocytes 

Tuan Huy Nguyen, Jacques Birraux, Barbara Wildhaber, 

Catherine Fux, Francois Trivin, Anne Myara, Claude Lecoultre, 

Didier Trono 

896 

Complete and Permanent Correction of Hyperbilirubinemia 

in Newborn Gunn Rats by Lentiviral-Mediated 


Tuan huy Nguyen, Marta Bellodi-Privato, Dominique Aubert, 

Anne Myara, Didier Trono, Nicolas Ferry 

897 

Recombinant AAV2/8 Vector-Mediated Gene Therapy 

Corrects Hyperphenylalaninemia in Murine PKU 

Cary O. Harding, Melanie B. Gillingham, Kelly Hamman, 

Andrew Bird, Dwight Koeberl 

898 

Liver-Directed, AAV- and Lentivirus-Mediated Gene 

Therapy in the Phenylketonuria Mouse Model Pah-enu2 

Zhaobing Ding, Panco Georgiev, Beat Thony 

899 

Glucose-Stimulated Insulin Secretion from an Engineered 

Human Liver Cell Line Is Regulated by ATP- 

Sensitive Potassium Channels and the L-Type Calcium 

Channel 

Ann M. Simpson, Edwin Ch’ng, Guo J. Liu, Chang Tao, 

Donald K. Martin, Mark Lutherborrow, Bernard E. Tuch 

900 

Overcoming Gender-Specific Barrier of AAV-Mediated 

Gene Therapy by Neonatal Intervention 

Akihiro Kume, Tsuyoshi Ogura, Hiroaki Mizukami, Shigeo 

Kure, Takashi Okada, Masashi Urabe, Takashi Matsushita, 

Keiya Ozawa 

901 

Recovery from Type 1 Diabetes by Insulin and Glucokinase 

Expression in Skeletal Muscle 

Joel Montane, Alex Mas, Xavier M. Anguela, Anne M. Douar, 

Pedro J. Otaegui, Fatima Bosch 

902 

In Toto Gene Correction by Meganuclease-Induced 

Homologous Recombination in Mouse Liver 

Agnes Gouble, Valerie Guyot, Jean-Pierre Cabaniols, Sophie 

Leduc, Sandra Rolland, Frederic Paques, Philippe Duchateau 

903 

Ornithine Transacarbamylase Deficiency Correction with 

Low Dose of Helper-Dependent Adenoviral Vector 

Nicola Brunetti-Pierri, Christian Clarke, Viraj Mane, Donna 

Palmer, Philip Ng, William O’Brien, Brendan Lee 

904 

High Efficiency Hepatic Transduction and Long-Term 

Transgene Expression by Delivering Helper-Dependent 

Adenoviral Vectors into the Surgically Isolated Liver of 

Nonhuman Primates 

Nicola Brunetti-Pierri, Thomas Ng, David Iannitti, William 

Cioffi, Donna Palmer, Arthur Beaudet, Milton Finegold, Dee 

Carey, Philip Ng 

905 

Hydrodynamic Injection of Helper-Dependent Adenoviral 

Vectors Increases Liver Transduction Efficiency and 

Decreases Acute Inflammatory Response 

Nicola Brunetti-Pierri, Viraj Mane, Donna Palmer, Arthur 

Beaudet, Milton Finegold, Philip Ng 

906 

High Efficiency Hepatic Transduction with Minimal 

Toxicity Following “Hydrodynamic” Injection of HDAd 

into Nonhuman Primates 

Nicola Brunetti-Pierri, Charles Mullins, Donna Palmer, Milton 

Finegold, Arthur Beaudet, Karen Rice, Dee Carey, Philip Ng


ABSTRACTS 


907 

Gene Therapy for Genetic Lipoprotein Lipase (LPL) 

Deficiency; an Update 

Jaap Rip, Melchior C. Nierman, Jeroen A. Sierts, Colin J. Ross, 

Michael R. Hayden, Albertine de Jong, Paul Dijkhuizen Wim 

Hermens, Jaap Twisk, John J. Kastelein, Jan Albert 

Kuivenhoven, Janneke M. Meulenberg 

908 

Evaluation of the Sleeping Beauty Transposon System in 

OTC Deficient Spf-Ash Mice: A Non-Viral Approach to 

a Cell Autonomous Metabolic Disease 

Andy Wilber, Joel Frandsen, Hiroki Morizono, Mendel 

Tuchman, R. Scott McIvor 

909 

Sustained Correction of Glycogen Storage Disease Type 

II by rAAV1 Vector-Mediated Gene Therapy 

Kerry O. Cresawn, Cathryn S. Mah, Thomas J. Fraites, Jr., 

Melissa A. Lewis, Irene Zolotukhin, Barry J. Byrne 

910 

AAV8-Mediated Expression of Glucocerebrosidase 

Ameliorates the Storage Pathology in the D409V/null 

Gaucher Mouse 

Kerry McEachern, John Marshall, Jennifer B. Nietupski, Karen 

M. Brennan, Christine M. Barbon, Wei-Lien Chuang, William 

Kennedy, Gregory A. Grabowski, Seng H. Cheng 

911 

Comparison of rAAV Serotype, Promoter, and Treatment 

Age for the Correction of Glycogen Storage Disease Type 

II 

Kerry O. Cresawn, Cathryn S. Mah, Irene Zolotukhin, Melissa 

A. Lewis, Barry J. Byrne 

912 

Long-Term Efficacy of AAV Vector-Mediated Gene 

Therapy in the Mouse Model for Glycogen Storage 

Disease Type la (GSD-Ia) 

Dwight D. Koeberl, Baodong Sun, Tirupapuliyur Damodaran, 

Talmage Brown, Andrew Bird, Ayn Schneider, David S. 

Millington, Y.-T. Chen 

913 

Development and Characterization of CF Airway 

Epithelial Cell Lines Complemented with Wild-Type and 

∆F508 CFTR cDNA 

Rosalie Maurisse, Beate Illek, Judy Cheung, Tusyoshi Shuto, 

Hirofumi Kai, Horst Fischer, Dieter C. Gruenert 

914 

A Dichotomous Role for Interleukin-10 in the Pathogenesis 

of Type 1 Diabetes 

Greg G. Simon, Fletcher Schwartz, Clive Wasserfall, Mark A. 

Atkinson 

915 

Adenovirus Transduction May Be Involved in the 

Transdifferentiation of the Liver to the Pancreas 

Alfred Y. Wang, Anja Ehrhardt, Hui Xu, Mark A. Kay 

Cardiovascular: Myocardial Gene Therapy 

916 

Sustained Whole-Body Functional Rescue by Systemic 

Delivery of AAV8 Vectors in Heart Failure and Muscular 

Dystrophy Hamsters 

Tong Zhu, Liqiao Zhou, Satsuki Mori, Zhong Wang, Charles 

Mctiernan, Chunping Qiao, Chunlian Chen, Daowen Wang, 

Juan Li, Xiao Xiao 

917 

Engineered Cell Therapy for Sustained Local Myocardial 

Delivery of Non-Secreted Proteins 

Jing Bian, Niladri Mal, Matt Kiedrowski, Farhad Forudi, Marc 

S. Penn 

918 

Nanoparticle Mediated Gene Delivery to Magnetized 

Implants 

Benjamin B. Yellen, Michael Chorny, Ilia Fishbein, Darryl N. 

Williams, Candice M. Klingerman, Ivan S. Alferiev, Origene 

Nyanguile, Gary Friedman, Robert J. Levy 

919 

Magnetic Nanoparticle Mediated Gene and Cell Delivery 

Darryl N. Williams, Michael Chorny, Benjamin B. Yellen, Ilia 

Fishbein, Stanley J. Stachelek, Origene Nyanguile, Gary 

Friedman 

920 

Muscle Stem Cells Provide Superior Infarct Repair When 

Compared with Myoblasts 

Hideki Oshima, Thomas R. Payne, Kenneth L. Urish, Tetsuro 

Sakai, Yiqun Ling, Burhan Gharaibeh, Kimimasa Tobita, 

Bradley B. Keller, James H. Cummins, Johnny Huard 


Session III to Poster Session II and will follow Abstract 

639.) 

921 

Steps toward Cardiomyopathy Gene Therapy: In Vivo 

Expression of Human VLCAD 

J. Lawrence Merritt II, Jerry Vockley, Dietrich Matern, Miao He, 

David B. Schowalter 

922 

Paracrine Alterations of Endothelial Cells Adhesion 

Complexes; Implications for Stem Cell Homing 

Ion S. Jovin, Jinette D. Abbott, Thomas Manes, Zhenrong Hao, 

Lei Li, Reed P. Hickey, Yan Huang, Dingang Liu, Martin S. 

Kluger, Frank J. Giordano

102 


ABSTRACTS 


923 

Muscle Stem Cells Genetically Modified to Express a 

VEGF Antagonist Display an Impaired Ability for Cardiac 

Repair 

Thomas R. Payne, Hideki Oshima, Masaho Okada, Nobuo 

Momoi, Kimimasa Tobita, Bradley B. Keller, Hairong Peng, 

Johnny Huard 

924 

High Transgene Expression Efficiency Does Not Directly 

Reflect High Plasmid DNA Uptake Efficiency: Comparison 

between Skeletal and Cardiac Muscle 

N. Sarkar, T. Gustavsson, A. Freyschuss, B. Norman, A. Wiik, 

P. Blomberg, V. Drvota, C. Sylven 

925 

pVGI.1 (VEGF2) Biodistribution in Swine Following 

Injection into Anterior Wall of the Left Ventricle Using 

the Stiletto Endocardial Direct Injection Catheter 

System 

Qing Wang, Yawen L. Chiang 

926 

Recombinant AAV Vectors Pseudotyped with Serotype-1 

Capsid Mediates Early Onset of Gene Expression in 

Mouse Hearts 

Hua Su, Yu Huang, Alicia Barcena, Janice Arakawa-Hoyt, 

Jianqin Ye, William Grossman, Yuet W. Kan 

927 

Human Mesenchymal and Hematopoietic Stem Cell 

Contribution to Cardiac Tissue Regeneration after Injury 

Ivana Rosova, Jan A. Nolta 

928 

Nonclinical Safety Study of Direct Myocardial Injection 

of pVGI.1 (VEGF2) Using the Stiletto Endocardial 

Direct Injection Catheter System in a Swine Model 

Qing Wang, Robert Rapoza, Wendy Naimark, Hsing-Yi Jan, 

Yawen L. Chiang 

929 

CD9 Gene Therapy Inhibits Cardiac Hypertrophy and 

Tachycardia, and Attenuates the Remodeling after 

Myocardial Infarction in Mice 

Hiroaki Ushikoshi, Tomoyuki Takahashi, Genzou Takemura, 

Yuwen Li, Masayasu Esaki, Ngin Cin Khai, Takao Kawai, 

Shinya Minatoguchi, Takako Fujiwara, Hisayoshi Fujiwara 

Ken-ichiro Kosai 

930 

Magnetically Responsive Polylactide-Based Nanoparticles 

Potently Enhance Non-Viral Gene Transfer 

Boris Polyak, Michael Chorny, Ivan Alferiev, Robert J. Levy 

931 

Refractory Period Prolongation by Fibroblasts 

Overexpressing the Mutant Kv1.3 H401W Channel in 

Computer Simulation and Pig Hearts 

Ariel Shabtay, Tal Bresler, Lior Yankelson, Lior Gepstein, 

Shimon Marom, Yair Feld 

932 

Gene Therapy for Diabetes Mellitus in Rats by Intramuscular 

Injection of Lentivirus Containing Insulin Gene 

Taekeun Oh, Mija Lee, Seungtaik Kim 

933 

Closed-Chest Cell Injections into Mouse Myocardium 

Guided by High-Resolution Echocardiography 

Matthew L. Springer, Richard E. Sievers, Mohan N. 

Viswanathan, Michael S. Yee, Elyse Foster, William Grossman, 

Yerem Yeghiazarians 

934 

Initial Results of the Treatment of a Cardiomyopathy in 

Myopathic Hamsters by IGF-1 Based on Plasmid DNA 


Armelle Serose, André Salmon, Marc Y. Fiszman, Yves Y. 

Fromes 

935 

Restoration of Impaired Myocardial Contractility by 

Overexpressing Sarcoplasmic Reticulum Calcium ATPase 

through Recombinant Adeno-Associated 

Xiaochun Lu, Haipeng Hui, Xiaoying Li, Xiaobing Wu 

936 

Vascular Tropism of Lentiviral Vectors Using Bunyaviral- 

Pseudotyped Lentiviral Vectors 

James R. Gilbert, Zhong Qian, Jody R. Arsenault, Jennifer E. 

Aquirre, Julio A. Lemos, Frank Park 

937 

Generation of Anti-LOX-1 Cytotoxic T Lymphocytes by 

AAV Manipulation of Dendritic Cells towards Preventing 

Atherosclerosis 

Jin Xie, Jay L. Mehta, Jawei Chen, Yong Liu, Paul L. 

Hermonat 

938 

Intramuscular Administration of AAV Vector Expressing 

Interleukin-10 in Dahl Salt-Sensitive Rats Prevents the 

Development of Hypertensive Heart Disease 

Mutsuko Sarukawa, Takashi Okada, Toru Yoshioka, Tatsuya 

Nomoto, Takayuki Ito, Yoshikazu Maeda, Masahisa Shimpo, 

Takashi Matsushita, Hiroaki Mizukami, Kuniko Shimazaki 

Akihiro Kume, Keiji Yamamoto, Uichi Ikeda, Kazuyuki 

Shimada, Keiya Ozawa


ABSTRACTS 



939 

Viral Vector-Mediated Chaperone Gene Therapy in a 

Mouse Model of Sporadic Parkinson Disease 

Zhizhong Dong, David P. Wolfer, Hans-Peter Lipp, Hansruedi 

Bueler 

940 

Neuroprotective Effects of XIAP Gene Therapy in 

Models of Neurodegenerative Diseases 

Sergei Musatov, Joshua Goldfein, Justin Fraser, John Pena, 

Michael G. Kaplitt 

941 

Delayed GDNF Exposure to Mature Dopaminergic 

Grafts Impairs Their Ability To Improve Motor Function 

in Parkinsonian Rats 

Biljana Georgievska, Christian Winkler, Thomas Carlsson, 

Benjamin Lacar, Deniz Kirik 

942 

Evaluation of Short (8 Weeks) and Long-Term (3 and 5 

Years) Gene Transfer Following Brain Delivery of AAV- 

AADC Vector in Parkinsonian Monkeys 

Marcel M. Daadi, Philip Pivirotto, John Bringas, Janet 

Cunningham, John Forsayeth, Jamie L. Eberling, Krystof S. 

Bankiewicz 

943 

Anti-alpha-Synuclein scFv Study 

Chun Zhou, Anne Messer 

944 

Silencing of Endogenous Human að-Synuclein by a 

Lentiviral Vector Expressing Short Hairpin RNA 

Mohan K. Sapru, Jonathan W. Yates, Martha C. Bohn 

945 

Prevention of Onset of Parkinson’s Disease by In Vivo 

Gene Transfer of Human Hepatocyte Growth Factor in 

Rodent and Primate Model 

Hiromi Koike, Akihiko Ishida, Munehisa Shimamura, 

Naoyuki Sato, Takuya Hayashi, Ryuichi Morishita 

946 

CD8 + T Cells Are Partially Mediate the Immune- 

Mediated Silencing of Adenoviral Vector-Encoded 

Transgene Expression in the Rat Brain 

Carlos Barcia, Clare Thomas, James F. Curtin, Jeffrey M. 

Zirger, Weidong Xiong, Olivier Boyer, David Klatzmann, 

Maria G. Castro, Pedro R. Lowenstein 

947 

Effects of Natural Pre-Existing Humoral Immunity to 

AAV on Administration of AAV2-NTN to the Brain of 

Non-Human Primates 

Marie A. Printz, Christopher D. Herzog, Alistair Wilson, 

Dawn Gammon, Richard Lin, Roberto Calcedo, Biplob Dass, 

Medhi Gasmi, Jeffrey H. Kordower, Raymond T. Bartus 

948 

Improvement of Symptoms of Parkinson’s Disease by In 

Vivo Gene Transfer of Human Hepatocyte Growth 

Factor - A Model of Gene Therapy for Parkinson’s 

Disease 

Hiromi Koike, Akihiko Ishida, Munehisa Shimamura, 

Naoyuki Sato, Ryuichi Morishida 

949 

AAV-Parkin Gene Therapy for Alpha-Synucleinopathy as 

Parkinson’s Diseased Model 

Hideki Mochizuki, Masanori Yamada, Yoshikuni Mizuno 

950 

Biodistribution and Safety Profile of CERE-120 (AAV2- 

NTN) Delivered to the Rodent Brain 

M. Landers, K. Kwok, E. Ketchum, C. Wilson, R. Lin, D. 

Gammon, M. Austin, L. Manlapaz, A. Wilson, J. 

Cunningham D. Vacante, M. Gasmi, K. Bishop, M. Printz, R. 

Bartus 

951 

Evaluation of HSV-Mediated Alzheimer Gene Therapy 

Vectors Using Novel Alzheimer Mouse Model: Applications 

of siRNA and Neprilysin Vectors to Adjust the 

Metabolic Imbalance of Abð in Alzheimer’s Disease 

Chang-Sook Hong, James R. Goss, William F. Goins, Joseph 

C. Glorioso 

952 

Effects of rAAV-BDNF Expression in the Striatum of the 

R6/1 Mouse 

Edgardo Rodriguez, Eileen Denovan-Wright, Ron J. Mandel 

953 

Progressive and Selective Striatal Degeneration in 

Primary Neuronal Cultures Using Lentiviral Vector 

Coding Mutant Huntingtin Fragment 

Nicole Deglon, Diana Zala, Alexandra Benchoua, Patrick 

Aebischer, Anne D. Zurn, Emmanuel Brouillet 

954 

Demonstration of In Vivo Off-Targeting Using rAAV- 

Delivered Ribozymes and shRNA Molecules in a Mouse 

Model of Huntington’s Disease 

Edgardo Rodriguez, Eileen Denovan-Wright, Alfred S. Lewin, 

Ron J. Mandel

104 


ABSTRACTS 


955 

Utilising Viral Gene Transfer to Model Polyglutamine 

Diseases and Study the Effects of Heat Shock Proteins 

Joanna L. Howarth, Stephen Kelly, Colin P. J. Glover, Michael 

E. Cheetham, Alison S. Bienemann, Darren J. Heywood, 

Russell J. Hobson, Jean-Marc Gallo, James B. Uney 

956 

HSV-1 Amplicon iBAC-FRDA Genomic Locus Delivery 

for the Treatment of Friedreich’s Ataxia 

Richard Wade-Martins, Alfredo Gimenez-Cassina, Javier Diaz- 

Nido, Filip Lim 

957 

Accurate Humanized Mouse Models of Friedreich Ataxia 

Joseph P. Sarsero, Timothy P. Holloway, Lingli Li, Panos A. 

Ioannou 

958 

Biomarker Studies of Interferon-Beta in Mice Following 

Gene Based Delivery 

Harald Petry, Linda Cashion, Oliver Ast, Ann Orme, Paul 

Szymanski, Cynthia Gross, Terry Hermiston, Maxine Bauzon, 

Peiyin Wang, Alan Brooks Perry Liu, Hu Sheng Qian, Jude R. 

Samulski, Gabor M. Rubanyi, Richard N. Harkins 

959 

Pharmacokinetic Studies of Human IFN-ß in Mice 

Following Gene-Based Delivery 

Linda Cashion, Ann Orme, Alan Brooks, Peiyin Wang, Paul 

Szymanski, Cecile Chartier, Maxine Bauzon, Hu Sheng Qian, 

Perry Liu, Heather Gibson Kathy White, Terry Hermiston, R. 

Jude Samulski, Gabor M. Rubanyi, Richard N. Harkins 

960 

Gene-Based Delivery of Interferon-beta Is Efficacious in 

a Murine Model of Acute Experimental Allergic Encephalomyelitis 

Caralee Schaefer, Toni Rose Arceo, Paul Szymanski, Hu Sheng 

Qian, Heather Gibson, Harald Petry, Claudia Goldman, Linda 

Cashion, Alan Brooks, Cynthia Gross Peiyin Wang, Gabor M. 

Rubanyi, Richard N. Harkins 

Neurologic: Other 

961 

AAV1-Mediated Gene Delivery to the Adult GM1- 

Gangliosidosis Mouse Hippocampus Leads to Marked 

Reduction in Storage in Physically and Synaptically- 

Connected Areas of the Brain 

Miguel Sena-Esteves, Marike Broekman, Fabricio F. Costa, 

Stanley G. LeRoy, Laryssa A. Comer 

962 

Treatment of Feline G Gangliosidosis with Mesenchy- 

M1 

mal Stem Cells and Lentiviral Gene Therapy 

Douglas R. Martin, Karin Haack, Glenn P. Niemeyer, Nancy 

R. Cox, Tal Kafri, Henry J. Baker 

963 

Efficient Suppression of GluR1 Receptor Expression In 

Vitro and In Vivo by Infection with HIV1 Vectors Expressing 

siRNA 

Atsushi Miyanohara, Kiyohiko Kinjoh, Mike Hefferan, Osamu 

Kakinohana, Martin Marsala 

964 

Adenoviral-Mediated Expression of Brain-Derived 

Neuroptrophic Factor or Neurotrophin-3 Promotes 

Sprouting of Lesioned Serotonergic Axons in the Central 

Nervous System 

Michael H. Grider, H. David Shine 

965 

Antioxidant Enzyme Protection from HIV-1 gp120- 

Induced Neuronal Apoptosis by Gene Delivery Using 

rSV40 Vectors: Implication for Gene Therapy of AIDS 

Lokesh Agrawal, David S. Strayer 

966 

AAV Delivered Single-Chain Antibodies as Passive 

Immuno-Therapy for Prion Disease 

Charles A. Wuertzer, Mark A. Sullivan, Howard J. Federoff 

967 

Transgenic Genomic Reporter Assays for Functional 

Studies and Stem Cell Therapy 

Joseph P. Sarsero, Samuel McLenachan, Timothy P. Holloway, 

Lingli Li, Kerry J. Fowler, Ivan Bertoncello, Lucille Voullaire, 

Sophie Gazeas, Panos A. Ioannou 

968 

Co-Expression of FGE Is Essential for Gene Therapy of 

MLD Based on Cross-Correction of ASA Deficiency 

Yukiko Takakusaki, Sanae Hisayasu, Atsushi Watanabe, Kumi 

Adachi, Takashi Shimada 

969 

Development of Non-Invasive Imaging Strategies to 

Assess the Impact of Gene Therapy: Phenotypic Assessment 

of the Neurodegenerative CNS Disorder Late 

Infantile Neuronal Ceroid Lipofuscinosis Using Magnetic 

Resonance Spectroscopy 

Neil R. Hackett, Dolan Sondhi, Stephen M. Kaminsky, Stefan 

Worgall, Lisa A. Arkin, Charleen A. Hollmann, Dikoma C. 

Shungu, Xiangling Mao, Jonathan P. Dyke, Linda Heier Mark 

Souweidane, Michael Kaplitt, Ronald G. Crystal


ABSTRACTS 


970 

p53 Expression and Serum IgE Levels in Patients with 

Neurofibromatosis type 1 

Mauro Geller, Marcia G. Ribeiro, Alexandra P. Q. C. Araújo, 

Kalynka S. Higino, Rafael B. Varella, Lisa B. Oliveira, Fabio P. 

Nunes, Joâo G. R. Gonçalves 

971 

Human Embroyonic Stem Cells Transplantation on 

Paralytic Patients 

Geeta Barthakur Shroff 

972 

HSC-Based Ex Vivo Gene Therapy Prolongs Survival 

and Improves the Phenotype of Globoid Cell Leukodystrophy 

Mice 

Alessandra Biffi, Ilaria Visigalli, Letterio S. Politi, Angelo 

Quattrini, Ubaldo Del Carro, David Wenger, Claudio 

Bordignon, Luigi Naldini 


973 

Targeting of CCR5 through Intrabody Mediated HIV-1 

Vector Gene Delivery Is Sufficient for Blocking HIV-1- 

Infection and Confers a T-Cell Survival and Growth 

Advantage 

Christina H. Swan, Kimberlee M. Fischer, Bernd Bühler, Mario 

P. Tschan, Carlos F. Barbas, III, Bruce E. Torbett 

974 

Towards Gene Knock out Therapy for AIDS/HIV: 

Targeted Disruption of CCR5 Using Engineered Zinc 

Finger Protein Nucleases (ZFNs) 

Yann Jouvenot, Elena Perez, Fyodor D. Urnov, Christian 

Beausejour, Jeremy Rock, Ya-Li Lee, Jeffrey C. Miller, Edward J. 

Rebar, Michael C. Holmes, Dale Ando Jim Riley, Philip D. 

Gregory, Carl June 

975 

Immunization with an Adenovirus Vector Expressing a P. 

aeruginosa OprF Epitope in the Capsid Enables Boosting 

of Anti-Epitope Immune Responses and Protects Against 

Pulmonary Infection with P. aeruginosa 

Stefan Worgall, JianPing Qiu, Anja Krause, Kyung-Hee Kim, 

Peter W. Roelvink, Joseph T. Bruder, Thomas J. Wickham, Imre 

Kovesdi, Ronald G. Crystal 

976 

IFNα Gene Therapy of Woodchuck Hepatitis with 

Adeno-Associated Virus: Differences in Duration of 

Gene Expression and Antiv 

iral Activity Using Intraportal or Intramuscular Routes 

Berraondo Pedro, Ochoa Laura, Crettaz Julien, Rotellar 

Fernando, Vales Africa, Martinez-Anso Eduardo, Zaratiegui 

Mikel, Ruiz Juan, Prieto Jesus, Gonzalez-Aseguinolaza Gloria 

977 

CD4 + T Cell Independent DNA Vaccination Against 

Pneumocystis Carinii in Mice 

Mingquan Zheng, Myle Robicchaux, Christoph Kliment, Judd 

E. Shellito, Jay K. Kolls 

978 

Production of SARS Coronavirus-Like Particles That 

Bind Host Cells and Serve as Vaccine Antigen 

Yi-Chun Yeh, En-Hau Lin, Chang-Jen Huang, Ning-Sun 

Yang, Pei-Wen Hsiao 

979 

Antigen-Transduced T Cell Blasts Specifically Delete 

Antigen-Specific Cytotoxic T Cells and Induce a 

CD4+CD25+ Regulatory T Cell Response 

Aaron E. Foster, Takayuki Okamura, Cliona M. Rooney 

980 

Specific Immune Response Against the Plasmodium 

falciparum CS Protein Mediated by Baculovirus Vectors 

Robert Strauss, Andreas Hüser, Nelson Di Paolo, Andre Lieber, 

Christian Hofmann 

981 

Modulation of Human Dendritic Cell Function Following 

Viral Vectors: Implications for Gene Therapy in 

Vaccination 

Peng H. Tan, Giovanna Lombardi, Andrew J. T. George 

982 

Lentiviral Vector Mediated Genetic Modification of 

Dendritic Cells as a Vaccine Against Hepatitis C 

Richard C. Koya, Yi Zheng, Emmanuelle Faure, Guann-yi Yu, 

Takahiro Kimura, Nori Kasahara, Renata Stripecke 

983 

Inhibition of Hepatitis C Virus Replication by In Vitro 

Selected Specific RNA Aptamers 

Jung-Hye Kim, Nam Yeon Kim, Seong-Wook Lee 

984 

Directed Molecular Evolution Generates Novel HIV-1 

Envelope Variants That Can Elicit Broadly Neutralizing 

Antibody Responses in Rabbits 

Li Xu, Xiaohan Du, Wenge Zhang, Susan Tang, NayWei 

Soong, Michael Zwick, Dennis Burton, Robert Whalen 

985 

Targeting CCR3 To Protect from Human Immunodeficiency 

Virus (HIV-1) 


986 

In Vivo Production of Anti-HIV-1 Neutralizing Antibody 

from Rhesus Macaque Muscle Using a Recombinant 

Adeno-Associated Virus Vector 

K. Reed Clark, Jianchao Zhang, David C. Montefiori, Philip 

R. Johnson

106 


ABSTRACTS 


987 

Foamy Virus Vectors That Block HIV Infection 

Jason Taylor, Ingrid Bahner, Lucia Vojtech, Erik Olson, James 

Allen, Dorothee von Laer, Donald B. Kohn, David W. Russell, 

Robert E. Richard 

988 

The Utility of rAAV as a Genetic Vaccine Vector for 

Malaria 

Grant Logan, Maolin Zhang, Ross Coppel, Peter Rowe, Lina 

Wang, Ian Alexander 

989 

Toward Multivalent Adeno-Based Vaccine for Malaria: 

Effects of Promoter Strength on Immunogenicity and 

Vaccine Efficacy 

Joseph T. Bruder, Maureen E. Stefaniak, Duncan McVey, 

Damodar Ettyreddy, Noelle B. Patterson, Fe Baraceros, Joseph J. 

Campo, C. R. King, Denise L. Doolan 

990 

Unexpected Immune Enhancement by Autologous 

Lentiviral Modified CD4 T Cells: Results from a Phase I 

Gene Transfer Trial of Lentiviral Vector-Modified T Cells 

Expressing Antisense Envelope 

Rob R. MacGregor, Bruce L. Levine, Xiaobin Lu, Laurent M. 

Humeau, Tessio Rebello, Peter Manilla, Yajin Ni, Gwendolyn 

K. Binder, Richard Carroll, Jean Boyer Frederic Bushman, 

Vladimir Slepushkin, Boro Dropulic, Carl H. June 

991 

Adenovirus-Mediated IL-12 Gene Transfer Increase Virus 

Specific CD8 T Cell Response in Aged Mice 

Jian Chen, PingAr Yang, Qi Wu, Allan J. Zajac, Xiaohong 

Wang, Kohtaro Fujihashi, Hui-Chen Hsu, John D. Mountz 

992 

Suppression of the HIV-1 Long Terminal Repeat by 

Transducible Artificial Transcription Factors 

Hyun-chul Shin, Heung-sun Kwon, Eui-chul Park, Jin-soo 

Kim 

993 

Mouse Model of Acute Hepatitis Using Adenovirus 

Vector Designed for Gene Therapy 

Tamon Nishino, Katsuhito Iikura, Toya Ohashi, Yoshikatsu Eto 

994 

E1 Adenovirus Gene Transfer Vectors Inhibit HIV-1 

Replication in Human Alveolar Macrophages 

Robert J. Kaner, Francisco Santiago, Ronald G. Crystal 

995 

Effect OF BCL-2 Overexpression on Plasmodium 

Infection 

Francisco Martinez-F, Jose Luis Ventura, Luis F. Covarrubias, 

Alejandro Zentella-D, Martha Legorreta-H 

Infectious Diseases and Vaccines: RNAi for Gene 

Therapy of Infections 

996 

Development of an siRNA Based Therapy for Ebola 

Virus Infection 

Thomas W. Geisbert, Lisa E. Hensley, Kristopher M. Curtis, 

Joan B. Geisbert, Kathleen M. Daddario, Elliott Kagan, Amy 

C. H. Lee, Lorne Palmer, Lloyd Jeffs, Ian MacLachlan 

997 

Loop Composition as Well as Orientation of the Guide 

Strand in shRNA May Dramatically Modulate Activity of 

the shRNA: Effect on the Development of RNAi-Based 

Inhibitors 

Alexander A. Kolykhalov, Michael W. Graham, David A. Suhy, 

Peter W. Roelvink, Annette R. Schroeder, Amy E. Parker, Luz 

M. Garcia, Mark A. Kay, Sara M. Cunningham, Linda B. 

Couto 

998 

The HIV-1 LTR-hsp70 Fusion Promoter Directs HIV-1 

Inducible Expression of Anti-Rev siRNA along with 

HIV-1 Rev Transdominant Mutant RevM10 

Hoshang J. Unwalla, Ingrid Bahner, Ha-Tang Li, Ming-Jie Li, 

Donald Kohn, John J. Rossi, Yuliya Gokhgauzer 

999 

siRNA-Mediated Interference of Double-Stranded RNA 

Binding Protein [E3L] Is an Effective Strategy to Inhibit 

Vaccinia Virus 

Dave S. Rajnish, James P. McGettigan, Giuseppe Nunnari, 

Matthias J. Schnell, Roger J. Pomerantz 

1000 

In Vitro and In Vivo Evaluation of Optimized Short- 

Hairpin RNAs for the Treatment of the Hepatitis C Virus 

Using Expression Cassettes Producing One, Two or 

Three Anti HCV shRNAs 

David A. Suhy, Linda B. Couto, Alexander A. Kolykhalov, 

Amy E. Parker, A. Rosanna Schroeder, Luz Maria Garcia, 

Gabriel Haniff, Sara M. Cunningham, Mark A. Kay, Peter W. 

Roelvink 

1001 

Knockdown of CXCR4 Expression Sufficient for Blocking 

HIV-1-Infection Requires Combinatorial Intrabody 

and siRNA Mediated HIV-1 Vector Gene Delivery 

Christina H. Swan, Mario P. Tschan, Carlos F. Barbas, III, 

Bruce E. Torbett 

1002 

Therapeutic Design Principles for Achieving Long-Term 

Suppression of HIV-1 with RNA Interference 

Joshua N. Leonard, David V. Schaffer 

1003 

Inhibition of HBV Replication In Vivo by HBx-Specific 

siRNA 

Sooin Kim, Duckhyang Shin, Meehyein Kim, Mahnhoon Park


ABSTRACTS 


Cancer - Targeted Gene Therapy: Targeting of Adenoviral 

Vectors 

1004 

Enhanced Survival Uncovers Liver Toxicity in Mice with 

Orthotopic Advanced Ovarian Cancer Treated with 

Gemcitabine and Ad5/3-Dð24, a Serotype 3 Receptor 

Targeted Oncolytic Adenovirus 

Mari Raki, Anna Kanerva, Ari Ristimaki, Renee A. Desmond, 

Dung T. Chen, Tuuli Ranki, Merja Sarkioja, Lotta 

Kangasniemi, Akseli Hemminki 

1005 

Replication and Anti-Tumor Efficacy of Conditionally 

Replicating Adenoviral Vectors for Glioma Treatment 

Maarten ter Horst, Mark Rodijk, Eric Brouwer, Rob C. 

Hoeben, Bertie de Leeuw, Peter A. E. Sillvis Smitt 

1006 

Analysis of Resistance to Viral Oncolysis in Primary 

Ovarian Cancer Cell Cultures 

Robert Strauss, Pavel Sova, Shaoheng Ni, Nancy Kiviat, Nicole 

Urban, Andre Lieber 

1007 

Genetic Strategies to Improve Oncolytic Adenoviruses 

for Targeted and Efficient Virotherapy of Malignant 

Melanoma 

Stephan Schierer, Daniela Kosmides, Angel A. Rivera, Andrea 

Hesse, Astrid Mainka, Andrea L. Volk, Gerold Schuler, David 

T. Curiel, Dirk M. Nettelbeck 

1008 

Prostate-Specific Oncolytic Adenovirus Based on 

Bidirectional TSTA System 

Makoto Sato, Mai Johnson, Sanjiv S. Gambhir, Michael Carey, 

Lily Wu 

1009 

Gene Therapy for Prostate Cancer by Controlling 

Adenovirus E1A and E4 Gene Expression with PSES 

Enhancer 

Xiong Li, YanPing Zhang, Hong-sup Kim, Kyung-Hee Bae, 

Keith M. Stantz, Sang-Jin Lee, Chaeyong Jung, Juan A. 

Jiménez, Thomas A. Gardner, Meei-Huey Jeng Ching-hai Kao 

1010 

A bð-hCG Expressing Trackable and Tumor Targeted 

Conditionally Replicating Adenovirus for Treatment of 


Maria Rajecki, Anna Kanerva, Lotta Kangasniemi, Kah-Whye 

Peng, Merja Sarkioja, Ulf-Hakan Stenman, Henrik Alfthan, 

Akseli Hemminki 

1011 

A Combination of Ceramide Analogue and FasL Gene 

Therapy Can Be a Potential Novel Therapy for Prostate 

Cancer 

Ahmed El-Zawahry, David Holman, Xiang Liu, Saeed 

Elojaem, Alicja Bielawaska, Jacek Bielwaski, Zdislaw Szulc, 

Yusuf Hannun, James S. Norris 

1012 

Survivin-Responsive Conditionally Replicating Adenovirus 

Exhibits Strictly Cancer-Specific and Efficient Viral 

Replication 

Ken-ichiro Kosai, Junichi Kamizono, Satoshi Nagano, Yoshiteru 

Murofushi, Setsuro Komiya 

1013 

Pharmacology of Tropism Modified Adenovectors for 

Therapy of Disseminated Cancer 

Masaki Akiyama, Selva Murugesan, David A. Einfeld, Douglas 

E. Brough, Jason G. Gall, Thomas J. Wickham, C. Richter 

King 

1014 

Adenovirus-Mediated MUC1-Driven Expression of the 

Sodium Iodide Symporter (NIS) in Pancreatic Tumors 

Provides for In Vivo Radioiodide Imaging and Therapy 

Roisin M. Dwyer, Elizabeth R. Bergert, Michael K. O’Connor, 

Sandra J. Gendler, John C. Morris 

1015 

Inhibition of Tumor Growth in Xenografted Nude Mice 

with Adenovirus-Mediated Endostatin Gene Comparison 

with Recombinant Endostatin Protein 

Zhihui Liang, Wenlin Huang 

1016 

Retargeted Selectively Replicating Adenoviruses in 

Combination with Antiangiogenic Therapy for the 

Therapy of Colon Cancer 

Krisztian Homicsko, Alexander Lukashev, Richard Iggo 

1017 

Selective Induction of Tumor-Associated Antigens in 

Murine Pulmonary Vasculature Using Double-Targeted 


Maaike Everts, SangAe Kim-Park, Meredith A. Preuss, Michael 

J. Passineau, Joel N. Glasgow, Alexander V. Pereboev, 

Parameshwar J. Mahasreshti, William E. Grizzle, Paul N. 

Reynolds, David T. Curiel 

1018 

Infectivity Enhancement for Ovarian Cancer and Glioma 

Via a Novel Human Adenoviral Vector with a Chimeric 

Fiber Derived from Canine Adenovirus Type 1 

Mariam A. Stoff-Khalili, Angel A. Rivera, Joel N. Glasgow, 

Long P. Le, Alexander Stoff, Maaike Everts, Yuko Tsuruta, 

Yusoke Kawakami, J. Michael Mathis, Larisa Pereboeva Peter 

Dall, David T. Curiel

108 


ABSTRACTS 


1019 

Enhanced Therapeutic Effects of Recombinant Adenovirus 

Ad-AFPDð19, Selectively Replicating in að-Fetoprotein-Producing 

Human Liver Cancer Cells 

Il-Kyu Choi, Dong Hyun Ko, Jaesung Kim, Ji-young Yoo, A- 

Rum Yoon, Min-Ju Kim, Joo Hyuk Sohn, Chae-Ok Yun, Joo- 

Hang Kim 

1020 

Noninvasive Optical Monitoring of Conditionally 

Replivative Adenovirus Replication 

Julia Davydova, Hidetaka A. Ono, Long P. Le, Tatyana 

Gavrikova, Seiji Yamamoto, Amy R. Nelson, David T. Curiel, 

Masato Yamamoto 

1021 

Development of a Syrian Hamster Animal Model To 

Study Replication Competent Adenovirus Vectors for 

Cancer Gene Therapy 

Maria A. Thomas, Jacqueline F. Spencer, Karoly Toth, Marie C. 

LaRegina, Ann E. Tollefson, Baoling Ying, Mohan 

Kuppuswamy, Jennifer M. Meyer, Drew L. Lichtenstein, Louis 

A. Zumstein William S. M. Wold 

1022 

Single-Dose Intravenous Toxicity Study with the Oncolytic 

Adenovirus Vector VRX-007, Adenovirus Type 5, 

and a Replication-Defective Adenovirus in Syrian 

Hamsters 

Karoly Toth, Jacqueline F. Spencer, Drew L. Lichtenstein, Ann 

E. Tollefson, Jennifer M. Meyer, Maria A. Thomas, Mohan 

Kuppuswamy, Baoling Ying, Louis A. Zumstein, William S. 

M. Wold 

1023 

Determination of the Maximum Tolerated Dose of the 

Replication-Competent Adenovirus Vector VRX-007 

Following Intravenous Administration in Syrian Hamsters 

Maria A. Thomas, Jacqueline F. Spencer, Jennifer M. Meyer, 

Marie C. LaRegina, Karoly Toth, Drew L. Lichtenstein, Ann 

E. Tollefson, Mohan Kuppuswamy, Baoling Ying, Louis A. 

Zumstein William S. M. Wold 

1024 

An Oncolytic Conditionally Replicating Adenovirus for 

Hormone-Dependent and Hormone-Independent 


Wing-Shing Cheng, Helena Dzojic, Berith Nilsson, Thomas 

H. Totterman, Magnus Essand 

1025 

A PPT Promoter-Based Adenovirus with Two-Step 

Transcriptional Amplification (TSTA) for Experimental 


Helena Dzojic, Wing-Shing Cheng, Magnus Essand 

1026 

In Vivo Imaging for Evaluation of Adenoviral Capsid 

Modifications for Systemic Delivery in an Orthotopic 

Model of Locally Advanced Lung Cancer 

Merja Särkioja, Anna Kanerva, Tanja Hakkarainen, Jarmo 

Salo, Lotta Kangasniemi, Mari Raki, Tuuli Ranki, Hongju 

Wu, David T. Curiel, Akseli Hemminki 

1027 

Cytotoxicity of Oncolytic Adenoviruses in Breast Cancer 


M. Bazan-Peregrino, K. Fisher, S. Hale, P. Farrow, D. Kirn, R. 

Iggo, K. Homicsko, R. Hernandez-Alcoceba, G. Hallden, V. 

Mautner Y. Shen, L. Seymour 

1028 

Reduction of Replication of Oncolytic Adenoviruses with 

Pharmacological Intervention: Treatment of Replication 

Associated Toxicity 

Anna Kanerva, Mari Raki, Lotta Kangasniemi, Renee A. 

Desmond, Helena Isoniemi, Krister Hockerstedt, Akseli 

Hemminki 

1029 

Integrin and Serotype 3 Receptor-Targeted Virotherapy 

Using a Telomerase-Selective Oncolytic Adenovirus 

Yosuke Kawakami, Angel A. Rivera, Minghui Wang, Dirk M. 

Nettelbeck, Mariam A. Stoff-Khalili, Mack N. Barnes, Gene P. 

Siegal, Ronald D. Alvarez, David T. Curiel 


Session I to Poster Session III and will follow Abstract 

1029. See page 65 for abstract title and authors.) 

Hematologic - New Advances 

1030 

A Rhesus Macaque Transplant Model for Drug Selection 

of Hematopoietic Stem Cells Transduced with MGMT 

Vectors 

Yan Shou, John Gray, Brian Agricola, Zhijun Ma, Derek A. 

Persons, Brian P. Sorrentino 

1031 

Purification of Cells with High Aldehyde Dehydrogenase 

Activity Reveals a CD133 Negative Population with 

Enhanced Repopulating Ability after Ex Vivo Culture 

David A. Hess, Louisa Wirthlin, Timothy P. Craft, Phillip E. 

Herrbrich, Jan A. Nolta 

1032 

Hepatocyte Growth Factor Maintains Hematopoietic 

Progenitors during Retroviral Transduction 

Todd E. Meyerrose, Phillip E. Herrbrich, Michael H. Creer, Jan 

A. Nolta


ABSTRACTS 


1033 

Enhanced Engraftment of Retrovirally Transuduced 

Hematopoietic Stem Cells Following Treatment with 

Diprotin-A 

Chaorui Tian, Jessamyn Bagley, Daron Forman, John Iacomini 

1034 

Automated Retroviral Insertion Site Sequence Analysis 

and Mapping Tool followed by Database Analysis 

Stephanie Laufs, Frank A. Giordano, Agnes Hotz-Wagenblatt, 

Uwe Appelt, Daniel Lauterborn, Kurt Fellenberg, W. Jens Zeller, 

Stefan Fruehauf 

1035 

Use of Multi Color p27 kip1 Based Flow Cytometric Assay 

To Evaluate and Characterize G 0 to G 1 Cell Cycle Related 

Changes in Human Hematopoietic Cells after Subjection 

to a Clinically Applicable Ex Vivo Transduction Protocol 

Jesper Bonde, Ryan Lahey, Phillip Herrbrich, Jan A. Nolta 

1036 

Engraftment of side population Stem Cells in Canine 

Recipients 

Andrea K. Vaags, David Caton, Krista Halling, Cathy Gartley, 

Suzana Rosic-Kablar, Stephen A. Kruth, Margaret R. Hough 

1037 

Adherent Human Umbilical Cord Blood Derived 

Mesenchymal Stem Cells Do Not Augment Viral Gene 

Transfer into Hematopoietic Stem/Progenitor Cells 

Todd E. Meyerrose, Phillip E. Herrbrich, Michael H. Creer, Jan 

A. Nolta 

1038 

Lentiviral Gene Transfer into HSC Is Enhanced by Early- 

Acting Cytokines without Impairing Stem Cell Properties 

and Involves Cellular Responses Distinct from Cell 

Cycle Control 

Francesca Santoni de Sio, Luigi Naldini 

1039 

Ex Vivo Pre-Selection Increases Marking Following 

Transplant of Mixtures of Bone Marrow from Wild Type 

and Green Fluorescent Protein-Methylguanine 

Methyltransferase P144K Fusion Protein Transgenic Mice 

Uimook Choi, Lanise Cardwell, Natalia Loktionova, Harry L. 

Malech 

1040 

Donor Origin of Multipotent Adult Progenitor Cells in 

Radiation Chimeras 

Morayma Reyes, Sheng Li, En Kimura, Jeffrey S. Chamberlain 

1041 

Delayed Infusion of Hematopoietic Stem Cells Improves 

Engraftment after Low-Dose Parenterally Administered 

Busulfan in a Congenic Murine Transplant Model 

Matthew M. Hsieh, Saskia Langemeijer, Bob Wesley, Elizabeth 

M. Kang, John F. Tisdale 

1042 

Creation of Tolerogenic Dendritic Cells by Non Viral 

Gene Transfer with Immunoliposomes 

Peng H. Tan, Sven C. Beutelspacher, Giovanna Lombardi, 

Andrew J. T. George 

1043 

In Vivo Gene Transfer into Rat Hematopoietic Stem 

Cells Using rSV40 Viral Vectors 

Bianling Liu, Jean-Pierre Louboutin, Judy Daviau, David S. 

Strayer 

1044 

Radioprotective Gene Therapy through Retroviral 

Expression of Manganese Superoxide Dismutase 

Thomas D. Southgate, Victoria Sheard, Michael D. Milsom, 

Rob J. Mairs, Marie Boyd, Leslie J. Fairbairn 

1045 

Overexpression of an ABCG2 Multidrug Transporter 

Variant Does Not Effect Proliferation or Differentiation 

of Hematopoetic Stem Cells 

Olga Ujhelly, Nora Kucsma, Judith Cervenak, Linping Chen, 

Manuel Grez, Balazs Sarkadi, Katalin Nemet 

1046 

Stable Correction of Hematopoietic Stem Cells with 

Non-Viral Gene Transfer 

Meenakshi Noll, Ryan Bennett, Stephen Yant, Mark A. Kay, 

Markus Grompe 

1047 

Chemoprotective Gene Therapy through Retroviral 

Expression of the X-Ray Cross Complementing Protein 

(XRCC1) 

Thomas D. Southgate, Katherine Clarke, Leslie J. Fairbairn 

1048 

Engineering and Development of I-AniI Homing 

Endonucleases for Gene Correction Applications 

Andrew M. Scharenberg, David J. Rawlings, Raymond J. 

Monnat, Barry L. Stoddard 

1049 

Serum Free Fedia Is the Best for Cord Blood Hematopoietic 

Cells Expansion 

Kamran Alimoghaddam, Mitra Khalili, Masoud Soleimani, Lili 

Moezi, Ardeshir Ghavamzadeh

110 


ABSTRACTS 


Gene Regulation: Regulated Systems and Tissue 

Specific Expression 

1050 

Adenoviral Clostridial Light Chain Expression in the 

Medial Forebrain Bundle: A Reversible Model of 

Dopamine Depletion 

Mary E. Garrity-Moses, Qingshan Teng, Jun Yang, Thais 

Federici, Erin Gilbert, Thyagarajan Subramanian, Nicholas M. 

Boulis 

1051 

Prostate Cancer-Targeted Suicide Gene Therapy 

Achieved Effective Tumor Destruction While Safeguarding 

Against Systemic Toxicity 

Mai Johnson, Makoto Sato, Burton Jeremy, Sanjiv S. Gambhir, 

Michael Carey, Lily Wu 

1052 

A Versatile Chimeric Enhancer Vector That Is Highly 

Inducible by Hypoxia and Metals 

Jonghoe Byun, Jae-Young Lee, Young-Sam Lee, Jeong-Min 

Kim, Hyung-Suk Jang, Koung Li Kim, In-Soon Shin, Jung- 

Sun Lee, Wonhee Suh, Eun-Seok Jeon Duk-Kyung Kim 

1053 

Novel Exogenously Regulated Stem Cell–Mediated 

Gene Therapy Platform for Spinal Fusion 

Gadi Pelled, Amir Hasharoni, Yoram Zilberman, Gadi 

Turgeman, Gregory A. Helm, Meir Libergall, Dan Gazit 

1054 

Bio-Distribution Studies of Lentiviral Vectors Injected 

Intravenously by Dynamic Bioluminescence Imaging 

Analyses: Development of APC-Transcriptionally 

Targeted Vectors for Genetic Vaccination Purposes 

Takahiro Kimura, Richard C. Koya, Nori Kasahara, Renata 

Stripecke 

1055 

Reduced Toxicity Selection of Modified Murine Repopulating 

Stem Cells Results in an Unchanged Differentiation 

Potential of Oligoclonal MGMT-P140K 

Overexpressing Murine Hematopoiesis 

C. Ball, M. Schmidt, C. von Kalle, H. Glimm 

1056 

Suppression and Enhancement of Gene Repair by the 

Modulation of ATM Function in Mammalian Cells 

Julia Engstrom, Luciana Ferrara, Hetal Parekh-Olmedo, Eric B. 

Kmiec 

1057 

Generating Efficient Promoters for Cardiovascular Gene 

Therapy Using Synthetic Promoter Libraries 

Stuart A. Nicklin, Anna F. Dominiczak, Andrew H. Baker 

1058 

Novel Prostate-Specific Vector Regulation System 

Incorporating a Probasin-Based Promoter, Tet-Regulated 

Transcriptional Silencer Protein, and a Positive Feedback 

Loop 

Jan Woraratanadharm, Semyon Rubinchik, Hong Yu, John 

(Jian-yun) Y. Dong 

1059 

Development of a Novel Regulatory System Allowing 

Doxycycline-Controlled Suppression of Target Genes in 


Lahouari Amar, Jacques Mallet, Roland Vogel 

1060 

An Efficient GLP-1 Expression System Using Two Step 

Transcription Amplification for Type 2 Diabetes Gene 


Kyung Soo Ko, Minhyung Lee, Seungjoon Oh, Eunsu Lee, 

Cheol-Hee Ahn, Byoung Doo Rhee 

1061 

An Ecdysone Receptor-Based Gene Switch for Precisely 

and Variably Regulated Expression of Genes in Mammalian 


Anand K. Katakam, Chris L. Herold, Prasanna Kumar 

1062 

Potential Utility of Heat Shock Protein 70 Promoter 

(HSP70-P) as a Stress Responsive Promoter in Gene 

Delivery: HSP70-P Regulated Expression of Transgenes 

SOD1 and GPX1 


1063 

Independent Modulation of Multiple Genes Using 

Modified Ecdysone Receptors 

Jennifer L. Friz, Robert E. Hormann, Prasanna Kumar, Dean 

E. Cress 

1064 

Possible Involvement of Hypoxia Inducible Factor and 

Inflammatory Cytokines in the Development of Rat 

Cerebral Collateral Circulation 

Mitsuhiro Anan, Tatsuya Abe, Takeshi Matsuda, Ryo Inoue, 

Keisuke Ishii, Minoru Fujiki, Hidenori Kobayashi 

1065 

Geneticin Targets Nonsense-Mediated mRNA Decay in 

Epidermolysis Bullosa 

Alfred Klausegger, Verena Wally, Kamil Oender, Helmut 

Hintner, Johann Bauer 

1066 

Optimisation of the Tet-On System for In Vivo Electro 

Gene Transfer to Muscle Tissue in Mice 

Pernille H. Moller, Jens Eriksen, Lluis M. Mir, Julie Gehl


ABSTRACTS 


1067 

Regulated Expression of Biotherapeutic Proteins and 

shRNA Using an Ecdysone Receptor-Based Gene Switch 

Prasanna Kumar, Anand K. Katakam, Chris L. Herold 

1068 

Luciferase as an In Vivo Reporter for Selective Liver 

Repopulation in Adult FAH-/- Mice 

Kirk J. Wangensteen, Andy Wilber, Guisheng Zeng, Joel L. 

Frandsen, R. Scott McIvor, Stephen C. Ekker, Xin Wang 

1069 

Increased Antioxidant Levels Following MnSOD-PL 

Transfection Results in Increased Radioprotection of 

Normal Tissue but Not Tumor Cells 

Michael W. Epperly, Suhua Nie, Xichen Zhang, Joel S. 

Greenberger 

1070 

Easy Search on Clinical Gene Transfer: The Euregenethy 

(EC-DG Research FP5-TN) Data Base on Pre-Clinical & 

Clinical Gene Transfer & Therapy 

Odile Y. D. Cohen-Haguenauer, Fatima Bosch, Manuel 

Carrondo, Klaus Cichutek, Nicole Deglon, Barbara Demeneix, 

George Dickson, Gosta Garhton, Bernd Gansbacher, Cecilia 

Melani Luigi Naldini, Felicia Rosenthal, Sandro Rusconi, 

Seppo Yla-Herttuala, Heinz Zwierzina 

1071 

Potential Utility of Adipose-Derived Stromal Cells 

(ASCs) as a Carrier for Ex Vivo Gene Therapy – Gene 

Transfer Efficiency of ASCs 

Rei Ogawa, Hideki Hanawa, Yukihiko Hirai, Toshiyuki 

Kurai, Hiroshi Mizuno, Hiko Hyakusoku, Takashi Shimada 

1072 

Superactivation of the SRað and CMV Promoters Using 

Designed Zinc Finger Proteins 

Victor V. Bartsevich, Andreas Reik, Yuanyue Zhou, Michelle 

Kong, Xiao-Yong Li, Lyndon Warfe, Lei Zhang, Casey C. 

Case, Trevor Collingwood, Philip D. Gregory Edward J. Rebar 

1073 

Down-Regulation of Epithelial Glycoprotein-2 and 

VEGF-D by Engineered Zinc Finger Protein Transcription 

Factors 

Willemijn M. Gommans, Robbert H. Cool, Pamela M. J. 

McLaughlin, Olli H. Laitinen, Seppo Yla-Herttuala, Bert J. 

van der Zaal, Hidde J. Haisma, Marianne G. Rots 



Clinical Gene Therapy 



The Use of Genetically Modified Antigen Presenting 

Cells to Generate Donor-Derived Virus-Specific Cytotoxic 

T Lymphocytes Reactive Against CMV and Adenoviral 

Antigens for Clinical Use 

Gary D. Myers, Ann Leen, Helen Huls, Elizabeth Buza, Jeff 

Chang, Robert A. Krance, George Carrum, Malcolm K. 

Brenner, Cliona M. Rooney, Helen E. Heslop Catherine M. 

Bollard 


Phase I Clinical Trial Demonstrates Safety and Feasibility 

of Autologous Cellular Therapy with Lentiviral Modified 

CD4 T Cells Expressing Anti-HIV Antisense in Patients 

with HAART Resistant HIV-1 Infection 

Boro Dropulic, Rob-Roy MacGregor, Tessio Rebello, Peter 

Manilla, Laurent Humeau, Xiaobin Lu, Vladimir Slepushkin, 

Gwendolyn K. Binder, Cathy Ybarra, Bruce L. Levine Frederic 

Bushman, Carl H. June 


Stable Polyclonal Hematopoietic Repopulation after 

Successful Clinical Gene Therapy of Chronic Granulomatous 

Disease (CGD) 

Manfred Schmidt, Kerstin Schwarzwaelder, Marion G. Ott, 

Stefan Stein, Hanno Glimm, Annette Deichmann, Ulrich 

Sieler, Dieter Hoelzer, Reinhard Seger, Manuel Grez Christof 

von Kalle 


Phase I Clinical Trial of Recombinant Adeno-Associated 

Virus (rAAV)-Alpha-1 Antitrypsin Vectors 

Mark L. Brantly, Margaret Humphries, Sihong Song, Thomas 

Conlon, Amy Poirier, Barry J. Byrne, Richard Snyder, Terence 

R. Flotte 


Therapeutic Misconception in Early Phase Gene Transfer 

Trials 

Gail E. Henderson, Michele E. Easter, Catherine Zimmer, 

Nancy M. P. King, Arlene M. Davis, Barbra Bluestone 

Rothschild, Larry R. Churchill, Benjamin S. Wilfond, Daniel 

K. Nelson 


Successful Myoblast Transplantation to Duchenne 

Muscular Dystrophy Patients 

Jacques P. Tremblay, Daniel Skuk, Sylvain Michel, Raynald 

Roy, Jean-Guy Lachance, Hélène Senay, Deschênes Louise, 

Goulet Marlyne, Brigitte Roy, Jean-Pierre Bouchard

112 


ABSTRACTS 




Room: 260/264 


rAAV Delivered Ribozyme Targeting Mouse Opsin to 

Rescue Vision in P23H Rats 

Marina S. Gorbatyuk, Adrian M. Timmers, Pang Jijing, 

William W. Hauswirth, Alfred S. Lewin 


Sustained Correction of Disease in Naïve and AAV2- 

Pretreated Hemophilia B Dogs Using Pseudotyped AAV 

Vectors 

Lili Wang, Roberto Calcedo, Timothy C. Nichols, Inder M. 

Verma, James M. Wilson 


Transduction Comparison of AAV Serotype Vectors in the 

Central Nervous System 

Sharon K. Reimsnider, Fredric Manfredsson, Nick Muzyczka, 

Ronald J. Mandel 


Systemic rAAV-CB-IL-10 Expression and Its Long Term 

Effects on CD4+CD25+ T Cell Populations 

Christian Muller, Sofia A. Braag, Ashley Martino, Mark A. 

Atkinson, Terence R. Flotte 


A Screen for Host Cellular Proteins That Interact with 

Adeno-Associated Virus Capsid Proteins 

Bassel Akache, Daria Glazer, Julie Park, Mark A. Kay 


Directed Evolution of AAV2 Leads to Improved Viral 

Function 

James T. Koerber, Narendra Maheshri, Brian Kaspar, David V. 

Schaffer 


Patterns of Gene Expression from In Utero Delivery of 

AAV Serotype 1 

Roberto Bilbao, Daniel P. Reay, Juan Li, Xiao Xiao, Paula R. 

Clemens 

1087 12:00 PM 

Adeno-Associated Virus-Mediated Transduction of 

VEGF165 Markedly Improves Functional Recovery of 

Infarcted Myocardium in Chronically Instrumented Dogs 

Matteo Ferrarini, Nikola Arsic, Serena Zacchigna, Fabio 

Recchia, Lorena Zentilin, Mauro Giacca, Thomas Hintze 


Adenovirus Vectors: Adaptive Immunity and Vaccines 

Room: 120/124 


Antigen Epitopes Expressed in Different Adenovirus 

Capsid Proteins Induce Different Levels of Epitope- 

Specific Immunity 

Anja Krause, Stefan Worgall, Ju H. Joh, Neil R. Hackett, Peter 

W. Roelvink, Joseph T. Bruder, Thomas J. Wickham, Imre 

Kovesdi, Ronald G. Crystal 


Examine the Cross-Reactivity of Vector-Specific T Cells 

between Human Adenoviral H5 and Simian Adenoviral 

C7 Vector in Mice 

Yan Zhi, Di Wu, Heather Jordan, Guangping Gao, James M. 

Wilson 


Adenovirus Fiber Incorporates an Immunodominant 

MHC Class I Restricted Epitope Peptide 

Jian Chen, Allan J. Zajac, Xin Xu, PingAr Yang, Qi Wu, 

Kohtaro Fujihashi, Hui-Chen Hsu, John D. Mountz 


Analysis of T-Cell Responses to Helper-Dependent 

Serotype 5-Based Capsid-Modified Adenovirus (HD.5/ 

35) or First-Generation Serotype 11- Based (Ad11) 

Vectors 

Nelson C. Di Paolo, Daniel Stone, Andre Lieber 


Adenovirus-Based Vaccine Prevents Pneumonia in 

Ferrets Challenged with the SARS Coronavirus 

Gary P. Kobinger, Thomas Rowe, Guangping Gao, Joanita M. 

Figueredo, Julio Sanmiguel, Peter Bell, Robert J. Hogan, James 

M. Wilson 


An Adenovirus-Retrotransposon Hybrid Vector Achieves 

Stable Transformation of Quiescent and Primary Human 

Somatic Cells 

Shuji Kubo, Harris S. Soifer, Marie del Carmen Seleme, John 

V. Moran, Haig H. Kazazian Jr., Noriyuki Kasahara 


A Novel Replication Competent, but Packaging Deficient 

Adenoviral(Ad) Vector [E1+, 100K-]hGAA for 

High Level hGAA Expression and Reduced Toxicity in 

Gene Therapy of Glycogen Storage Disease II (GSD-II) 

Fang Xu, Zachary Hartman, Delila Serra, Andrea Amalfitano 

1095 12:00 PM 

Silica Gel in Targeted and Controlled Viral Gene Therapy 

Mika K. Koskinen, Mervi Toriseva, Mika J. Jokinen, Harry 

Jalonen, Jukka I. Salonen, Veli-Matti Kähäri


ABSTRACTS 



Naked DNA Gene Transfer: Chromosomal Integration 

Room: 263/267 


Physical Determinants for Efficient Hydrodynamic Gene 

Delivery to Liver 

Takeshi Suda, Regis Vollmer, Dexi Liu 


Complete and Persistent Phenotypic Correction of 

Phenylketonuria in Mice by Site-Specific Genome 

Integration of Phenylalanine Hydorxylase cDNA 

Li Chen, Savio L. C. Woo 


Duration of Expression of Sleeping Beauty Transposase by 

Hydrodynamic Injection of C57/BL6 Mice 

Jason B. Bell, Elena L. Aronovich, Beth E. Larson-DeBruzzi, 

Brenda L. Koniar, Roland Gunther, Chester B. Whitley, R. 

Scott McIvor, Perry B. Hackett 


Beyond LoxP: Defining Functional Lox-Like Sequences 

for the Cre Recombinase 

Jamie E. Sheren, Elizabeth Ketner, Jarred Keith, Stephen J. 

Langer, Leslie A. Leinwand 


Site-Specific Transposon Integration 

Joseph M. Kaminski, Maragatha Vally, Todd Tenenholz, James 

B. Summers, Craig J. Coates 


Fusion Proteins Consisting of the Sleeping Beauty 

Transposase and the Polydactyl Zinc Finger Protein E2C 

Direct Transposon Integration into a Unique Human 

Chromosomal Sequence 

Stephen R. Yant, Yong Huang, Mark A. Kay 


Stable, Non-Viral Gene Transfer to Human CD34 + 

Hematopoietic Progenitor Cells Using the Sleeping 

Beauty Transposon 

Sarah Nightingale, Roger P. Hollis, Karen Pepper, Donald B. 

Kohn 

1103 12:00 PM 

Site-Specific Chromosomal Integration in Human 

Mesenchymal Stem Cells Mediated by PhiC31 Integrase 

Annahita Keravala, Juergen Hoelters, Sohail Jarrahian, Thomas 

W. Chalberg, Marisa Ciccarella, Peter Neth, Michele P. Calos 


Molecular Conjugates: Vector Engineering 

Room: 265/266 


Imaging of sec-R Directed and PEG-Stabilized Gene 

Transfer Nanoparticles in CF Mice 

Maxwell Kotlarchyk, Zhenghong Lee, Mark Cooper, Pamela B. 

Davis, Assem G. Ziady 


DQAsomes Deliver Mitochondrial Leader Peptide 

Conjugates of Plasmid and Linear Double-Stranded 

DNA into Mitochondria within Living Mammalian Cells 

Gerard G. M. D’Souza, Sarathi V. Boddapati, Robert N. 

Lightowlers, Volkmar Weissig 


Development and Testing of Ternary Peptide Copolymerized 

Gene Delivery Systems 

Changpo Chen, Ji-seon Kim, Dijie Liu, Marie McAnuff, Kevin 

G. Rice 


Cellular Internalization and Distribution of DNA 

Complexes from Cell-Adhesive Substrates 

Zain Bengali, Lonnie D. Shea 


Ad5 Penton Base Recombinant Protein: Intracellular 

Trafficking &ð Implications for Gene Delivery 

Altan Rentsendorj, Jiansong Xie, Michelle MacVeigh, Hasmik 

Agadjanian, Sarah F. Hamm-Alvarez, Lali K. Medina-Kauwe 


Single-Cell Observations of Intracellular Gene Delivery 

Particles: pH Buffering and Trafficking 

Swaroop Mishra, Rajan P. Kulkarni, Scott E. Fraser, Mark E. 

Davis 


Selective Chemical Modification on Polyethylenimine 

and Its Effects on Transfection Efficiency and Cytotoxicity 

Xiang Gao, Dexi Liu 

1111 12:00 PM 

TAT-Streptavidin: A Novel Drug Delivery Vector for the 

Intracellular Uptake of Macromolecular Cargo 

Brian Albarran, Richard To, Patrick Stayton

114 


ABSTRACTS 





Room: 261/262 


The Treatment with Intra Muscular (IM) Vascular 

Endothelial Growth Factor (VEGF) Gene Compared 

with Placebo for Patients with Diabetes Mellitus and 

Critical Limb Ischemia (CLI), a Double Blind Phase III 

Study 

Yoka H. Kusumanto, Vincent Weel, Nanno H. Mulder, Andries 

J. Smit, Jan J. Dungen, Anneke M. Hooymans, Wim J. Sluiter, 

Rene A. Tio, Paul H. Quax, Rijk O. Gans Robin P. Dullaart, 

Geke A. Hospers 


Spliceosome Mediated RNA Trans-Splicing (SMaRT) 

Strategy To Increase the Production of Human 

Apolipoprotein (ApoA1) 

M. Puttaraju, Jun Wang, Alan T. Remaley, Bryan H. Brewer, Jr, 

Gerard J. McGarrity, Mariano A. Garcia-Blanco 


Antithrombotic Effects of Endogenous Expressed 

Activated Protein C (aPC) in Murine Models 

Joerg Schuettrumpf, Alexander Schlachterman, Jianxiang Zou, 

Jianhua Liu, Christian Furlan Freguia, Valder R. Arruda 


Apo A-I and ABCA1 Gene Transfer both Increase HDL 

Cholesterol but Have Opposing Effects on Progression 

of Early Atherosclerosis in apo E Deficient Mice 

Joke Lievens, Jan Snoeys, Yingmei Feng, Désiré Collen, Bart De 

Geest 


Gene Transfert of SERCA2a Inhibits Smooth Muscle 

Proliferation and Reduce Neointima Formation after 

Balloon Injury in the Rat 

Larissa Lipskaia, Federica Del Monte, Roger J. Hajjar, Anne- 

Marie Lompre 


Gene Therapy in a Rat Model of Pulmonary Hypertension 

Stacy L. Porvasnik, Tankut Onal, Yoshi Sakai, Raquel Torres, 

Glenn A. Walter, Barry J. Byrne, Carolyn T. Spencer 


A Skin Gene Therapy Approach to Treat Hypertension 

Using Long-Term Expression and Systemic Delivery of 

Atrial Natriuretic Peptide (ANP) from the Skin 

Jean-Philippe Therrien, Christine L. Tock, Manabu Ohyama, 

Jonathan C. Vogel 

1119 12:00 PM 

Isolation of a Novel Therapeutic Angiogenic Peptide, 

AG-30, by High Throughput Screening Using the HVJ-E 

Vector 

Tomoyuki Nishikawa, Hironori Nakagami, Akito Maeda, 

Katsuto Tamai, Yasufumi Kaneda 



Connective Tissue 

Room: 230/231 


Use of Gene Activated Muscle Grafts for the Repair of 

Large Bone Defects 

Volker M. Betz, Oliver B. Betz, Ara Nazarian, Carmencita G. 

Pilapil, Mary L. Bouxsein, Louis C. Gerstenfeld, Thomas A. 

Einhorn, Mark S. Vrahas, Christopher H. Evans 


Osteogenic Growth Peptide Preferentially Enhances 

Trabecular Bone Mass 

Todd E. Meyerrose, Elisheva Smith, Jan A. Nolta, Baruch 

Frenkel 


Transgenic Vasoactive Intestinal Peptide Shows Local 

Disease Modifying Effects in a Murine Model of 

Sjögren’s Syndrome 

Beatrijs M. Lodde, Fumi Mineshiba, Jianghua Wang, Ana P. 

Cotrim, Sandra Afione, Paul P. Tak, Bruce J. Baum 


Adenovirus-Mediated Expression of Sonic Hedgehog 

Increases the Number of CD34+ Cells in the Epidermal 

Stem Cell Niche in Hair Follicles 

Howard Lou, Andrey Panteleyev, Angela M. Christiano, Ronald 

G. Crystal, Philip L. Leopold 


Correction of Glycogen Storage Disease Type II by an 

AAV Vector Encoding Highly-Secreted, Chimeric Acid 

að-glucosidase 

Baodong Sun, Talmage Brown, Andrew Bird, Ayn Schneider, 

Sarah P. Young, Alison McVie-Wylie, Y.-T. Chen, Dwight D. 

Koeberl 


AAV-Mediated Myostatin Propeptide Gene Delivery 

Results in Growth and Hypertrophy of Skeletal but Not 

Cardiac Muscles 

Chunping Qiao, Jianbin Li, Tong Zhu, Chunlian Chen, Terry 

O’Day, Jon Watchko, Juan Li, Xiao Xiao 


Further Characterization of Msx1 De-Differentiated 


Qiang Liu, Wen Liu, Yaming Wang


ABSTRACTS 


1127 12:00 PM 

Perivascular CD45 - :Sca-1 + :CD34 - Cells Are Derived 

from Bone Marrow Cells and Participate in Dystrophic 

Skeletal Muscle Regeneration 

Sheng Li, Morayma Reyes, En Kimura, Jessica Foraker, Miki 

Hagakura, Leonard Meuse, Brent Fall, Jeffrey S. Chamberlain 


Cancer - Immunotherapy: Genetic Strategies to Induce 

and Augment Anti-Tumor Immunity 

Room: 123/127 


Ad-sig-TAA/ecdCD40L Vector Prime, TAA/CD40L 

Protein Boost Vaccine Breaks Tolerance for Tumor 

Assoicated Antigens (TAA) and Suppresses Growth and 

Metastasis of Epithelial Neoplastic Cells 

Yucheng Tang, Jonathan Maynard, Xiang Ming Fang, Wei Wei 

Zhang, Albert Deisseroth 


Ad.Interferon Beta Is an Effective Treatment in an 

Orthotopic Model of Broncho-Alveolar Non-Small Cell 

Lung Cancer Via NK Cell and CD 8+ T Cell Mechanisms 

Michael J. Wilderman, Jin Sun, Anil Vachani, Eiji Suzuki, 

Dan H. Sterman, Larry R. Kaiser, Steve M. Albelda 


A Single-Component CD40-Targeted Adenoviral Vector 

for Efficient Transduction and Activation of Cutaneous 

Dendritic Cells 

Nikolay Korokhov, Sam C. Noureddini, David T. Curiel, 

Saskia J. A. M. Santegoets, Rik J. Scheper, Tanja D. de Gruijl 


Generation of Whole Cell Vaccines for Acute Myeloid 

Leukaemia by Lentivirus Mediated IL-2/CD80 Transduction 

Lucas Chan, Nicola Hardwich, Dave Darling, Joop Gäken, 

Joanna Galea-Lauri, Steve Devereux, Ghulam Mufti, Farzin 

Farzaneh 


Role of Akt in Dendritic Cell (DC) Survival and Application 

of an Akt “LIFE SWITCH” in DC-Based Tumor 

Vaccines 

Dongsu Park, Mamatha R. Seethammagari, David M. Spencer 


A Phase I Study of Antitumor Vaccination Using Genetically 

Modified Tumor Cells Expressing 

α(1,3)Galactosyltransferase in Patients with Refractory or 

Recurrent Non-Small Cell Lung Cancer (NSCLC): 

Preliminary Results 

John C. Morris, John E. Janik, Nicholas Vahanian, Leslie 

Moses, Diana O’Hagan, Lucinda Tennant, Stefania Pittaluga, 

Wendy Gao, Paul Albert, Tatiana Seregina Charles J. Link 


Intratumoral Injection of Dendritic Cells Engineered to 

Secrete Interleukin-12 by Recombinant Adenovirus in 

Patients with Metastatic Gastrointestinal Carcinomas 

Guillermo D. Mazzolini, Carlos Alfaro, Bruno Sangro, 

Esperanza Feijoo, Juan Ruiz, Alberto Benito, Inigo Tirapu, 

Ainhoa Arina, Josu Sola, Maite Herraiz Felipe Lucena, Cristina 

Olague, Jose Subtil, Jorge Quiroga, Ignacio Herrero, Belen 

Sadaba, Maurizio Bendandi, Cheng Qian, Jesus Prieto, Ignacio 

Melero 

1135 12:00 PM 

Results of a Phase I/II Clinical Trial with OncoVEXGM-CSF , 

a Second Generation Oncolytic Herpes Simplex Virus 

J. C. Hu, R. S. Coffin, C. J. Davis, N. J. Graham, N. Groves, 

P. J. Guest, K. J. Harrington, N. D. James, C. A. Love, I. 

McNeish L. C. Medley, A. Michael, C. M. Nutting, H. S. 

Pandha, C. A. Shorrock, J. Simpson, J. Steiner, N. M. Steven, 

R. C. Coombes

116 


ABSTRACT AUTHOR INDEX 

Abbott, Jinette D. 922 

Abbruzzese, James L. 82 

Abdelfattah, El Moataz 275 

Abe, Kuniya 255 

Abe, Tatsuya 1064 

Abitbol, Marc 92 

Abken, Hinrich 798 

Abraham, John M. 325 

Abraham, Roselle 381 

Ackermann, Mathias 724 

Adachi, Kumi 968 

Adachi, Yasuo 679 

Adams, April J. 448 

Addison, Christina 694 

Adler, Rima 387 

Adusumilli, Prasad S. 762 

Aebischer, Patrick 54, 953 

Afione, Sandra 1122 

Africa, Vales 976 

Agadjanian, Hasmik 211, 1108 

Agarwal, Sumit 838 

Ageyama, Naohide 833 

Agrawal, Lokesh 965, 985, 1062 

Agricola, Brian 1030 

Agudo, Judith 236 

Aguila, Alian 453 

Aguilar, Laura K. 298 

Aguilar-Cordova, Estuardo 298 

Ahmed, Atique 267, 804 

Ahn, Cheol-Hee 1060 

Ahn, Hae-Sook 278 

Ahn, Mee-Young 717 

Ahrens, Eric T. 657 

Airenne, Kari J. 97, 165, 758 

Aiuti, Alessandro 330, 331, 345 

Aizawa, Emi 23 

Akache, Bassel 1084 

Akbulut, Hakan 444 

Aker, Mari 824 

Akiba, Eiji 451 

Akita, Geoffrey Y. 628 

Akita, Hidetaka 578, 592, 746, 748 

Akiyama, Hideo 96 

Akiyama, Masaki 875, 1013 

Akkina, Ramesh 439 

Al-Abd, Ahmed M. 541 

Alakhov, Valery 784 

Al-Allaf, Faisal 394 

Al-Assah, Rana S. 327 

Albarran, Brian 1111 

Albelda, Steve M. 1129 

Albert, Paul 1133 

Albritton, Lorraine M. 479 

Aldrich, Wayne A. 849 

Alemany, Ramon 526 

Alemany-Bonastre, Ramon 553 

Alexander, Ian E. 485, 839, 988 

Alfaro, Carlos 1134 

Alferiev, Ivan S. 623, 879, 918, 930 

Alfthan, Henrik 299, 1010 

Alhava, Esko 323 

Al-Hendy, Ayman 259, 264, 552 

Alhonen, Leena 308 

Ali, Robin R. 391 

Alimoghaddam, Kamran 1049 

Aljamali, Majed N. 39, 84, 606 

Allen, Cory 720 

Allen, David G. 839 

Allen, James M. 266, 386, 513, 791, 987 

Alley, Michael C. 453 

Allocca, Mariacarmela 117 

Almeida-Porada, Graca 750 

Almqvist, Per M. 306 

Alter, Julia 788 

Alton, Eric W. F. W. 355, 356, 842 

Alvarez, Ronald D. 1029 

Alwin, Stephen 371 

Amalfitano, Andrea 407, 736, 1094 

Amar, Lahouari 1059 

Amaya, Fumimasa 643 

An, Dong Sung 95 

Anan, Mitsuhiro 1064 

Anderson, Daniel G. 777, 786 

Anderson, Jennifer M. 544 

Anderson, Joseph 439 

Anderson, Patrick N. 391 

Anderson, Scott C. 531 

Anderson, Wayne B. 750 

Ando, Dale 43, 974 

Andrade, Christian M. 662 

Andre, Franck M. 188 

Andreeff, Michael 447, 727 

Anguela, Xavier M. 901 

Annoni, Andrea 468 

Anover, S. 341 

Ansari, Aslam 318 

Anson, Donald S. 360, 491 

Anton, Eva 752 

Anwer, Khursheed 227, 279 

Aoki, Chieko 679 

Aoki, Kazunori 708 

Aoyama, Takuma 46, 50 

Appelt, Uwe 1034 

Apperley, Jane 796 

Aquirre, Jennifer E. 936 

Araújo, Alexandra P. Q. C. 970 

Arakawa-Hoyt, Janice 619, 926 

Araki, Kimi 255 

Arbetman, Alejandra 130 

Arbos, Anna 248 

Arceo, Toni Rose 960 

Arena, Sabrina 373 

Arfi, Audrey 233 

Ariazi, Eric A. 204 

Ariga, Tadashi 35 

Arii, Shigeki 489 

Arina, Ainhoa 1134 

Arkin, Lisa A. 969 

Armeanu, Sorin 840 

Armentano, D. 245 

Arnold, Pizzey 711 

Aronovich, Elena L. 244, 1098 

Arpke, Robert 778 

Arroyo, Trini 311 

Arruda, Valder R. 84, 603, 609, 1114 

Arsenault, Jody R. 936 

Arsenijevic, Yvan 667 

Arsic, Nikola 114, 121, 1087 

Art, Riggs 86 

Arya, Suresh 476 

Asada, Kiyozo 486 

Asano, Shigetaka 103 

Aslan, Hadi 184 

Aslanidi, George 400, 418 

Ast, Oliver 170, 172, 958 

Astrakhan, A. 341 

Athanasopoulos, Takis 258, 261 

Atkinson, John P. 137 

Atkinson, Mark A. 914, 1083 

Attra, Heather 178 

Aubert, Dominique 896 

Aubourg, Patrick 429 

Augustus, Sheldon 379 

Auricchio, Alberto 117, 240, 250, 

435, 669 

Ausar, S. Fernando 561 

Austin, M. 950 

Auyeung, Vincent C. 95 

Ave, Patrick 417 

Avenarius, Matthew 747 

Awedikian, Rafi 512 

Ayala, John P. 367 

Ayuso, Eduard 236 

Azam, Selina 105



Azzouz, Mimoun 55 

Bacher, John D. 458 

Bae, Kyung-Hee 1009 

Baek, Hyun-Jung 161 

Bagley, Jessamyn 1033 

Bahner, Ingrid 987, 998 

Bai, Qing 161 

Bai, Yuan Son 472 

Bai, Yuansong 103 

Baila, Stefano 603 

Bailey, Kent R. 47 

Baker, Andrew H. 620, 811, 851, 1057 

Baker, Carl C. 293 

Baker, Henry J. 962 

Baker, Kim 665 

Balaban, Alexandru T. 588 

Balaggan, Kamaljit S. 391 

Baliulis, Giedrius 630 

Ball, C. 1055 

Balla, Sunita 764 

Ballabio, Andrea 250, 435 

Ballas, Christopher 492 

Baltimore, David 95 

Bamber, Jeff 484 

Banasik, Marisa 100 

Banerjee, Sanjeev 691 

Bangari, Dinesh S. 152 

Bankiewicz, Krys S. 124, 658, 942 

Baraceros, Fe 989 

Barajas, Miguel 547 

Barbas, III, Carlos F. 371, 973, 1001 

Barbon, Christine M. 245, 249, 910 

Barcena, Alicia 619, 926 

Barcia, Carlos 430, 745, 946 

Barcia-Gonzalez, Carlos 143 

Bardelli, Alberto 373 

Bardenheuer, Walter 309 

Barkats, Martine 92 

Barnes, Mack N. 1029 

Barquinero, Jordi 432 

Barratt-Boyes, Simon 66 

Barrera-Saldana, Hugo A. 298, 553 

Barrio, Jorge R. 808 

Barry, Michael A. 24, 29, 415 

Bartido, Shirley M. 558 

Bartlett, David L. 755 

Bartlett, Derek W. 70, 208 

Bartlett, Jeffrey S. 72, 74, 113, 509, 848 

Barton, Kenneth N. 153 

Bartsevich, Victor V. 1072 

Bartus, Raymond T. 947, 950 

Barzon, Luisa 536 

Bash, Ryan 822 

Bass, Caroline 859 

Bates, Matthew A. 292 

Bates, Susan 453 

Battaglia, Anna 648 

Battaglia, Manuela 468 

Battista, Sabrina 683 

Battmer, Karin 368 

Bauer, Gerhard 288, 810 

Bauer, Johann 1065 

Bauer, Jr., Thomas R. 458 

Baum, Bruce J. 466, 471, 520, 672, 

877, 1122 

Baum, Christopher 829 

Bauzon, Maxine 319, 958, 959 

Bazan-Peregrino, M. 1027 

Beagles, Karen E. 440, 455 

Beard, Brian C. 5, 440, 455, 456 

Beaudet, Arthur L. 535, 607, 815, 904, 

905, 906 

Beausejour, Christian 337, 346, 974 

Beech, Derrick J. 296 

Beerens, Antoine M. 753 

Behr, Jean Paul 583 

Belcher, Angela 231 

Bell, Jason B. 176, 1098 

Bell, Peter 112, 506, 1092 

Belladonna, Maria L. 698 

Bellinger, Dwight A. 32 

Bellodi-Privato, Marta 896 

Bellu, Anna Rita 408 

Belmonte, Nathalie 446 

Belur, Lalitha 244 

Bemelmans, Alexis-Pierre 667 

Benabdallah, Basma F. 102, 690 

Benatuil, Lorenzo 109, 737 

Benchoua, Alexandra 953 

Bendandi, Maurizio 1134 

Bengali, Zain 204, 769, 1107 

Benihoud, Karim 524 

Benito, Alberto 1134 

Benjamin, Reuben 711 

Bennett, Jean 117, 135 

Bennett, Ryan 1046 

Bennicelli, Jeannette L. 135 

Benraiss, Abdellatif 429 

Bercury, S. D. 245, 249 

Berger, Gideon O. 596 

Bergert, Elizabeth R. 1014 

Berges, Bradford K. 157 

Bergqvist, David 630 

Bergvall, Peder 173 

Berk, Arnold J. 874, 890 

Bernal, Jan 658 

Bernard, Robert 559 

Bernardi, Massimo 819 

Bernshausen, Alexandra 532 

Bernt, Kathrin 145 

Berraondo, Pedro 547 

Berthoux, Lionel 2 

Bertolotti, Roger 383 

Bertoncello, Ivan 967 

Bertoni, Carmen 262, 265, 622, 789 

Betz, Oliver B. 889, 1120 

Betz, Volker M. 889, 1120 

Beutelspacher, Sven C. 1042 

Biagi, Ettore 801 

Bian, Jing 917 

Bielawaska, Alicja 307, 450, 1011 

Bielawaska, Jacek 307, 1011 

Bieler, Kurt 764 

Bienemann, Alison S. 656, 955 

Biessen, Erik 413 

Biffi, Alessandra 426, 972 

Bigger, Brian W. 238, 394 

Bikram, Malavosklish 681, 892 

Bilbao, Roberto 1086 

Binder, Gwendolyn K. 62, 477, 

990, 1075 

Binkley, Erin 670 

Binley, Katie 96 

Bird, Andrew 897, 912, 1124 

Birraux, Jacques 895 

Bishop, K. 950 

Bitzer, Michael 840 

Bjorkegren, Emma 31 

Black, Esther 736 

Black, Keith L. 692, 698 

Blackburn, Mandy 422 

Blanchard, Karin 438 

Blanco, Patricia V. 78 

Blankinship, Michael J. 266, 791 

Blau, C. Anthony 465 

Blau, Helen 467 

Blechacz, Boris R. A. 721 

Blissard, Gary W. 1 

Blits, Bas 662 

Blomberg, Pontus 630, 924 

Blomley, Martin 356 

Bloom, David C. 861 

Blumenthal, Martina 805 

Boan, Jose F. 808 

Bobik, Thomas A. 115

118 



Bocangel, Dora 287, 809 

Bochner, Bernard H. 710 

Bockmann, Miriam 310 

Boddapati, Sarathi V. 1105 

Bogan, Janet 792 

Bohn, Kimberly 6 

Bohn, Martha C. 124, 944 

Bohne, Jens 829 

Boling, Susan 316 

Bollard, Catherine M. 795, 1074 

Bonde, Jesper 212, 783, 1035 

Bonifacino, Aylin 737 

Bonini, Chiara 819 

Bonnel, Sebastien 92 

Bonnin, Delphine 429 

Bonyhadi, Mark 288, 707, 709, 

739, 810 

Boquet, Michael P. 142, 148, 412 

Bordignon, Claudio 331, 426, 819, 972 

Bortolanza, Sergia 555 

Bosch, Fatima 236, 248, 901, 1070 

Bosh, Pablo 892 

Bossow, Sascha 840 

Bosticardo, Marita 333, 349 

Bote, Alex 432 

Both, Gerald W. 886 

Bouchard, Jean-Pierre 1079 

Bouchentouf, Manaf 102, 690 

Boucher, Paul D. 807 

Boudreaux, Mary K. 340 

Bou-Gharios, George 788 

Boulis, Nicholas M. 113, 433, 482, 

855, 1050 

Bourget, Mylene 256 

Bouxsein, Mary L. 1120 

Bouxsein, Nathan F. 771 

Bovolenta, Chiara 64 

Bowers, William J. 431 

Bowman, Amy M. 893 

Bowman, Katherine 200, 779 

Bowman, Keith 438 

Boyd, Anita 839 

Boyd, Marie 1044 

Boyer, Jean 575, 990 

Boyer, Julie L. 65, 67, 853 

Boyer, Olivier 946 

Braag, Sofia A. 1083 

Brachman, Erin 375, 385 

Braeckmans, Kevin 580 

Bragadottir, Ragnheidur 434 

Brambilla, Riccardo 426 

Brantly, Mark L. 1077 

Braun, S. E. 62 

Breakefield, Xandra O. 76, 164 

Breen, Wendy 438 

Bregni, Marco 819 

Brennan, Karen M. 910 

Brenner, Malcolm K. 795, 798, 801, 

1074 

Brenner, Michael 543 

Brenner, Sebastian 234 

Brentjens, Renier J. 707, 799 

Bresler, Tal 931 

Breul, Astrid 565 

Brewer, Jr, Bryan H. 1113 

Breymann, Laura M. 283 

Bridges, Charles R. 42 

Briggs, Simon S. 197 

Brignetti, Dominic 881 

Brill-Almon, Einat 342 

Bringas, John 942 

Britton, Nicola 851 

Brody, Steven 357 

Broekman, Marike 858, 866, 961 

Brooks, Alan R. 768, 958, 959, 960 

Brooks, David C. 734 

Brossart, Peter 840 

Brough, Douglas E. 60, 664, 875, 1013 

Brouillet, Emmanuel 953 

Brouwer, Eric 1005 

Brown, Brian D. 36 

Brown, Jennifer L. 887 

Brown, Patricia A. 673 

Brown, Rhonda 887 

Brown, Stephen L. 153 

Brown, Talmage 912, 1124 

Bruder, Joseph T. 975, 989, 1088 

Brunbeck, Julie 561 

Brunetti-Pierri, Nicola 607, 815, 903, 

904, 905, 906 

Brunhoeber, Elaine 227 

Brunn, Gregory 449 

Bruscia, Emanuela 568, 651 

Brzezinski, Margaret 459 

Bu, Jie 110, 427 

Buch, Mamta 238 

Buch, Prateek 391 

Buchholz, Christian J. 732, 846 

Buchsbaum, Donald J. 529 

Buck, Charles 396 

Buckley, Shannon 338 

Buckley, Suzanne M. K. 394 

Buddhikot, Madhavee 232 

Bueler, Hansruedi 939 

Buening, Hildegard 502, 863 

Bueno Lopez, Carlos A. 183 

Bueren, Juan 236 

Bühler, Bernd 973 

Buning, Hildegard 851 

Burger, Corinna 59 

Burgess, Angela P. H. 533 

Burkholder, Tanya 458 

Burkman, James M. 42 

Burnight, Erin R. 1 

Burstein, Haim 677 

Burton, Dennis 984 

Burton, Mark A. 26 

Burzynski, Joseph 561, 575 

Bush, Nigel 484 

Bushman, Frederic 990, 1075 

Butler, Brian E. 286, 298, 695 

Buza, Elizabeth 795, 1074 

Byrne, Barry J. 44, 354, 400, 514, 818, 

909, 911, 1077, 1117 

Byrnes, Andrew P. 144 

Byun, Jonghoe 625, 633, 1052 

Cabaniols, Jean-Pierre 417, 902 

Cáceres, Javier F. 534 

Cafferata, Eduardo A. 78 

Cai, Kexia 352 

Cai, Shanbao 474 

Caillaud, Catherine 233 

Calcedo, Roberto 139, 404, 500, 

506, 947, 1081 

Callahan, Shellie M. 142, 148, 412, 414 

Callan, Mary Beth 606 

Calos, Michele P. 194, 195, 257, 

265, 392, 622, 770, 

821, 884, 1103 

Camire, Rodney M. 39 

Campbell, Kevin 44 

Campbell-Thompson, Martha 400, 418 

Campo, Joseph J. 989 

Campochiaro, Peter A. 96 

Campos, Samuel K. 29 

Candolfi, Marianela 143 

Candotti, Fabio 35, 333, 341, 349 

Cao, Chuanhai 693 

Cao, O. 419 

Cao, Xiaobo X. 301 

Capitani, Ombretta 683 

Capotondo, Alessia 426 

Carbonaro, Denise A. 37, 805 

Cardo, Carlos C. 710 

Cardone, Monica 250 

Cardwell, Lanise 1039



Carette, Jan E. 526 

Carey, Dee 815, 904, 906 

Carey, Michael 1008, 1051 

Carlson, Corey M. 176 

Carlsson, Fredrik 630 

Carlsson, Thomas 941 

Carlucci, Filippo 331 

Carnes, Aaron 560 

Carrabino, Salvatore 358 

Carrer, Alessandro 114 

Carroll, Richard 990 

Carrondo, Manuel 1070 

Carrum, George 795, 1074 

Carter, Barrie 116 

Carthy, Janine M. 55 

Cartier, Nathalie 429 

Casadevall, Nicole 342 

Casanova, Jean-Laurent 349 

Casciola-Rosen, Livia A. 672 

Case, Casey C. 43, 1072 

Casey, Ann E. 431 

Cashion, Linda 170, 172, 715, 

958, 959, 960 

Cashman, Siobhan 670 

Cass, Brian 518 

Castellani, Stefano 358 

Castillo, Mireia 432 

Castillo, Pablo J. J. 696 

Castro, Maria G. 143, 269, 273, 

430, 745, 946 

Catchpole, Ian R. 228 

Cathomen, Toni 371 

Caton, David 1036 

Cattaneo, Roberto 106, 720, 732, 

803, 843, 844 

Cavanagh, Heather M. A. 26 

Cearley, Cassia N. 856 

Cecilia, Marini 121 

Cellerino, Alessandro 435 

Certoux, Jean Marie 739 

Cerullo, Vincenzo 598, 885 

Cervenak, Judith 1045 

Cesana, Daniela 389 

Chabardes, Stephan 433 

Chada, Sunil 287, 290, 691, 809 

Chadeuf, Gilliane 512 

Chae, Soo Yeong 827 

Chaki, Shinji 587 

Chalberg, Thomas W. 194, 770, 1103 

Chamberlain, Jeffrey S. 266, 513, 787, 

791, 793, 1040, 1127 

Champion, Hunter C. 46 

Chan, Cygni (Howie) 864 

Chan, Diane 335 

Chan, Lawrence 638 

Chan, Lucas 1131 

Chan, Mei-Ki 762 

Chan, Rebecca J. 374 

Chandler, Simon 43 

Chandra, Saurabh 829 

Chang, Alex H. 604 

Chang, Jeff 1074 

Chang, Jin Woo 122 

Chang, Lung-Ji 823 

Chang, Patricia L. 220 

Chang, Tan 832 

Changqing, Su 705 

Chao, Hsu 638 

Chapdelaine, Pierre 257 

Charron, Catherine E. 422, 423 

Chartier, Cecile 21, 882, 959 

Chatterjee, Saswati 19, 402 

Chattopadhyay, Munmun 642, 671 

Chaturvedi, Anuhar 368 

Chau, David 718 

Chaudhuri, Tandra R. 448 

Chavez, Raymond A. 644, 645 

Cheetham, Michael E. 955 

Chemaly, Elie R. 45 

Chen, C. 62 

Chen, Changpo 198, 1106 

Chen, Chien-Lun 289 

Chen, Chris T. 674 

Chen, Christopher 477 

Chen, Chunlian 398, 916, 1125 

Chen, Dung T. 1004 

Chen, Eleanor 445 

Chen, Irvin S. Y. 95 

Chen, Jaiwei 629, 937 

Chen, Jian 140, 608, 612, 991, 1090 

Chen, Kai-Yun 304 

Chen, Li 1097 

Chen, Linping 1045 

Chen, Liyuan 11 

Chen, Ming 361 

Chen, Minghan 103 

Chen, Qin 647 

Chen, Si-Yi 285 

Chen, Steven C. 611 

Chen, Y.-T. 912, 1124 

Chen, Yong 196 

Chen, Yong Hong 420 

Chen, Zhaoxia 86 

Chen, Zhi-Ying 557, 563 

Cheng, Joseph 147 

Cheng, Kin-Yuen 280 

Cheng, Pi-Wan 778 

Cheng, Qian 555 

Cheng, S. H. 245, 249 

Cheng, Seng H. 497, 628, 910 

Cheng, Shi-Yuan 304 

Cheng, Wing-Shing 1024, 1025 

Chernajovsky, Yuti 78 

Cherry, Simon R. 743 

Cheung, Judy 913 

Cheung, Wing T. 564 

Chiang, Yawen L. 571, 925, 928 

Chien, Kenneth 41 

Chinje, Edwin C. 327 

Chinnasamy, Dhanalakshmi 276 

Chinnasamy, Nachimuthu 276 

Chiocca, Antonio E. 283 

Chiorini, John A. 396, 523 

Chipchase, Daniel 395 

Chiuchiolo, Maria 67 

Chmielnicki, Eva 51 

Ch’ng, Edwin 899 

Cho, Byoung Chul 702 

Cho, Dae Hyun 827 

Cho, Hongchan 278 

Cho, Seok-Goo 103 

Cho, Sung-Ha 717 

Cho, Sung-Rae 51 

Cho, Young-Hwa 717 

Choi, Christopher 462 

Choi, Hye Jin 541 

Choi, Il-Kyu 1019 

Choi, Jin-Ho 625 

Choi, Joon Sig 202 

Choi, Kyung-Ju 541, 702 

Choi, Uimook 234, 1039 

Choi, Vivian W. 18, 343, 403 

Choi, Yoon Hee 122 

Choi, Young-Kook 511, 514 

Chong, Heung 267, 704 

Chono, Hideto 486 

Chorny, Michael 623, 879, 918, 

919, 930 

Chou, Ting-Chao 762 

Chou, Wayne 823 

Chow, Samson A. 393 

Christensen, Jeffrey 459 

Christian, Lindsey 650 

Christiano, Angela M. 1123 

Christman, Gregory 259

120 



Christoferson, Laura 864 

Chu, Qiuming 531 

Chuah, Marinee 40 

Chuang, Wei-Lien 910 

Chung, Daniel C. 135 

Chung, Ji Hyung 633 

Churchill, Larry R. 1078 

Ciapetti, Gabriela 683 

Ciccarella, Marisa 1103 

Ciceri, Fabio 819 

Cichutek, Klaus 7, 475, 836, 

846, 1070 

Cingolani, Eugenio 381 

Cioffi, William 904 

Citkowicz, Andrzej 170, 172 

Clackson, Tim 466 

Clague, Hodgson 560 

Clapp, D. Wade 470 

Clark, Erin A. 148, 412 

Clark, K. Reed 986 

Clark, Karl J. 192, 347 

Clarke, Christian 149, 598, 903 

Clarke, Jennifer 110, 497 

Clarke, Katherine 1047 

Clemens, Paula R. 1086 

Clement, Nathalie 20 

Cmielewski, Patricia 360 

Coates, Allan L. 365 

Coates, Craig J. 1100 

Cockrell, Adam S. 478, 822 

Coffey, Matt 173 

Coffin, R. S. 1135 

Cohen, Edward P. 697 

Cohen, Justus B. 161, 162 

Cohen-Haguenauer, Odile Y. D. 1070 

Colbern, Gail 311 

Cole, Caroline 271, 449 

Coll, Jean Luc 583 

Colledge, William H. 228 

Collen, Désiré 40, 413, 530, 

548, 636, 1115 

Collie, David 842 

Collier, Lara S. 176 

Collingwood, Trevor 1072 

Colosi, Peter 130, 850 

Combee, Lynn A. 134 

Comer, Laryssa A. 866, 961 

Compos, Bradley 646 

Condiotti, Reba 837 

Condliffe, Steve 817 

Conese, Massimo 358 

Conlon, Thomas J. 115, 134, 1077 

Connault, Elisabeth 524 

Connole, M. 62 

Connor, Robert J. 544 

Contag, Christopher H. 195, 756 

Converse, Andrea D. 826 

Conwell, Christine C. 203, 776 

Cool, Robbert H. 1073 

Coombes, R. C. 1135 

Coon, Rob G. 378 

Cooney, Ronan J. 634 

Cooper, Mario 132 

Cooper, Mark J. 655, 668, 1104 

Coppel, Ross 988 

Copreni, Elena 358 

Cornetta, Kenneth 2, 94, 492, 

494, 825 

Cortese, Katia 435 

Cortez-Gonzales, Xocthil 179 

Cosma, Maria Pia 250 

Cossette, Travis L. 115 

Costa, Fabricio F. 866, 858, 961 

Cotrim, Ana P. 471, 1122 

Cotugno, Gabriella 435, 669 

Coulibaly, Cheick 475 

Cournil-Henrionnet, Christel O. 188 

Coutelle, Charles 209, 394 

Couto, Linda B. 437, 443, 997, 1000 

Covarrubias, Luis F. 995 

Cowan, Morton J. 338 

Cowen, Rachel L. 327 

Cox, Nancy R. 962 

Cox, Peter 365 

Craft, Timothy P. 460, 1031 

Cranston, Aaron N. 310 

Crawford, Gregory E. 333 

Crawford, Keith 889 

Creer, Michael H. 1032, 1037 

Cresawn, Kerry O. 354, 909, 911 

Cress, Dean E. 1063 

Crettaz, Julien S. 547 

Crews, Leslie 56 

Cristiano, Richard J. 780 

Crompton, Mark 258 

Cross, Daisy P. 864 

Croyle, Maria A. 142, 148, 412, 414 

Crumine, Jennette 848 

Cruz, Pedro E. 644 

Cruz, Yolmari 699 

Crystal, Ronald G. 65, 67, 147, 335, 

367, 427, 508, 660, 

812, 814, 847, 853, 

969, 975, 994, 1088, 1123 

Cucchiarini, Magali 120, 522, 676 

Cullen, Karyn 240, 602 

Cummins, James H. 920 

Cunningham, J. 950 

Cunningham, Janet 658, 942 

Cunningham, Sara M. 437, 443, 997, 

1000 

Curiel, David T. 25, 28, 30, 140, 

299, 314, 316, 322, 328, 

528, 537, 540, 542, 679, 

811, 873, 883, 886, 1007, 

1017, 1018, 1020, 1026, 

1029, 1130 

Curran, Michael A. 837 

Curtin, James F. 143, 269, 273, 946 

Curtis, Kristopher M. 996 

Custard, Emily J. 599 

Cuzange, Alain 583 

Daadi, Marcel M. 942 

Daddario, Kathleen M. 996 

Dai, Hui 196 

Dale, David 459 

Dall, Peter 1018 

Dam, Wendy A. 189 

Dambach, Megan J. 159 

Damodaran, Tirupapuliyur 912 

Daniel, Jonathan 301 

Daniels, Gregory 267 

Dankó, István 187, 567, 765 

Danos, Olivier 261, 790, 794, 820 

Dao, Mo A. 457 

Dapas, Marina 126 

Darai, Gholamreza 843 

Darling, Dave 1131 

Darnell, Jeffrey 181, 574 

Dass, Biplob 947 

Date, Taro 628 

Daud, Adil 699 

Daudi, Nili 735 

Davern, Timothy J. 501 

Daviau, Judy 1043 

David, Michael 272 

Davidoff, Andrew M. 118, 711 

Davidson, Beverly L. 52, 420, 427, 

601, 656, 816 

Davie, Earl W. 611 

Davies, Graham 31 

Davies, Lee A. 351, 362 

Davies, William R. 47 

Davis, Adam 113 

Davis, Alan R. 286, 681, 892 

Davis, Arlene M. 1078



Davis, C. J. 1135 

Davis, John J. 539 

Davis, Mark E. 70, 208, 1109 

Davis, Pamela B. 201, 1104 

Davoust, Jean 790 

Davydova, Julia 1020 

Day, Denise M. 55 

Day, Terry 450 

D’Azzo, Alessandra 250 

De, Bishnu P. 367, 508, 812, 847 

De Felici, Massimo 568 

De Geest, Bart 413, 530, 548, 

636, 1115 

De Grand, Alec M. 49 

de Gruijl, Tanja D. 1130 

de Haan, Xander A. M. 9 

de Jong, Albertine 907 

De Palma, Michele 446 

De Smedt, Stefaan C. 580, 594, 774 

de Vos, John 739 

De Vrij, Jeroen 878 

Dean, David A. 372, 570, 572, 767 

DeBaets, Marc H. 189 

Deckert, Martina 77 

DeDeyn, Peter 429 

DeGiulio, James V. 572 

Deglon, Nicole 953, 1070 

Deichmann, Annette 33, 1076 

Deisseroth, Albert 444, 1128 

Del Carro, Ubaldo 426, 972 

del Monte, Federica 45, 48, 850, 1116 

Delgado, Antonio 519 

Delgado-Enciso, Ivan 553 

Dembinski, Jennifer 447, 727 

Demeester, Joseph 580, 594, 774 

Demeneix, Barbara 1070 

Demorest, Zachary L. 445 

Denes, Bela 155 

Deng, Hui 75 

Deng, Xiaoli 637 

Denovan-Wright, Eileen 53, 952, 954 

Deol, Jatinderpal R. 256 

DePhillips, Peter A. 893 

DeRuisseau, Lara R. 354, 818 

Descamps, Delphyne 524 

Deschamps, Marina 739 

Desfossé, Laurie 884 

Desmond, Renee A. 1004, 1028 

Desnick, R. J. 249 

Devaux, Patricia 106 

Devereux, Steve 1131 

Dewhurst, Stephen 533 

Di Giorgio, Christophe 591 

Di Noto, Rosa 683 

Di Paolo, Nelson C. 151, 980, 1091 

Di Pasquale, Giovanni 523 

Di Vicino, Umberto 435 

Diaz, Rosa Maria 267, 449, 723, 

802, 804 

Diaz-Nido, Javier 956 

Dickinson, Brent 438 

Dickson, George J. 258, 261, 1070 

Dighe, Niraja 394 

Dijkhuizen, Paul 907 

Dilling, Christine M. 892 

Dimmock, David 815 

Ding, Qingqing 82 

Ding, Qiulan 605 

Ding, Wei 399, 521 

Ding, Zhaobing 898 

Dingli, David 297 

DiPasquale, Janet 293 

DiPersio, John F. 288, 709, 810 

Dirven, Clemens M. F. 537 

Dmitriev, Igor P. 873 

Do, H. 245 

Dobois, Ludwig 329 

Dobrzynski, E. 419 

Dodge, James C. 110, 427, 497 

Doglioni, Claudio 389 

Doig, Andrew 178, 182 

Domansky, Karel 205 

Domenici, Luciano 117, 435 

Dominiczak, Anna F. 1057 

Donahue, Robert E. 387, 737 

Dong, Ai-wen 722 

Dong, John (Jian-yun) Y. 1058 

Dong, Xiao Yan 127 

Dong, Xiaoyan 108 

Dong, Zheng 393 

Dong, Zhizhong 939 

Donis, Ruben O. 66 

Donsante, Anthony 237, 424 

Doolan, Denise L. 989 

Doria, Monica 240 

Dorigo, Oliver 874, 890 

Doroudi, Roya 309 

Dotti, Gianpietro 798, 801 

Douar, Anne M. 794, 901 

Douglas, Joanne T. 30, 886 

Down, Julian D. 738 

Doyonnas, Regis 467 

Draghia-Akli, Ruxandra 190, 673, 763 

Dranitzki-Elhale, Michal 342 

Dropulic, Boro 62, 990, 1075 

Drosten, Matthias 310 

Drury, Miya 375 

Drvota, V. 924 

D’Souza, Gerard G. M. 1105 

Du, Lily M. 340 

Du, Xiangying 200 

Du, Xiaohan 984 

Duan, Dongsheng 515, 688, 793 

Duan, Hongzhe 12, 464 

Duan, Maoli 666 

Duan, Yuyou 99 

Duchateau, Philippe 417, 902 

Duda, Jennifer 195 

Duffy, Aoife 550 

Duffy, Lisa 825 

Dullaart, Robin P. 1112 

Dumont, Marcel 690 

Dunbar, Cynthia E. 387, 737 

Dungen, Jan J. 1112 

Duns, Gerben 314 

Dupré, Loïc 330, 345 

Durán, Yanaí 391 

Durocher, Yves 518 

Dutheil, Nathalie 20 

Dutzar, Benjamin 116 

Duvshani-Eshet, Maayan 569 

Düzgünes, Nejat 590 

Dwyer, Roisin M. 1014 

Dyke, Jonathan P. 969 

Dzojic, Helena 1024, 1025 

Eades, Bill 288 

Easter, Michele E. 1078 

Eberling, Jamie L. 942 

Ecay, Margarita 320 

Economides, Aris 51 

Eder, Matthias 368 

Eduardo, Martinez-Anso 976 

Efferson, Clay 691 

Effertz, Karin 371 

Ehrhardt, Anja 22, 441, 600, 821, 915 

Ehsani, Ali 86 

Eiden, Maribeth 750 

Einfeld, David A. 875, 1013 

Einhorn, Thomas A. 1120 

Eisenberg, David P. 762 

Eixarch, Herena 432 

Ekker, Stephen C. 445, 1068 

Ekstrom, Tomas J. 306 

El-Banna, Mukarram 427 

Eliopoulos, Nicoletta 334

122 



Ellefsen, Barry 559 

Ellis, James 79 

Elojeimy, Saeed N. 307, 450, 1011 

El-Zawahry, Ahmed M. 307, 324, 

450, 1011 

Embury, Jennifer E. 423 

Emery, David W. 80, 81, 388, 390, 824 

Enback, Juulia 38 

Endell, Jan 502, 863 

Eng, F. 62 

Engelhardt, John F. 116, 399, 498, 

499, 521, 793 

Engler, Heidrun 544 

Engler, Jeffrey A. 600 

Engstrom, Julia 375, 1056 

Epperly, Michael W. 729, 1069 

Erbacher, Patrick 583 

Erger, Kirsten E. 115, 134 

Eriksen, Jens 1066 

Erne, Megan 132 

Ernstberger, Aaron G. 474 

Esaki, Masayasu 929 

Esapa, Margaret 96 

Espejo, Carmen 432 

Esposito, Maria Teresa 683 

Essand, Magnus 1024, 1025 

Esselin, Stéphanie 524 

Essner, Jeffrey J. 192, 347 

Esteban-Maria, Jacinto 298 

Eto, Yoshikatsu 993 

Ettyreddy, Damodar 989 

Evangelista, Monica 370 

Evans, Christopher H. 674, 675, 

835, 889, 1120 

Evans, Heather M. 771 

Evans-Galea, Marguerite V. 831 

Everett, Ruth S. 359, 407, 736 

Everts, Maaike 540, 883, 1017, 1018 

Ewert, Kai 771 

Excoffon, Katherine J. D. A. 747 

Ezell, Ed L. 588 

Fabb, Stewart 261 

Fabio, Karine 591 

Fabre, Sylvie 678 

Fabrizio, Benedicenti 389 

Fairbairn, Leslie J. 1044, 1047 

Fairbanks, Lynette D. 31 

Fall, Brent 787, 1127 

Fan, SheungTat 712 

Fang, Bingliang 539 

Fang, Jennifer 205 

Fang, Juan 340 

Fang, Xiang Ming 1128 

Farese, Anne M. 234 

Farinati, Fabio 555 

Farjo, Rafal 668 

Farley, Daniel C. 481 

Farley, Ray 356 

Farrow, P. 1027 

Farson, Deborah 881 

Farzaneh, Farzin 305, 1131 

Fasano, Stefania 426 

Fattal, Elias 229 

Faure, Emmanuelle 982 

Fazio, Vito Michele 706 

Federici, Thais 433, 482, 855, 1050 

Federico, Maurizio 64 

Federoff, Howard J. 431, 966 

Federspiel, Mark J. 47, 496, 721 

Feijoo, Esperanza 1134 

Feld, Yair 931 

Feldmann, Frederik 141 

Feldmann, Heinz 141 

Feliu, Juan E. 248 

Fellenberg, Kurt 1034 

Felmet, Kelly 161 

Feng, Yingmei 548, 636, 1115 

Fengler, Sabine 836 

Ferguson, Cole 387 

Fernandes, Alda 403 

Fernandez, Juliet L. 76 

Fernando, Rotellar 976 

Ferrand, Christophe 739 

Ferrara, Luciana 375, 384, 1056 

Ferrari, Giuliana 83 

Ferrari, Stefano 356 

Ferrarini, Matteo 1087 

Ferry, Nicolas 896 

Ferstl, Peter 519 

Fewell, Jason 227, 279 

Fielding, Adele K. 292, 803 

Fiette, Laurence 417 

Figueredo, Joanita M. 112, 139, 146, 

500, 1092 

Filareto, Antonio 568 

Fincati, Karina 536 

Finegold, Milton 149, 535, 815, 

904, 905, 906 

Fink, David J. 640, 642, 671 

Finn, Eric E. 513 

Finocchiaro, Liliana M. E. 696 

Fioretti, Daniela 706 

Fischer, Anthony J. 816 

Fischer, Horst 913 

Fischer, Kimberlee M. 973 

Fischer, Yvonne 77 

Fishbein, Ilia 623, 879, 918, 919 

Fisher, K. 1027 

Fiszman, Gabriel L. 696 

Fiszman, Marc Y. 934 

Fleckenstein, Katharina 326 

Fleming, Jane 485 

Fletcher, Bradley S. 34 

Fletcher, Misty D. 462 

Fletcher-Turner, Anita 655 

Flores-Gutierrez, Juan P. 298 

Flotte, Terence R. 115, 134, 644, 

654, 1077, 1083 

Fodor, Istvan 155 

Fodor, Nadia 155 

Fojo, Tito 453 

Follenzi, Antonia 331, 468 

Fong, Yuman 762 

Fontana, Elizabeth J. 283 

Foraker, Jessica 1127 

Forman, Daron 469, 1033 

Forman, Stephen J. 19, 402 

Forsayeth, John R. 658, 942 

Forshaw, Michael 271, 723 

Fortes, Puri 759 

Forudi, Farhad 917 

Foster, Aaron E. 800, 979 

Foster, Elyse 933 

Foster, Helen 261 

Fouletier-Dilling, Christine 681 

Foust, Kevin D. 654 

Fowler, Kerry J. 967 

Fradette, Julie 156 

Fraefel, Cornel 724 

Fraites, Jr., Thomas J. 818, 909 

François, Achille 512 

François, Moïra 312, 334 

Frandsen, Joel L. 338, 908, 1068 

Frangioni, John V. 49 

Frank, Michael M. 407 

Fraser, Justin 867, 940 

Fraser, Nigel W. 157 

Fraser, Scott E. 1109 

Fratantoni, Joseph C. 192 

Frederik, Peter 548 

Freeman, Arnold I. 806 

Freeman, Gregory L. 617 

Freese, Andrew B. 445 

Frenkel, Baruch 1121 

Freund, Christian T. F. 286 

Freund, Olivier 583



Freyschuss, A. 924 

Freytag, Svend O. 153 

Fridberger, Anders 666 

Friedman, Gary 918, 919 

Friedman, Peter 641 

Friedmann, Theodore 186, 241 

Friz, Jennifer L. 1063 

Frizzell, Raymond A. 817 

Frndova, Helena 365 

Fromes, Yves Y. 229, 934 

Frost, Lindsay 650 

Fruehauf, Stefan 131, 326, 865, 1034 

Fu, Haiyan 235, 425, 854 

Fuess, Sally 504, 516 

Fujihashi, Kohtaro 991, 1090 

Fujiki, Minoru 1064 

Fujimuro, Masahiro 68 

Fujino, Masayuki 263 

Fujita, Tetsuo 695 

Fujiwara, Hisayoshi 929 

Fujiwara, Takako 929 

Fukuhara, Hiroshi 761 

Fukunaga, Yoshitaka 242 

Fukushima, Yoshimitsu 618 

Fulci, Giulia 283 

Fuller, David D. 354, 818 

Furlan Freguia, Christian 603, 1114 

Futaki, Shiroh 68, 581, 587, 592 

Futami, Muneyoshi 103 

Futerman, Anthony H. 233 

Fux, Catherine 895 

Gackowski, Judith 27 

Gade, Terence P. F. 797 

Gage, Fred H. 56, 57, 650 

Gaggar, Anuj 137, 151, 409 

Gagnon, Raymonde F. 334 

Gaidosh, Gabriel 44 

Gäken, Joop 1131 

Galanis, Evanthia 720 

Galea-Lauri, Joanna 1131 

Galipeau, Jacques 268, 312, 334 

Gall, Jason G. 875, 1013 

Gallaher, Sean D. 874, 890 

Galli, Rossella 446 

Gallo, Jean-Marc 955 

Galun, E. 766 

Galun, Eithan 342, 735, 744, 806, 837 

Galy, Anne 330, 345 

Gambhir, Sam 808 

Gambhir, Sanjiv S. 743, 1008, 1051 

Gambotto, Andrea 66, 150, 891 

Gammon, D. 950 

Gammon, Dawn 947 

Ganesh, Shanthi 538, 545, 725 

Gans, Rijk O. 1112 

Gansbacher, Bernd 532, 1070 

Ganser, Arnold 368 

Gao, Guangping 112, 139, 141, 404, 

406, 500, 503, 506, 

508, 660, 812, 847, 

872, 1089, 1092 

Gao, Kai 127 

Gao, Liquan 796 

Gao, Wei 621, 637 

Gao, Wendy 1133 

Gao, Wentao 66, 150, 891 

Gao, Xiang 1110 

Gao, Yi 712, 714, 718 

Gao, Yongxin 627 

Garces, Silvia 432 

Garcia, Luis 261, 790, 820 

Garcia, Luz Maria 443, 997, 1000 

Garcia, Miquel 248 

Garcia, Racel 598 

Garcia-Blanco, Mariano A. 293, 1113 

Gardner, Thomas A. 1009 

Garhton, Gosta 1070 

Garrison, Brian S. 573 

Garrity-Moses, Mary E. 433, 482, 

855, 1050 

Gartley, Cathy 1036 

Garza-Guajardo, Raquel 298 

Gasiorowski, Joshua Z. 372 

Gasmi, M. 950 

Gasmi, Medhi 947 

Gaspar, Bobby 33 

Gaspar, Hubert B. 31 

Gaster, Richard 879 

Gattone, Vince 494 

Gauldie, Jack 694 

Gavrikova, Tatyana 1020 

Gazeas, Sophie 967 

Gazit, D. 766 

Gazit, Dan 184, 1053 

Gazit, Zulma 184 

Geddes, Duncan 356 

Geddes, Duncan M. 842 

Gehl, Julie 1066 

Geiger, R. Christopher 570 

Geisbert, Joan B. 996 

Geisbert, Thomas W. 996 

Gelbman, Brian D. 814 

Geller, Mauro 970 

Gendler, Sandra J. 1014 

Gennaro, Citro 568 

Gentry, Brian G. 807 

George, Andrew J. T. 773, 981, 1042 

George, George J. T. 171 

Georgiev, Panco 898 

Georgievska, Biljana 941 

Gepstein, Lior 931 

Gerard, Christopher J. 869 

Gere, Maja B. 371 

Gerloni, Mara 179 

Gerritsen, Winald R. 9, 526 

Gerstenfeld, Louis C. 1120 

Gerull, Sabine 455 

Geurts, Aron M. 176, 193 

Ghahramani Seno, 258 

Mohammad Mahdi 

Gharaibeh, Burhan 920 

Ghashghaei, Troy 752 

Ghavamzadeh, Ardeshir 1049 

Ghivizzani, Steven C. 835 

Ghosh, Arka 793 

Ghosh, Arkasubhra 515 

Ghosh, Debadyuti 24 

Giacca, Mauro 17, 114, 121, 

126, 370, 852, 1087 

Giangrande, Paloma H. 377 

Gianni, Davide 283 

Giannobile, William V. 677 

Gibson, Heather 715, 768, 959, 960 

Giering, Jeffery C. 88 

Gieselmann, Volkmar 429 

Giger, Urs 606 

Gil, Jose S. 874, 890 

Gilbert, Erin 1050 

Gilbert, James R. 936 

Gilbert, Rénald 256, 884 

Gilbert, Richard 699 

Gill, Deborah R. 351, 362, 366 

Gillingham, Melanie B. 897 

Gilmour, Kimberly C. 31 

Gimenez-Cassina, Alfredo 956 

Ginhoux, Florent 790 

Ginn, Samantha L. 485, 839 

Giordano, Frank A. 1034 

Giordano, Frank J. 43, 922 

Giorgio, Todd D. 772 

Giroglou, Tsanan 77 

Glasgow, Joel N. 28, 528, 873, 

886, 1017, 1018 

Glauser, Daniel 724 

Glazer, Daria 1084

124 



Glazer, Peter M. 369 

Glick, Roberta P. 697 

Glikin, Gerardo C. 696 

Glimm, H. 1055 

Glimm, Hanno 33, 1076 

Gloria, Gonzalez-Aseguinolaza 976 

Glorioso, Joseph C. 156, 161, 162, 

640, 641, 642, 646, 

671, 757, 760, 951 

Glover, Colin P. J. 656, 955 

Gobin, Andre M. 681 

Goebel, W. S. 474 

Goins, William F. 156, 161, 162, 

657, 671, 951 

Gojkovic, Zoran 306 

Gokhgauzer, Yuliya 998 

Goldfein, Joshua 940 

Goldman, Claudia 960 

Goldman, Steven A. 51 

Goldnau, Daniela 502, 863 

Goldsmith, Corinne M. 520 

Goldsmith, Merrill E. 453 

Goldstein, Steven A. 677 

Goltsov, Alexei 275, 695 

Gomar, Celia 555 

Gommans, Willemijn M. 314, 1073 

Gomori, John M. 806 

Gonçalves, Joâo G. R. 970 

Gong, Xiaoyan 817 

Gonzalez, Hugo L. 177, 178, 182 

Gonzalez-Aseguinolaza, Gloria 547 

Gonzalez-Guerrero, Juan F. 298 

Goodison, Steven 328 

Goodrich, Laurie R. 674, 675 

Gopal, Kapil 42, 603 

Gorbatyuk, Marina S. 1080 

Gorecki, Dariusz C. 784 

Gorschluter, Marcus 284 

Goss, James R. 671, 951 

Goto, Takahiro 107 

Goto, Yoshinobu 833 

Gotoh, Akinobu 546 

Gottschalk, Stephen 795, 800 

Gouble, Agnes 417, 902 

Gough, Michael 802, 804 

Gould, David 78 

Gouze, Elvire 835 

Gouze, Jean-Noel 835 

Goverdhana, Shyam 143 

Goyenvalle, Aurelie 820 

Grabowski, Gregory A. 910 

Grabski, Lauren E. 219 

Graham, Frank L. 694 

Graham, Ian R. 258, 261 

Graham, Michael W. 437, 997 

Graham, N. J. 1135 

Grainger, Simon M. 869 

Grandi, Paola 76 

Grant, Rebecca 139, 500 

Gray, John T. 118, 1030 

Greathouse, Kristin C. 246 

Green, Anthony P. 561, 575 

Greenberg, Daniel 611 

Greenberger, Joel S. 729, 1069 

Greene, Michael R. 733 

Gregorevic, Paul 266, 791 

Gregory, Lisa 394 

Gregory, Philip D. 43, 85, 337, 346, 

379, 646, 974, 1072 

Greiner, Suzanne 73 

Gresik, Vicky 795 

Grez, Manuel 332, 336, 1045, 1076 

Grider, Michael H. 964 

Griesenbach, Uta 350, 355, 356, 842 

Griesmann, Guy E. 496 

Griffith, Linda G. 205 

Griffiths, Denise 110 

Grillot-Courvalin, Catherine 564 

Grimm, Dirk 87, 857 

Grimm, Elizabeth 809 

Griot-Wenk, Monika 606 

Grist, John 648 

Grizzle, William E. 1017 

Grobe, Justin 627 

Grogery, Richard J. 628 

Grompe, Markus 1046 

Gropp, Michal 735 

Gross, Cynthia 958, 960 

Gross, David A. 790 

Grossman, William 619, 926, 933 

Groth, Amy C. 81 

Grott, Emmanuelle 396 

Groves, N. 1135 

Gruenert, Dieter C. 913 

Grunebaum, Eyal 339 

Gu, Danling 749 

Gu, Jin-fa 722, 731 

Gu, Yu-chen 458 

Guan, Min 876 

Guang, Jin 335 

Guerrini, Matteo 331 

Guest, P. J. 1135 

Guhl, Eva 371 

Guibinga, Ghiabe H. 186 

Guilbaud, Mickael 512 

Gunset, Gertrude 797 

Gunther, Roland 1098 

Guo, Wei 539 

Guo, Yanhong 621, 637 

Guo, Zongsheng 755 

Gupta, Ananya 157 

Gupta, Sanjeev 99 

Guschin, Dmitry 43 

Gustavsson, T. 924 

Gustrau, Annett 309 

Guyot, Valerie 417, 902 

Gvili, Jacob 185 

Haack, Karin 962 

Hacker, Caroline V. 395, 830 

Hackett, Christopher S. 176 

Hackett, Neil R. 65, 67, 147, 367, 

427, 508, 660, 812, 

853, 969, 1088 

Hackett, Perry B. 176, 192, 347, 1098 

Hadaczek, Piotr 124 

Haenggeli, Christine 652, 653 

Hagakura, Miki 1127 

Hagen, Otto 377 

Hagenbeek, Anton 461 

Hai, Mehreen 458 

Haisma, Hidde J. 314, 408, 753, 1073 

Hajjar, Roger 48 

Hajjar, Roger J. 45, 49, 850, 1116 

Hakkarainen, Tanja 97, 323, 487, 1026 

Häkkinen, Katja 97 

Hakumaki, Juhana 165 

Hale, S. 1027 

Hallden, G. 1027 

Hallek, Michael 502, 851, 863 

Hallenbeck, Paul L. 725 

Halling, Krista 1036 

Hama, Susumu 748 

Hamada, Hirofumi 587 

Hamada, Hiromi 125 

Hamada, Katsuyuki 546 

Hamilton, Melissa 875 

Hamm-Alvarez, Sarah F. 211, 1108 

Hamman, Kelly 897 

Han, Jaeseok 416 

Han, Jun-Ming 304 

Han, Xiaodong 501 

Hanawa, Hideki 3, 93, 831, 1071 

Hanazawa, Kisaburo 472 

Hanenberg, Helmut 470, 474 

Haniff, Gabriel 443, 1000 

Hankins, Gerald R. 649, 686



Hannaman, Drew 559 

Hannun, Yusuf 1011 

Hannun, Yusuf A. 450 

Hao, Zhenrong 922 

Hao, Shuanglin 640 

Hara, Toshiro 103 

Hara, Yuko 489 

Harada, Akiko 322, 364 

Harada, Taishi 322, 364 

Haraguchi, Miki 787 

Harashima, Hideyoshi 68, 376, 566, 

578, 581, 587, 

592, 746, 748 

Harati, Menahem 735 

Harbottle, Richard P. 209 

Harden, Paul 319, 882 

Harding, Cary O. 897 

Harding, Thomas 881 

Hardwich, Nicola 1131 

Hardy, Sarah 402 

Harkey, Michael 465 

Harkins, Richard N. 170, 172, 715, 

768, 958, 959, 960 

Harper, Scott Q. 52 

Harrington, K. J. 1135 

Harris, Steven S. 772 

Hart, Daniel 796 

Hartl, Irene 846 

Hartman, Zachary 407, 736, 1094 

Hartung, Rudolf 532 

Hartwell, Jennifer R. 474 

Harvey, Ben-Gary 367 

Harvey, Brandon K. 133 

Harvey, Mary 73 

Harvey, Susan 170 

Hasegawa, Kosei 297 

Hasegawa, Mamoru 355, 451, 833, 

841, 842 

Hasharoni, Amir 1053 

Hashimoto, Tomoko 210 

Haskins, Mark E. 32, 240, 247, 

421, 602 

Hassan, Mohamad 309 

Hassen, Waleed 797 

Hata, Ryuichiro 618 

Hatano, Norikazu 35 

Hauben, Ehud 36 

Hauck, Bernd 132, 505, 605 

Hauck, Ellen S. 577, 579, 584 

Haurigot, Virginia 236 

Hauschka, Stephen D. 266 

Hauswirth, William W. 667, 741, 

861, 1080 

Haviv, Yosef S. 537 

Hawes, M. L. 249 

Hawley, Robert G. 89 

Hawley, Teresa S. 89 

Hayakawa, Takao 69, 525, 527, 

748, 888 

Hayase, Motoya 48, 49 

Hayashi, Takuya 945 

Hayden, Michael R. 907 

He, Cheng-Yi 557 

He, Ling-feng 722, 731 

He, Miao 921 

He, Suiwen 782 

He, Weijing 617 

He, Xiaohua 52 

Hebel, Henry L. 177, 178, 181, 

182, 574 

Hecker, James G. 577, 579, 584 

Heeney, Jonathan 764 

Hefferan, Mike 963 

Heguy, Adriana 367, 508, 812, 814 

Heid, Irma 724 

Heidel, Jeremy D. 70, 208 

Heideman, Danielle A. M. 526 

Heier, Linda 969 

Heikura, Tommi 758 

Heller, Loree 699 

Heller, Richard 693, 699 

Helm, Gregory A. 184, 315, 649, 

686, 766, 1053 

Hemminki, Akseli 299, 328, 1004, 

1010, 1026, 1028 

Hempstead, Barbara 147 

Henckaerts, Els 20 

Hendershott, Karen 762 

Henderson, David 155 

Henderson, Gail E. 1078 

Hendrickson, Benjamin 101 

Hengge, Ulrich R. 309 

Hensley, Lisa E. 996 

Henson, Michael S. 803 

Herlihy, Amy 356 

Hermann, Felix 63 

Hermann, Manuel M. 77 

Hermens, Wim 907 

Hermiston, Terry 21, 319, 715, 

882, 958, 959 

Hermonat, Paul L. 128, 629, 937 

Hernandez-Alcoceba, Ruben 320, 547, 

555, 1027 

Herold, Chris L. 1061, 1067 

Herraiz, Maite 1134 

Herrbrich, Phillip 1035 

Herrbrich, Phillip E. 243, 460, 810, 

1031, 1032, 1037 

Herrero, Ignacio 1134 

Hersh, Louis B. 57 

Hershfield, Michael S. 35 

Herskind, Carsten 131, 326 

Herzer, Sibylle U. 173, 174 

Herzog, Christopher D. 947 

Herzog, Roland 132, 419, 615, 687 

Heslop, Helen E. 795, 798, 1074 

Hess, David A. 212, 460, 783, 1031 

Hesse, Andrea 1007 

Hew, Paggy 694 

Heywood, Darren J. 955 

Hiatt, Peter 815 

Hickey, Melissa A. 1, 100, 601 

Hickey, Reed P. 43, 922 

Hickstein, Dennis D. 458 

Hidaka, Chisa 674, 675 

High, Katherine A. 13, 14, 39, 84, 397, 

603, 605, 606, 608, 

609, 612, 614, 734 

Higino, Kalynka S. 970 

Hilken, Gero 310 

Hill, Vanessa 261 

Himeda, Charis L. 266 

Himmelreich, Uwe 77 

Hinh, Linda 43 

Hinkkanen, Ari 97 

Hintner, Helmut 1065 

Hinton, Andrew C. 186 

Hintze, Thomas 1087 

Hirai, Yukihiko 242, 1071 

Hiraoka, Kei 834 

Hirata, Koji 58 

Hiratsuka, Masaharu 742 

Hironaka, Takashi 841, 842 

Hiroyama, Takashi 472 

Hirsch, Raphael 678, 682 

Hisanaga, Michiyoshi 597 

Hisano, Terumasa 154 

Hisayasu, Sanae 968 

Hitchens, Moira R. 294 

Hitt, Mary M. 694 

Hiyama, Yaeko 576 

Ho, Ivy A. W. 163 

Ho, Seong-Hyun 278 

Hobson, Russell J. 955 

Hockerstedt, Krister 1028

126 



Hodges, Bradley L. 531 

Hodges, Joanna 277 

Hodgson, Clague P. 180 

Hoeben, Rob C. 543, 878, 1005 

Hoehn, Mathias 77 

Hoelters, Juergen 1103 

Hoelzer, Dieter 332, 336, 1076 

Hofling, Alex A. 243, 460 

Hofmann, Christian 980 

Hogan, Robert J. 1092 

Holder, Maxine 394 

Hollis, Roger P. 1102 

Hollmann, Charleen A. 969 

Holloway, Timothy P. 957, 967 

Holman, David H. 307, 450, 1011 

Holmes, Mark D. 811 

Holmes, Michael C. 85, 337, 346, 

379, 974 

Holt, Ingeborg 333 

Hombach, Andreas 798 

Homicsko, K. 1027 

Homicsko, Krisztian 1016 

Homma, Junpei 270 

Hong, Chang-Sook 951 

Hong, Hyo Jeong 728 

Hong, Youngtae 104 

Honma, Kimi 199 

Hood, David M. 673 

Hooymans, Anneke M. 1112 

Hope, Andrew 353 

Hopwood, John 421 

Hormann, Robert E. 1063 

Hornfeld, Dana 667 

Horst, Maarten ter 543, 1005 

Horwitz, Mitchell E. 234 

Hoshijima, Masahiko 41 

Hoshino, Kozo 48, 49 

Hospers, Geke A. 189, 1112 

Hotz-Wagenblatt, Agnes 1034 

Houchin, Tiffany L. 775 

Houdt, Winan J. 537 

Hough, Christine 610 

Hough, John 155 

Hough, Margaret R. 1036 

Hovanes, Bruce 279 

Howard, Doug B. 133 

Howard, Jeffrey 853 

Howarth, Joanna L. 955 

Howe, Steven 33, 391 

Howell, Hennessy 510 

Howell, John McC 253 

Howng, Sheng-Long 549 

Hoyt, Jason J. 195 

Hsiao, Pei-Wen 978 

Hsieh, Matthew M. 1041 

Hsieh, Mo-Ying 13, 397 

Hsu, Chi 875 

Hsu, Hui-Chen 608, 612, 991, 1090 

Hu, J. C. 1135 

Hu, Jian 640 

Hu, Jim 363, 365, 815 

Hu, Jinwei 692, 698 

Hu, Shiu-Lok 440 

Huang, Bijun 589 

Huang, Bonnie 205 

Huang, Chang-Jen 978 

Huang, Cui-fen 714, 718 

Huang, Jing Hwa 702 

Huang, JunJian 712, 714, 718 

Huang, Leaf 203, 586, 776 

Huang, Li-chun 571 

Huang, Samuel Chao-Ming 751 

Huang, Shaohua 641, 757 

Huang, Shao-LIng 626 

Huang, Wenlin 1015 

Huang, Yan 43, 922 

Huang, Yen-Chen 223 

Huang, Yong 1101 

Huang, Yu 619, 926 

Huang, Zan 405 

Huard, Johnny 254, 920, 923 

Hubbell, Jeffrey A. 217 

Huffer, Heather H. 677 

Hughes, Jackie 288 

Hughes, Travis 644, 645 

Huhtala, Tuulia 165 

Hui, Daniel 614 

Hui, Haipeng 935 

Hui, Kam M. 163 

Huie, Philip 770 

Hu-Lieskovan, Siwen 70 

Huls, Helen 795, 1074 

Humblet-Baron, S. 341 

Humeau, Laurent M. 477, 990, 1075 

Humphries, Margaret 1077 

Humphries, R. Keith 456 

Hung, Judy 48 

Hung, Mien-Chie 82 

Hunt, Kelly 287 

Husain, Tupur 129 

Hüser, Andreas 980 

Huxley, Clare 564 

Hwang, Seock-Yeon 717 

Hwang, Tae-Ho 167, 168 

Hyakusoku, Hiko 1071 

Hyde, Stephen C. 351, 362, 366 

Hytönen, Vesa P. 758 

Iacomini, John 109, 469, 737, 1033 

Iaina, Adrian 342 

Iankov, Ianko 719 

Iannitti, David 904 

Ichimura, Keiichi 663 

Idamakanti, Neeraja 311 

Iggo, R. 1027 

Iggo, Richard 1016 

Iida-Tanaka, Naoko 221, 226 

Iikura, Katsuhito 993 

Ikeda, Uichi 616, 938 

Ikeda, Yasuhiro 833 

Ikegami, Mie 355 

Ikemoto, Madoka 253 

Ikezawa, Makoto 251, 252, 255 

Ilies, Marc A. 588 

Illek, Beate 913 

Inagaki, Katsuya 504, 516 

Inoue, Hiroyuki 154 

Inoue, Makoto 355, 451, 841, 842 

Inoue, Ryo 1064 

Ioannides, Constantine G. 691 

Ioannou, Panos A. 957, 967 

Iqball, Sharifah 96 

Irving, Charles S. 806 

Isagai, Takeo 58 

Isayeva, Tatyana 119 

Ishibashi, Tatsuro 833 

Ishida, Akihiko 945, 948 

Ishii, Hajime 472 

Ishii, Keisuke 1064 

Isoniemi, Helena 1028 

Ito, Kaori 251, 252, 255 

Ito, Makoto 452 

Ito, Rie 578, 748 

Ito, Susumu 216 

Ito, Takayuki 616, 868, 938 

Ito, Tomoko 221, 224, 226 

Iurescia, Sandra 706 

Iwasaki, Hitoshi 841 

Izawa, Keiko 624, 631 

Izawa, Kiyoko 472 

Izumikawa, Masahiko 664 

Jack, Puymirat 260 

Jacob, Danielle 518 

Jacobs, Andreas H. 77, 724 

Jacobs, Michael A. 15, 386



Jaffar, Saeeda 231 

Jagadeesh, Jayashree G. 333 

Jain, Rakesh K. 76 

Jakobsson, Johan 91 

Jalonen, Harry 1095 

James, C. David 720 

James, N. D. 1135 

James, Williams 560 

Jan, Hsing-Yi 571, 928 

Jang, Hyung-Suk 625, 1052 

Jang, Jae-Hyung 769, 775 

Jang, Yangsoo 633 

Janik, John E. 1133 

Jänne, Juhani 308 

Jaquet, Muriel 667 

Jarrahian, Sohail 265, 622, 1103 

Jaskolka, Jennifer 357 

Jasti, Aparna 494, 825 

Jeff, Giering A. 821 

Jeffs, Lloyd 438, 996 

Jekich, Brian 644, 645 

Jeng, Meei-Huey 1009 

Jenkins, Connie B. 849 

Jenkins, Denny D. 467 

Jennings, Jerillyn S. 235, 425, 854 

Jennings, Lisa K. 296 

Jensen, Kristi 438 

Jeon, Eun-Seok 625, 1052 

Jeong, Jae-Gyun 278 

Jeong, Ji Hoon 207, 218 

Jeong, Jin-Sook 313 

Jeong, Moonsup 300 

Jeremie, Lavoie 260 

Jeremy, Burton 1051 

Jervis, Eric 79 

Jesus, Prieto 976 

Ji, J. 8 

Ji, Jianfei 436 

Ji, Lin 539 

Jia, Hong Peng 816 

Jia, Jidong 128 

Jia, Zhen 187, 567, 765 

Jian, Haiyan 603 

Jiang, Canwen 628 

Jiang, Haixiang 407 

Jiang, Jianyuan 263 

Jiang, Jie 637 

Jiang, Xuan 196 

Jiang, Ying 760 

Jie, Chen 705 

Jijing, Pang 1080 

Jimenez, Daniel F. 462, 743 

Jiménez, Juan A. 1009 

Jimenez, Veronica 236 

Jin, David 147 

Jin, Fang 21, 882 

Jin, Xiangyang 37 

Jo, Eui-Cheol 295 

Jofra Hernández, Raisa 330, 331 

Joh, Ju H. 1088 

John, Rossi J. 86 

Johns, David C. 289 

Johnson, Betty H. 588 

Johnson, Duane T. 883 

Johnson, Kirk W. 644, 645, 658 

Johnson, Lance 180 

Johnson, Larry G. 359 

Johnson, Mai 1008, 1051 

Johnson, Philip R. 986 

Johnson, R. P. 62 

Johnson, Robert P. 63 

Johnson, Shane 178, 182 

Johnson, T. 94 

Johnson, Terry 492, 494 

Johnston, Julie 112, 500, 503 

Joho, Shuji 619 

Jokinen, Mika J. 1095 

Jolly, Doug 825 

Jones, Bryony L. 366 

Jones, Martin 648 

Jones, Nicholas 238 

Jones, Steven 141 

Jones, W. Keith 782 

Jones III, John P. 378 

Jonkman, James 75 

Joo, Chang Wan 104 

Joo, Chul-Hyen 871 

Jordan, Heather 1089 

Jordan, V. Craig 204 

Jorgensen, Michael R. 593 

Joseph, G. 94 

Jouvenot, Yann 646, 974 

Jovin, Ion S. 922 

Juan, Ruiz 976 

Judge, Adam D. 438, 582 

Julien, Crettaz 976 

Julkunen, Ilkka 97 

Jun, Hee-Sook 416 

June, Carl H. 974, 990, 1075 

Jung, Chaeyong 1009 

Jung, Sandra 495 

Justilien, Verline 741 

Kaartinen, Vesa 90 

Kachapati, Kritika 476 

Kachi, Michiko 96 

Kachi, Shu 96 

Kadar, Elisabeth 432 

Kafri, Tal 343, 478, 822, 962 

Kagan, Elliott 996 

Kähäri, Veli-Matti 1095 

Kahl, Christoph A. 462 

Kai, Hirofumi 913 

Kaiser, Julia 7 

Kaiser, Larry R. 1129 

Kakeda, Minoru 742 

Kakinohana, Osamu 963 

Kaliberov, Sergey A. 529 

Kaliberova, Lyudmila N. 529 

Kalinke, Ulrich 475 

Kalle, Wouter H. J. 26 

Kamel, Marwa 259 

Kamen, Amine 518 

Kaminski, Joseph M. 1100 

Kaminsky, Stephen M. 427, 969 

Kamiya, Hiroyuki 376, 566, 578 

Kamizono, Junichi 1012 

Kamps, Gera 408 

Kamps, Jan 408 

Kan, Takatsugu 325 

Kan, Yuet W. 619, 926 

Kaneda, Yasufumi 216, 452, 1119 

Kanehira, Masahiko 69 

Kanehiro, Hiromichi 597 

Kaner, Robert J. 994 

Kanerva, Anna 299, 328, 1004, 

1010, 1026, 1028 

Kang, Bong-Su 717 

Kang, David W. 749 

Kang, Elizabeth M. 234, 1041 

Kang, Jeong Hun 206 

Kang, Lu 235, 425, 854 

Kang, Seok 278 

Kang, Yoon-A 551 

Kang, Yubin 601 

Kangasniemi, Lotta 299, 328, 1004, 

1010, 1026, 1028 

Kao, Ching-hai 1009 

Kao, Haihwa 658 

Kaplan, Eli 342 

Kaplan, Jean Claude 820 

Kaplitt, Michael G. 867, 940, 969 

Karara, Armando L. 696 

Karaus, Elke 332 

Karolewski, Brian A. 510

128 



Karpati, George 256 

Karppinen, Anne 308 

Karvelis, Kastytis 153 

Kasahara, Noriyuki 61, 710, 834, 982, 

1054, 1093 

Kashentseva, Elena 873 

Kaspar, Brian K. 650, 652, 653, 1085 

Kass, David A. 46 

Kastelein, John J. 907 

Kasturi, Sudhir P. 225 

Katakam, Anand K. 1061, 1067 

Katayama, Yasushi 486 

Katayama, Yoshiki 206, 213 

Katkin, Julie 815 

Kato, Ikunoshin 486 

Kato, Masanari 489 

Katoh, Motonobu 742 

Katovich, Michael 627 

Kattman, Steven 20 

Katz, Jacqueline M. 66 

Katz, Martin L. 434 

Kaul, Gunter 676 

Kaul, Rajinder 15, 386 

Kauser, Katalin 170, 715 

Kawabata, Kenji 525, 527, 888 

Kawai, Takao 929 

Kawakami, Yosuke 528, 1018, 1029 

Kawamura, Kiyoko 282 

Kawamura, Nobuaki 35 

Kawano, Hiroe 252, 255 

Kawano, Hiroki 701 

Kawano, Kouichiro 691 

Kawano, Takahito 213 

Kawase, Yoshiaki 48, 49 

Kawazu, Takeshi 214 

Kay, Mark A. 22, 87, 88, 191, 405, 

437, 441, 443, 467, 504, 

516, 557, 563, 573, 597, 

600, 821, 857, 915, 997, 

1000, 1046, 1084, 1101 

Kaye, David M. 41 

Kaynor, G. Campbell 887 

Kazazian Jr., Haig H. 1093 

Kean, Thomas 215 

Kedar, Eli 735 

Keiser, Nicholas W. 135 

Keith, Jarred 382, 1099 

Kelkar, Samir A. 847 

Keller, Bradley B. 920, 923 

Keller, Gordon 20 

Keller, Michael 593 

Kelly, Liam 79 

Kelly, Patrick F. 387 

Kelly, Stephen 955 

Kendle, Ryan 277 

Kennedy, Marion 20 

Kennedy, William 910 

Kennel, Stephen J. 586 

Keravala, Annahita 195, 1103 

Ketchum, E. 950 

Ketner, Elizabeth 1099 

Ketola, Anna 845 

Khai, Ngin Cin 929 

Khalil, Ikramy A. 581 

Khalili, Mitra 1049 

Khamis, Harry 630 

Khan, Amir S. 190, 763 

Khan, Zahidul 306 

Khazi, Fayaz R. 13, 14, 734 

Khim, S. 341 

Khwaja, Asim 711 

Kiang, Anne 407, 736 

Kida, Miyuki 35 

Kiedrowski, Matt 917 

Kiem, Hans-Peter 5, 145, 386, 440, 

455, 456, 726 

Kienast, Yvonne 209 

Kierlin, Monique N. 378 

Kikuchi, Eiji 710 

Kim, Bo Ra 827 

Kim, Byong-Moon 300, 633 

Kim, Chae-Young 300, 633 

Kim, Duk-Kyung 625, 633, 1052 

Kim, Eun Jung 556, 728 

Kim, Gyuyoup 161 

Kim, Hogeun 702 

Kim, Hong Sung 556, 728 

Kim, Hong-sup 1009 

Kim, Hyunhee 541 

Kim, In-Hoo 313 

Kim, Jae Ho 153 

Kim, Jae-Seok 167 

Kim, Jae-seok 168 

Kim, Jaesung 541, 702, 1019 

Kim, Jaesung Kim 551 

Kim, Jeong-Min 625, 633, 1052 

Kim, Jin-soo 992 

Kim, Ji-seon 198, 1106 

Kim, Jong Chul 556, 728 

Kim, Jong-Mook 278 

Kim, Joo-Hang 541, 551, 702, 1019 

Kim, Joon-Hyeong 167 

Kim, Ju Hyun 313 

Kim, June-Hyung 168 

Kim, Jung-Hye 983 

Kim, Kenneth 337 

Kim, Keun-Sik 556, 728 

Kim, Koung Li 625, 1052 

Kim, Kyung-Hee 975 

Kim, Meehyein 1003 

Kim, Min-Ju 1019 

Kim, Minjung 702 

Kim, Nam Yeon 983 

Kim, Pyung Hwan 541 

Kim, Seungtaik 932 

Kim, Sooin 1003 

Kim, Soon Sun 827 

Kim, Sujeong 104, 278 

Kim, Sung Jin 122, 871 

Kim, Sung Wan 207, 218, 585 

Kim, Sun-Kyung 717 

Kim, Sunyoung 104, 278 

Kim, Won Jong 207, 218, 585 

Kim, Yong Hee 207 

Kim, Yong-Hee 218 

Kim, Yoo Kyum 871 

Kim, Young 284 

Kim, Young-Min 717 

Kim, Youn-Ju 717 

Kim-Park, SangAe 1017 

Kimura, En 787, 1040, 1127 

Kimura, Hiromitsu 489 

Kimura, Shigemi 251, 252, 255 

Kimura, Takahiro 61, 834, 982, 1054 

Kimura, Takeshi 49 

Kindy, Mark S. 57 

King, C. R. 989 

King, C. Richter 875, 1013 

King, Doug 31 

King, Gwendalyn D. 143, 269, 273 

King, Nancy M. P. 1078 

King, Susan 863 

Kingsman, Alan J. 648 

Kingsman, Susan M. 55, 395, 481, 

599, 648, 830 

Kingston, Paul A. 620 

Kinjoh, Kiyohiko 963 

Kinnon, Christine 31, 391 

Kinoh, Hiroaki 451 

Kipp, K. 341 

Kirby, Patrick J. 194 

Kirik, Deniz 941 

Kirn, D. 1027 

Kirn, David H. 167, 168, 169 

Kishimoto, Atshuhiro 23 

Kita, Toru 49



Kitagawa, Tatsuya 210 

Kitay, Brandon M. 662 

Kiviat, Nancy 1006 

Kiviluoto, Tuula 299 

Kizana, Eddy 381, 839 

Kizawa, Takuji 685 

Klatzmann, David 946 

Klausegger, Alfred 1065 

Kleinschmidt, Jurgen A. 865 

Kliment, Christoph 977 

Klingerman, Candice M. 918 

Kluckman, Kim D. 478 

Kluger, Martin S. 922 

Kmiec, Eric B. 375, 380, 384, 

385, 1056 

Knapp, Alixandra A. 456 

Ko, Derek 881 

Ko, Dong Hyun 551, 1019 

Ko, Kerry 638 

Ko, Kyung Soo 202, 1060 

Ko, Saiho 597 

Kobayashi, Hidenori 1064 

Kobayashi, Ryouji 35 

Kobayashi, Seiichiro 103 

Kobinger, Gary 139 

Kobinger, Gary P. 141, 1092 

Kochanek, Stefan 27, 547, 876 

Kodama, Kota 206 

Koeberl, Dwight D. 897, 912, 1124 

Koehl, Ulrike 336 

Koehler, David R. 363, 365, 815 

Koehler-Vargas, Nadia 532 

Koerber, James T. 1085 

Kogelberg, Frank 495 

Koggel, Ingrid F. H. 461 

Kogure, Kentaro 68, 578, 581, 

587, 592 

Kohlbrenner, Erik 400, 418 

Kohn, Dieter 120, 522, 676 

Kohn, Donald B. 37, 90, 341, 462, 

463, 743, 805, 987, 

998, 1102 

Kohrt, Nicole M. 219 

Koike, Hiromi 945, 948 

Koistinaho, Jari 98 

Koizumi, Naoya 527 

Kok, Robbert J. 883 

Koldej, Rachel M. 491 

Kole, Ryszard 343, 321 

Kolls, Jay K. 977 

Kolykhalov, Alexander A. 437, 443, 

997, 1000 

Komarova, Svetlana 542 

Komiya, Setsuro 1012 

Kondo, Satoshi 834 

Kong, Michelle 1072 

Kong, Spring 361 

Koniar, Brenda L. 1098 

Konno, Akihiro 333 

Kono, Riichiro 833 

Konopka, Krystyna 590 

Koo, Tae Young 541 

Koop, Sue 825 

Koota, Suvi 38 

Koponen, Jonna K. 98 

Kordower, Jeffrey H. 947 

Korja, Sanna 323 

Kornegay, Joseph 792 

Korokhov, Nikolay 28, 30, 316, 1130 

Kosai, Ken-ichiro 58, 473, 929, 1012 

Koskinen, Mika K. 1095 

Kosmides, Daniela 1007 

Kostic, Corinne 667 

Kotin, Robert M. 52, 517, 716 

Kotlarchyk, Maxwell 1104 

Kottke, Timothy 267, 271, 449, 723 

Kovesdi, Imre 975, 1088 

Koya, Richard C. 61, 982, 1054 

Koyama, Nobuto 486 

Koyama, Yoshiyuki 221, 224, 226 

Kozyak, Benjamin W. 42 

Kral, John G. 754 

Kramer, M. Gabriela 320, 555, 876 

Krance, Robert A. 1074 

Krasnykh, Victor 529 

Krause, Anja 67, 975, 1088 

Krause, Diane 651 

Krausova, Valentina 155 

Krendelchtchikova, Valentina 529 

Krendelshchikov, Alexander 30 

Kreppel, Florian 27 

Kretschmer, Peter 21, 882 

Krewet, James A. 285 

Kriegl, Jaclynn M. 677 

Krishna, Delfi 483 

Krisky, David M. 156, 641, 642, 

646, 757, 760 

Kritz, Angelika B. 620 

Kroutov, Vadim 418 

Kruth, Stephen A. 1036 

Kubo, Koji 221 

Kubo, Shuji 1093 

Kucsma, Nora 1045 

Kuehlcke, Klaus 336, 495 

Kuge, Hiroyuki 597 

Kuhel, David 6 

Kuhn, Irene 21, 319 

Kuhr, Christian S. 791 

Kuivenhoven, Jan Albert 907 

Kulkarni, Rajan P. 1109 

Kulomaa, Markku S. 758 

Kumaki, Satoru 344 

Kumar, Pankaj 479 

Kumar, Prasanna 1061, 1063, 1067 

Kumar, Sanjay 136, 849 

Kumar-Singh, Rajendra 670 

Kume, Akihiro 125, 517, 616, 663, 

860, 862, 868, 900, 938 

Kung, Hsiang-fu 712, 714, 718 

Kunis, Mike 43 

Kunkel, Hana 336 

Kunugiza, Yasuo 624, 631, 685 

Kuo, Hus-Ping 82 

Kuo, Yu-Hung 867 

Kuppuswamy, Mohan 1021, 1022, 1023 

Kurai, Toshiyuki 1071 

Kure, Shigeo 900 

Kurita, Ryo 154, 472 

Kuriyama, Takayuki 282 

Kuroda, Toshi 761 

Kurtz, Andreas 761 

Kusaga, Akira 58 

Kusaka, Nobuyuki 695 

Kushida, Miwa 708 

Kusmic, Claudia 121 

Kusumanto, Yoka H. 1112 

Kutner, Robert H. 482, 493 

Kutzler, Michelle 693 

Kwack, Seung Jun 827 

Kwok, K. 950 

Kwon, Byung-Su 313 

Kwon, Heechung 161 

Kwon, Heung-sun 992 

Kwon, In-Ho 300 

Kwon, Oh-Joon 551 

Laakkonen, Pirjo 328 

Labelle, Andrea 410, 610 

Lacar, Benjamin 941 

Lachance, Jean-Guy 1079 

LaFace, Drake 544 

Lahey, Ryan 212, 783, 1035 

Lahtinen, Mika 630 

Lai, Chiou-Lian 549 

Lai, Li-Ju 751 

Lai, Yi 793 

Laing, Ken 258

130 



Laipis, Philip J. 422, 423 

Laitinen, Olli H. 758, 1073 

Lakshmipathy, Uma 193, 338 

Lam, George 147 

Lam, Karen S. 123 

Lam, Paula Y. P. 163 

Lambin, Phillipe 329 

Lambowitz, Alan M. 378 

Lan, Michael S. 730 

Landázuri, Natalia 480 

Landers, M. 950 

Lang, Susanne 704 

Langemeijer, Saskia 1041 

Langer, Robert 777, 786 

Langer, Stephen J. 382, 644, 645, 1099 

Langfield, Kirsten K. 496 

Langridge, William H. R. 155 

LaRegina, Marie C. 1021, 1023 

Largaespada, David A. 176, 193, 445 

Larsen, Brent 170, 172 

Larsen, Steven H. 11 

Larson-DeBruzzi, Beth E. 1098 

LaRusso, Nicholas F. 721 

Laryssa, Comer 858 

Lau, George K. 712 

Lau, Ying-Ka I. 781 

Lauer, Ulrich M. 840 

Lauffenburger, Douglas A. 205 

Laufs, Stephanie 131, 326, 865, 1034 

Laura, Ochoa 976 

Lauterborn, Daniel 1034 

Lavigne, Matthieu D. 784 

Lawhorn, Ingrid 786 

Lawrence, Theodore S. 361 

Lawrencia, Carmel 74 

Layzer, Juliana 377 

Lazarus, A. 766 

Lazarus, Alan H. 610 

Le, Hong T. 148, 412, 414 

Le, Long P. 25, 1018, 1020 

Le Doux, Joseph M. 480, 483 

Leapley, Alyssa C. 462 

Lebeche, Djamel 45 

Leboeuf, Marylène 790 

Lecoultre, Claude 895 

Leddon, Jennifer L. 883 

Leduc, Sophie 417, 902 

Lee, Amy C. H. 438, 996 

Lee, Boyoung 122 

Lee, Brendan 149, 535, 598, 885, 903 

Lee, Chang I. 462, 743 

Lee, Eunsu 1060 

Lee, Gary 545 

Lee, Haeyul 363 

Lee, Heuiran 122, 871 

Lee, Hwang-Jae 278 

Lee, Jae-Young 625, 1052 

Lee, Ji Eun 556 

Lee, John 812 

Lee, Jung-Sun 625, 1052 

Lee, Kang-Eun 717 

Lee, Kyuhyun 295 

Lee, Mija 932 

Lee, Minhyung 202, 207, 218, 1060 

Lee, Rhee Da 827 

Lee, Robert 283 

Lee, Sang-Jin 1009 

Lee, Seong-Wook 313, 983 

Lee, Ya-Li 85, 337, 974 

Lee, Young-Sam 625, 1052 

Lee, Young-Sook 551 

Lee, Zhenghong 1104 

Lee†, Jung-Sun 633 

Leege, Tobias 348 

Leen, Ann M. 800, 1074 

Leeuw, Bertie de 543, 1005 

Legorreta-H, Martha 995 

LeGuern, Christian 738 

Lehmann, Romy 234 

Lehmusvaara, Saara 97, 487 

Lei, Chi-Man 498 

Leinwand, Leslie A. 382, 644, 

645, 1099 

Lem, Janis 667 

Lemmens, Raf 173 

Lemonnier, François 790 

Lemos, Julio A. 936 

Leng, Qixin 317 

Lenting, Peter 335 

Leonard, Joshua N. 519, 1002 

Leong, Kam W. 196, 200, 779 

Leopold, Philip L. 847, 1123 

Leppanen, Pia 38 

Leps, Walter T. 173 

Lerner, Seth P. 286 

LeRoy, Stanley G. 866, 961 

Lesniak, Maciej S. 537 

Lessard, Ruth 658 

Leturcq, France 820 

Leung, Kitty 365 

Leurs, Cordula 470 

Levine, Bruce L. 990, 1075 

Levine, Robert A. 48 

Levine, Ronald H. 363 

LeVisage, Catherine 200 

Levy, Beth 237, 424 

Levy, Robert J. 623, 740, 879, 

918, 930 

Lewin, Alfred S. 53, 59, 422, 741, 

861, 954, 1080 

Lewis, Aaron 735 

Lewis, John A. 893 

Lewis, Melissa A. 44, 909, 911 

Leyendecker, Peter 495 

Li, Anna A. 220 

Li, Changlong 80, 388, 390 

Li, Chengwen 464 

Li, Dayuan 629 

Li, Dexi 595 

Li, Fang 479 

Li, Ha-Tang 998 

Li, Hongwei 315, 627 

Li, Hua 123 

Li, J. Z. 766 

Li, Jianbin 1125 

Li, Jin Zhong 184, 315, 649, 686 

Li, Jing 25, 540 

Li, Juan 133, 254, 398, 751, 

792, 916, 1086, 1125 

Li, Lei 922 

Li, Li 326 

Li, Lijing 19, 402 

Li, Lina 716 

Li, Linda 316 

Li, Ling 46 

Li, Lingli 957, 967 

Li, Linhong 192 

Li, M. J. 8 

Li, Ming-Jie 998 

Li, Qiliang 348 

Li, Qingfen 291 

Li, S. 8 

Li, Senlin 617 

Li, Sheng 787, 1040, 1127 

Li, Shulin 272, 700, 703 

Li, Song 586 

Li, Weiming 401 

Li, Xiao L. 274 

Li, Xiaojin 103 

Li, Xiao-Kang 263, 489 

Li, Xiaoying 935 

Li, Xiao-Yong 1072 

Li, Xiong 1009 

Li, Yan 111 

Li, Yanjun 16, 401



Li, Ying 289 

Li, Yining 265 

Li, Yong 254 

Li, Yuanhao 311, 538, 545, 881 

Li, Yuwen 929 

Li, Zheng 82 

Li, Zhuoya 291 

Li, Zong-Yi 145, 151, 411, 726 

Liang, Haidong 356 

Liang, Wei 635 

Liang, Yuxin 43 

Liang, Zhihui 1015 

Libergall, Meir 1053 

Liberman, Yael 342 

Libson, Eugene 806 

Lichtenstein, Drew L. 1021, 1022, 1023 

Lichtor, Terry 697 

Lieber, Andre 137, 145, 151, 348, 

409, 411, 726, 880, 

980, 1006, 1091 

Lieu, Ann-Shung 649, 686 

Lievens, Joke 413, 548, 636, 1115 

Liew, Aaron 550 

Liggitt, Denny 459 

Lightowlers, Robert N. 1105 

Liimatainen, Timo 165 

Lillicrap, David 410, 610 

Lim, Filip 956 

Lim, Leland E. 262 

Lima, Deolinda 158 

Lima e Silva, Raquel 96 

Limberis, Maria 146 

Lin, Chung-Ren 643 

Lin, Darshong 424 

Lin, En-Hau 978 

Lin, Ken-Kuo 751 

Lin, Marie C. 712, 714, 718 

Lin, R. 950 

Lin, Richard 947 

Lin, Seppi 467 

Lin, Yanjuan 554 

Lind, Miia M. 758 

Linden, R. Michael 20 

Lindholm, Leif 878 

Linetsky, Eduard 806 

Ling, Yiqun 920 

Link, Charles J. 1133 

Lintmaer, Ingrid 465 

Linton, Gilda 234 

Lipp, Hans-Peter 939 

Lippin, Yitzhak 342 

Lipskaia, Larissa 1116 

Lisowski, Leszek 4 

Liszewski, M. Kathryn 137 

Little, Marie-Terese 791 

Liu, Bianling 659, 813, 1043 

Liu, Chunyan 269, 273, 745 

Liu, Dexi 1110, 1096 

Liu, Dijie 198, 1106 

Liu, Dingang 922 

Liu, Dinggang 43 

Liu, Feng 203, 776 

Liu, Geyi 176 

Liu, Guei-Sheung 549 

Liu, Guo J. 899 

Liu, James 113 

Liu, James K. 855 

Liu, Jia 861 

Liu, Jiaguo 700 

Liu, Jianhua 84, 1114 

Liu, Kan 689 

Liu, Li 34 

Liu, Linda N. 192 

Liu, Mingju 688, 793 

Liu, Paul Y. 680, 684, 689 

Liu, Perry 715, 768, 958, 959 

Liu, Qiang 1126 

Liu, Quing 283 

Liu, Renbin 160 

Liu, Ta-Chiang 761 

Liu, Wei-li 197 

Liu, Wen 1126 

Liu, Xiang 307, 450, 1011 

Liu, Xiaoming 498, 499, 521 

Liu, Xin-yuan 722, 731 

Liu, Xiuhua 66 

Liu, Yanna 287 

Liu, Yanxin 305 

Liu, Yanzheng 444 

Liu, Yemin 782 

Liu, Yi-Lin 13, 397, 614 

Liu, Yong 629, 937 

Liu, Yuhe 663 

Liu, Yuli 239 

Lloyd, Janice 369 

Lo, Hsin-Lung 832 

Lobel, Peter 427 

Lochrie, Michael 130 

Lock, Martin 112, 500 

Lockridge, Jennifer A. 438 

Lodde, Beatrijs M. 466, 672, 1122 

Logan, Grant 988 

Logg, Christopher R. 710, 834 

Loi, Roberto 357 

Loktionova, Natalia 1039 

Lombardi, Giovanna 981, 1042 

Lombardo, Angelo 36, 346, 392, 468 

Lombardo, Barbara 683 

Lompre, Anne-Marie 1116 

Looper, David 749 

Lopez, Maria V. 78 

Lord, E. M. 687 

Lorenzo, Damaris 244 

Losen, Mario H. 189 

Lotti, Francesco 83 

Lou, Howard 1123 

Louboutin, Jean-Pierre 111, 659, 

661, 813, 1043 

Louca, Emily 365 

Louise, Deschênes 1079 

Love, C. A. 1135 

Low, David W. 42 

Low, Walter C. 244, 445 

Lowenstein, Pedro R. 143, 269, 273, 

430, 745, 946 

Lowrey, Christopher H. 733 

Lu, Baogen 537 

Lu, Feizhou 263 

Lu, Jiamo 477 

Lu, Mei 153 

Lu, Qi L. 788 

Lu, X. 62 

Lu, Xiaobin 477, 990, 1075 

Lu, Xiaochun 935 

Lu, Xiaoli 817 

Lu, Yi 296 

Lu, You 404, 500, 506 

Lu, Yuanqing 511 

Luban, Jeremy 2 

Luc, Charland 260 

Lucas, Bart 774 

Lucena, Felipe 1134 

Lucia, Sergi Sergi 389 

Lugo-Trampe, Angel 553 

Lukashev, Alexander 1016 

Luke, Jeremy 180 

Lum, ChingTung 712 

Lumbers, Eugenie 485 

Lundberg, Cecilia 91, 666 

Luo, Meihui 498, 499, 521 

Luo, Vivian 393 

Luo, Zhengyu 628 

Lupo, Rossella 64 

Lupo Stanghellini, Maria T. 819 

Lustig, Kurt H. 677

132 



Lutherborrow, Mark 899 

Lutzko, Carolyn 101, 463 

Lux, Kerstin 502, 863 

Luxembourg, Alain 559 

Ma, Chunyan 522, 859 

Ma, Hong 478, 822 

Ma, Hsin-I 304 

Ma, Xiucui 239 

Ma, Zhijun 1030 

Maatta, Ann-Marie 323 

Macauley, Shannon 424 

MacDonald, Robert C. 576, 626 

MacGregor, Rob Roy 990, 1075 

Machluf, Marcelle 185, 569 

MacLachlan, Ian 438, 582, 996 

MacLaren, Robert E. 391 

MacNeil, Angus 391 

MacVeigh, Michelle 1108 

MacVittie, Thomas J. 234 

Maden, Malcolm 648 

Madoiwa, Seiji 860 

Madry, Henning 120, 522, 676 

Maeda, Akito 1119 

Maeda, Yoshikazu 616, 938 

Maeyama, Yoshihiro 35 

Magnani, Zulma 819 

Maguire, Albert 135 

Maguire, Katie 380 

Maguire-Zeiss, Kathleen A. 431 

Mah, Cathryn S. 44, 354, 818, 

909, 911 

Mahasreshti, Parameshwar J. 1017 

Maheshri, Narendra 1085 

Mähönen, Anssi J. 758 

Mahoney, John 644, 645 

Mahoney, Melissa J. 644 

Mai, Jeffrey C. 294 

Maier, Patrick 326 

Maier, Steven F. 644, 645 

Mainka, Astrid 1007 

Mairey, Émilie 884 

Mairs, Rob J. 1044 

Mäkelä, Kari A. 758 

Mäkinen, Kimmo 323 

Makinen, Petri I. 38, 98 

Mal, Niladri 917 

Malech, Harry L. 234, 1039 

Mallet, Jacques 92, 1059 

Malm, Tarja M. 98 

Maminska, Marie E. 696 

Mandai, Masaki 708 

Mandel, Ronald J. 53, 59, 654, 

952, 954, 1082 

Mane, Viraj P. 149, 535, 903, 905 

Manes, Thomas 922 

Maneval, Daniel C. 544 

Manevitch, Alexandra 735 

Manevitch, Zakharia 735 

Manfredsson, Fredric P. 59, 1082 

Mango, Ruggiero 568 

Manickan, Elanchezhiyan 144 

Manilla, Peter 990, 1075 

Manlapaz, L. 950 

Mano, Miguel 222 

Mansfield, Gary S. 521 

Mantwill, Klaus 532 

Manunta, Maria 773 

Mao, Hai-Quan 196, 779 

Mao, Qinwen 52, 427 

Mao, Si-Hua 95 

Mao, Xiangling 969 

Marangoni, Francesco 330, 345 

Marban, Eduardo 381 

Marcello, Alessandro 17, 852 

Marchesini, Sergio 426 

Marcos, Josep 232 

Marerro, Barbara 693 

Margaritis, Paris 39, 84 

Margison, Geoffrey 829 

Marigo, Valeria 435 

Marini, Alessandra 309 

Marini, Frank 447, 727 

Marinov, Anthony D. 682 

Marion, Patricia L. 441 

Markovitz, Nancy S. 159 

Marlyne, Goulet 1079 

Marom, Shimon 931 

Marr, Robert A. 56, 57 

Marsala, Martin 963 

Marsh, J. 94 

Marshall, John 910 

Martens, Anton C. M. 461 

Marti-Climent, Josep M. 808 

Martin, Donald K. 899 

Martin, Douglas R. 962 

Martin, J. Tyler 43 

Martin, Natalia 159 

Martinez, Laura 759 

Martinez Serrano, Alberto 183 

Martinez-Davila, Irma A. 553 

Martinez-F, Francisco 995 

Martino, Ashley 1083 

Martino, Silvana 345 

Martins, Ines H. 52 

Martins, Isabel 158 

Martuza, Robert L. 160, 761 

Mas, Alex 901 

Masi, Giulia 536 

Masikat, Mary Rose 170 

Masliah, Eliezer 56, 57 

Massie, Bernard 884 

Masterson, Steve 559 

Mastrangelo, Michael A. 431 

Masuda, Tomoya 746 

Mata, Marina 640, 642, 671 

Matar, Majed 227, 279 

Matern, Dietrich 921 

Mathis, J. Michael 25, 274, 540, 1018 

Mathis, Michael 316 

Matsuda, Takeshi 1064 

Matsui, Takashi 46, 50 

Matsuishi, Toyojiro 58 

Matsukura, Makoto 251, 252, 255 

Matsumoto, Kazumasa 286 

Matsumoto, Yoko 252 

Matsuo, Msafumi 252 

Matsuoka, Yumiko 66 

Matsushita, Miko 799 

Matsushita, Takashi 616, 868, 900, 938 

Matt, Coffey 174 

Mauleon, Itsaso 547 

Maurilio, Ponzoni 389 

Maurisse, Rosalie 913 

Mautner, V. 1027 

Mavilio, Fulvio 64, 819 

Maxwell, Dustin J. 212, 783 

Maynard, Jonathan 444, 1128 

Mayr, Christine 284 

Mazarakis, Nicholas D. 55, 395, 648 

Mazur, Marina 507 

Mazzolini, Guillermo D. 808, 1134 

McAllister, Christopher S. 771 

McAllister, Diane 749 

McAnuff, Marie 1106 

McCabe, Cillian T. 632 

McCart, Judith A. 75 

McCarty, Douglas M. 18, 403, 854 

McClintock, Kevin 582 

McCombs, Michelle R. 596 

McConnell, Ruth 815 

McCormack, Michael 598 

McCorquodale, Stephanie 32, 607 

McCown, Thomas 478 

McCray, Jr., Paul B. 1, 100, 601, 816



McCroquodale, Stephanie 609 

McDonagh, Anthony F. 535 

McEachern, Kerry 910 

McGarrity, Gerard J. 1113 

McGettigan, James P. 999 

McGregor, Christopher G. A. 47 

McGregor, Jennifer 48 

McIntosh, Jenny 711 

McIvor, R. Scott 192, 193, 244, 

338, 347, 826, 

908, 1068, 1098 

McKay, Karen 485 

McKee, Trevor D. 76 

McKerlie, Colin 365 

McKillop, John C. 450 

McLachlan, Gerry 842 

McLane, Brienne 416 

McLaughlin, Pamela M. J. 314, 1073 

McLenachan, Samuel 967 

McMahon, Stephen B. 648 

McNamara, Andrew 646 

McNeish, I. 1135 

McPherson, David D. 626 

McPherson, Sylvia A. 140 

McSwiggen, James A. 438 

Mctiernan, Charles 916 

McVey, Duncan 875, 989 

McVie-Wylie, Alison 1124 

Meager, Tony 711 

Medina-Kauwe, Lali K. 149, 211, 1108 

Medley, L. C. 1135 

Mehta, Jay L. 629, 937 

Mehta, Navneet 75 

Mei, Manxue 32, 602 

Mei–Zahav, Chava 342 

Meijer, Coby 189 

Meinking, K. 8 

Melani, Cecilia 1070 

Melcher, Alan 271 

Melero, Ignacio 320, 1134 

Meltzer, Stephen J. 325 

Menendez, Silvia 710 

Mengchao, Wu 705 

Menger, Michael D. 522 

Mengesha, Asferd 329 

Meriläinen, Outi 97, 487 

Merkler, Kathleen 699 

Merricks, Elizabeth 607 

Merritt II, J. Lawrence 921 

Mertens, Geertrui 413 

Messer, Anne 943 

Metgcalf, Jason 421 

Meulenberg, Janneke M. 907 

Meuse, Leonard A. 266, 787, 1127 

Meyer, Jennifer M. 1021, 1022, 1023 

Meyerrose, Todd E. 243, 353, 810, 

1032, 1037, 1121 

Meyn, Raymond 287 

Mezquita, Pau 5 

Mhashilkar, Abner M. 809 

Mi, Zhibao 817 

Miao, Carol H. 341, 611, 613, 785 

Miccio, Annarita 83 

Michael, A. 1135 

Micheaux, Beatrice 417 

Michel, Sylvain 1079 

Middaugh, C. Russell 561 

Migita, Hideyuki 715 

Miike, Teruhisa 251, 252, 255 

Mikel, Zaratiegui 976 

Mikkelsen, Jacob G. 22, 573 

Miletic, Hrvoje 77 

Millan, Maria T. 554 

Miller, Aaron 588 

Miller, Andrew D. 593 

Miller, Carole 494 

Miller, Daniel G. 15, 386 

Miller, Jeffrey C. 85, 337, 346, 379, 974 

Miller, Jim 139, 404 

Miller, Justin 181, 574 

Millican, C. Leigh 883 

Milligan, Erin D. 644, 645 

Millington, David S. 912 

Milsom, Michael D. 1044 

Mima, Hidetoshi 216 

Mimuro, Jun 125, 860, 862 

Minakuchi, Yoshiko 199 

Minatoguchi, Shinya 929 

Mineno, Junichi 486 

Mineshiba, Fumi 466, 672, 1122 

Minggao, Guo 705 

Minghong, Jiang 705 

Mingle, B. L. 419 

Mingozzi, Federico 132, 609, 614 

Miquel, Sena Esteves 858 

Mir, Lluis M. 188, 1066 

Mirek, Hanna 124 

Mirmohammadsadegh, Alireza 309 

Mishra, Swaroop 1109 

Miskin, James E. 395, 481, 830 

Mistry, Ajay 394 

Mitani, Ko 23 

Mitchell, Christine B. 175 

Mitchell, James B. 471 

Mitchell, Lloyd G. 293, 521 

Mitchell, Marilyn A. 42 

Mitomo, Katsuyuki 355 

Mitrophanous, Kyriacos A. 55, 395, 

481, 599, 830 

Mitrovic, Branka 170 

Mitsui, Masaru 701 

Mittal, Suresh K. 152 

Miwa, Takeshi 251 

Mixson, A. James 317 

Miyagi, Tohko 489 

Miyagishi, Makoto 592 

Miyagoe-Suzuki, Yuko 253 

Miyahara, Ryo 691 

Miyahara, Yasuko 691 

Miyamae, Takako 682 

Miyanohara, Atsushi 186, 963 

Miyashita, Hiroshi 616 

Miyazaki, Masanori 833 

Miyoshi, Hiroyuki 103 

Mizrachi, Sarah 342 

Mizuguchi, Hiroyuki 69, 525, 527, 

748, 888 

Mizukami, Hiroaki 125, 517, 616, 

663, 860, 862, 

868, 900, 938 

Mizuno, Hiroshi 1071 

Mizuno, Yoshikuni 949 

Mo, Lian 545 

Mochizuki, Hideki 949 

Mody, Disha A. 274 

Moezi, Lili 1049 

Moffitt, Michael 433 

Moimas, Silvia 114, 121 

Mok, Hoyin 415 

Mok, Wilson 76 

Moller, Pernille H. 1066 

Molnar, Fanni E. 770 

Momoi, Nobuo 923 

Monnat, Raymond J. 1048 

Montalban, Xavier 432 

Montane, Joel 901 

Montcuquet, Nicolas 739 

Montecalvo, Angela 66, 891 

Montefiori, David C. 986 

Montini, Eugenio 389 

Montoya, Fernando 193 

Mooij, Petra 764 

Moon, Joon-Shik 256 

Moore, Peter M. 173 

Moran, John V. 1093 

Moratto, Daniele 333

134 



Moreno, Jesus 723 

Mori, Satsuki 916 

Mori, Yuriko 325 

Moriguchi, Rumiko 68, 581, 592 

Morishita, Mariko 107 

Morishita, Ryuichi 624, 631, 685, 

945, 948 

Morizono, Hiroki 908 

Morris, Emma 796 

Morris, John C. 26, 1014, 1133 

Morris, Julia C. 5 

Morris, Kevin V. 86 

Morrissey, David V. 438 

Mortellaro, Alessandra 331 

Moses, Leslie 1133 

Motooka, Yasuhiko 445 

Moullier, Philippe 512 

Mountz, John D. 140, 608, 612, 

991, 1090 

Moy, Sheryl S. 235 

Muehlebach, Michael 7 

Mueller, Lars 829 

Muench, Marcus O. 501 

Muenk, Carsten 836 

Muenzer, Joseph 235, 250, 425, 854 

Mufti, Ghulam 1131 

Mukherjee, Atish 57 

Mulder, Nanno H. 189, 1112 

Muller, Christian 1083 

Müller, Markus 565 

Mullins, Charles 906 

Munier, Francis L. 667 

Munson, Keith 116 

Murakami, Akira 210 

Muramatsu, Shin-Ichi 663 

Muraro, Sara 819 

Murata, Toshinori 833 

Murofushi, Yoshiteru 58, 1012 

Murugesan, Selva R. 875, 1013 

Musatov, Sergei 940 

Mushiake, Hiroyuki 294 

Muul, Linda M. 333 

Muzyczka, Nicholas 59, 400, 1082 

Myara, Anne 895, 896 

Myers, Gary D. 1074 

Myers, Michael P. 852 

Naash, Muna I. 668 

Nabel, Gary J. 708 

Nagahara, Shunji 199 

Nagano, Satoshi 58, 1012 

Nagasaki, Takeshi 214 

Nagasawa, Yasuo 465 

Nagata, Keiko 742 

Nagoshi, Tomohisa 46 

Naimark, Wendy 928 

Najm, Imad 433 

Naka, Hiroyuki 597 

Nakagami, Hironori 1119 

Nakai, Hiroyuki 504, 516 

Nakajima, Satoru 35 

Nakajima, Yoshiyuki 597 

Nakakura, Takayo 517 

Nakamra, Takashi 68 

Nakamura, Gerald 559 

Nakamura, Takafumi 73, 297, 720 

Nakanishi, Mahito 107 

Nakanishi, Yoichi 154, 322, 364 

Nakano, Kenji 162 

Nakayama, Masaharu 886 

Nakazaki, Yukoh 154, 472 

Nalbantoglu, Josephine 256, 518 

Naldini, Luigi 36, 330, 331, 345, 

346, 358, 373, 389, 

392, 426, 446, 468, 

972, 1038, 1070 

Nam, Young Ran 122, 871 

Nambiar, Sandeep 309 

Nantz, Michael H. 577, 579, 584, 596 

Narfstrom, Kristina 434 

Naruishi, Koji 695 

Narvanen, Ale 165 

Nasevicious, Aidas 192 

Nash, Kevin 53, 400 

Nathwani, Amit C. 118, 711 

Naundorf, Sonja 495 

Naylor, Stuart 96 

Nazarian, Ara 1120 

Negrin, Robert 756 

Neilan, Tom G. 48 

Nelson, Amy R. 1020 

Nelson, Anthony 42 

Nelson, Daniel K. 1078 

Nelson, Robert J. 490 

Nemet, Katalin 1045 

Nervi, Bruno 288, 709, 810 

Neth, Peter 1103 

Nettelbeck, Dirk M. 1007, 1029 

Netti, Paolo 683 

Neubert, Wolfgang J. 840 

Neuenfeldt, James 276 

Neumann, Harald 77 

Neves, Silvia S. 302 

Nevins, Joseph R. 377, 736 

Newby, Andrew C. 534 

Newrzela, Sebastian 63 

Ng, Cathy C. Y. 711 

Ng, Philip 143, 273, 363, 365, 

430, 535, 607, 815, 

885, 903, 904, 905, 906 

Ng, Samuel S. 712, 714, 718 

Ng, Thomas 904 

Nguyen, Natalie 311 

Nguyen, Tuan Huy 895, 896 

Ni, Shaoheng 145, 151, 411, 

726, 1006 

Ni, Yajin 990 

Nichols, Timothy C. 32, 603, 607, 

609, 1081 

Nicklin, Stuart A. 851, 1057 

Nicolais, Luigi 683 

Nie, Suhua 1069 

Nielsen, Petur G. 160 

Niemeyer, Glenn P. 962 

Nienhuis, Arthur W. 10, 93, 387, 831 

Nierman, Melchior C. 907 

Nietupski, Jennifer B. 910 

Nightingale, Sarah 1102 

Niidome, Takuro 206, 213 

Niidome, Yasuro 213 

Nikhamin, Yan 799 

Nikles, David E. 883 

Nikolai, Bryan 838 

Nilsson, Berith 1024 

Nimura, Keisuke 452 

Nishikawa, Makiya 71, 701 

Nishikawa, Tomoyuki 1119 

Nishimoto, Norihiro 679 

Nishimura, Ken 107 

Nishino, Tamon 993 

Nishiyama, Akiyo 253 

Niu, Yunyun 729 

Niven, Tiffany B. 65, 67 

Nivsarkar, Megha 238, 394 

Niwa, Hitoshi 255 

Nixon, Alan J. 674, 675 

Niyibizi, Christopher 889 

Noble, Charles 124 

Nolan, Garry P. 837 

Noll, Meenakshi 1046 

Nolta, Jan A. 212, 243, 288, 353, 

457, 460, 783, 810, 

927, 1031, 1032, 1035, 

1037, 1121 

Nomoto, Tatsuya 616, 663, 938 

Nonaka-Sarukawa, Mutsuko 868 

Nordlund, Henri R. 758



Norman, B. 924 

Norona, Wilma 535 

Norris, James S. 307, 450, 1011 

Notka, Frank 764 

Nougirat, Cynthia 465 

Noureddini, Sam C. 30, 1130 

Novelli, Francesco 349 

Novelli, Giuseppe 568, 651 

Novikov, Mikhail 50 

Novo, Javier F. 784 

Nukiwa, Toshihiro 69 

Nunes, Fabio P. 970 

Nunnari, Giuseppe 999 

Nutting, C. M. 1135 

Nwanegbo, Edward C. 150 

Nyanguile, Origene 623, 879, 918, 919 

Obenauer-Kutner, Linda J. 414 

O’Brien, Timothy 490, 550, 632, 634 

O’Brien, William 903 

Ochalski, Rafal 651 

Ochiai, Hiroshi 566 

Ochiya, Takahiro 199 

Ochoa, Laura 547 

O’Connor, Michael K. 1014 

O’Connor, Timothy P. 814 

Ochs, H. 341 

Odaka, Yoshinobu 274 

O’Day, Terry 1125 

O’Donnell, Patty 32 

Oender, Kamil 1065 

Ogawa, Koichi 242 

Ogawa, Rei 1071 

Ogura, Tsuyoshi 125, 860, 862, 900 

Oh, Jin Hwan 122 

Oh, Jong-yeong 168 

Oh, Jong-Young 167 

Oh, Seungjoon 1060 

Oh, Seunguk 445 

Oh, Sung-Min 278 

Oh, Taekeun 932 

O’Hagan, Diana 1133 

Ohashi, Kazuo 597 

Ohashi, Masaki 708 

Ohashi, Toya 993 

Ohbayashi, Fumi 23 

Ohga, Shoichi 103 

Ohlfest, John R. 445 

Ohyama, Manabu 1118 

Oi, Keiji 47 

Oishi, Jun 206 

Oka, Kazuhiro 638 

Okada, Masaho 923 

Okada, Takashi 125, 616, 663, 860, 

862, 868, 900, 938 

Okamura, Takayuki 979 

Okano, Motohiko 35 

Okuno, Norihiko 286 

Okuyama, Hiromi 486 

Olague, Cristina 1134 

Olivares, Eric C. 194, 265 

Oliveira, Lisa B. 970 

Olmsted-Davis, Elizabeth A. 681, 892 

Olsen, John 752 

Olsen, John C. 828 

Olson, Erik 386, 987 

O’Malley, Mark E. 755 

O’Malley, Thomas 240, 247, 421 

Onal, Tankut 1117 

Ong, Hooi-Tin 297, 303 

Ong, John M. 698 

Ono, Hidetaka A. 1020 

Onodera, Masafumi 35, 344 

Opolon, Paule 188, 524 

O’Riordan, Catherine R. 110, 497 

Orme, Ann 958, 959 

Ortiz-Lopez, Rocio 298, 553 

Osako, Mariana Kiomy 624, 631, 685 

Osawa, Kanako 742 

Osborne, William 459 

O’Shea, Ciara A. 490 

Oshima, Hideki 920, 923 

Oshimura, Mitsuo 742 

O-Sullivan, InSug 697 

Otaegui, Pedro J. 901 

Othman, Maha 410, 610 

Otsu, Makoto 35 

Ott, Marion G. 332, 336, 1076 

Otto, Ed 293 

Ovechkina, Y. 341 

Overlid, Nathan 590 

Owaki, Toshiyuki 842 

O-Wang, Jiyang 282 

Owens, Roland A. 404 

Oyeyumi, Moses 223 

Ozasa, Shiro 251, 252, 255 

Ozawa, Keiya 125, 517, 616, 663, 

860, 862, 868, 900, 938 

Ozturk, Ferhat 750 

Ozu, Choichiro 710 

Ozuer, Ali 757 

Pabo, Carl O. 379 

Pacak, Christina A. 44 

Pacenti, Monia 536 

Padmanabhan, Prabhu M. 45 

Painter, Hazel 362 

Paiva, Artur 222 

Paiva, Melissa 694 

Palanker, Daniel V. 770 

Palmarini, Massimo 886 

Palmer, Donna J. 143, 273, 363, 365, 

430, 607, 815, 885, 

903, 904, 905, 906 

Palmer, Glyn D. 835, 889 

Palmer, Lorne 996 

Palmieri, Lucia 358 

Palomeque, Julieta 850 

Palù, Giorgio 536 

Pan, Dao 6 

Pan, Dongfeng 315 

Pandey, Kusum 87, 857 

Pandha, H. S. 1135 

Panet, Amos 806 

Pang, Cho Y. 363 

Pang, Jijing 741 

Pannier, Angela K. 204 

Pantaleo, Giuseppe 764 

Panteleyev, Andrey 1123 

Papa, Milton 466 

Papayannopoulou, Thalia 348, 465 

Papo, Orit 735 

Paques, Frederic 417, 902 

Pardini, Silvia 121 

Parekh-Olmedo, Hetal 380, 384, 

385, 1056 

Parikh, Harsh 576 

Park, Byeong-Ho 167, 168 

Park, Dongsu 1132 

Park, Eui-chul 992 

Park, Eun-Jung 717 

Park, Frank 936 

Park, John W. 124 

Park, Julie 191, 1084 

Park, Keerang 717 

Park, Mahnhoon 1003 

Park, Paul 750 

Park, Tae Gwan 207 

Park, Yong Serk 556, 728 

Parker, Amy E. 443, 997, 1000 

Parsley, Katherine 31 

Parsons, David W. 360 

Partridge, Terrence 788 

Passa, Ourania 209 

Passineau, Michael J. 1017 

Passini, Marco A. 110, 427, 497 

Pastore, Lucio 683

136 



Patel, Manij 752, 828 

Patrick, Michael 293 

Pattarini, Lucia 114, 121 

Patterson, Noelle B. 989 

Patterson, Sarah 85, 379 

Paulson, Henry L. 52 

Payne, Thomas R. 920, 923 

Paz, Helicia 19 

Pearse, Damien D. 662 

Peder, Bergvall 174 

Pedersen, Mikkel W. 730 

Pedersen, Nina 730 

Pedro, Berraondo 976 

Pedroso de Lima, Maria C. 222, 302 

Peeters, Liesbeth 580 

Pegoraro, Gianluca 852 

Pelled, Gadi 766, 1053 

Pellicciotta, Ilenia 179 

Pellinen, Riikka 97, 323 

Peltomaa, Erik 758 

Peluso, Richard 869 

Pena, John 940 

Peng, Hairong 923 

Peng, Jian Qiang 127 

Peng, Kah-Whye 73, 297, 303, 

721, 1010 

Peng, Weidan 777 

Penn, Marc S. 917 

Penuelas, Ivan 320, 808 

Pepper, Karen A. 90, 101, 462, 

805, 1102 

Perabo, Luca 502, 863 

Pereboev, Alexander V. 274, 679, 1017 

Pereboeva, Larisa 528, 542, 1018 

Perera, O. P. 422 

Perez, Antonio 235 

Perez, Christophe 417 

Perez, Elena 974 

Perez, Norma 261 

Pergolizzi, Robert G. 335, 853 

Perouzel, Eric 593 

Perret, Christine 320 

Perri, Sabrina R. 312 

Perricaudet, Michel 524 

Persons, Derek A. 10, 93, 1030 

Perutka, Jiri 378 

Pescarollo, Alessandra 819 

Petek, Lisa M. 15 

Peter, Moore M. 174 

Petersen, Amy S. 529 

Peterson, Daniel A. 660 

Petersen, Denise 37, 90, 101 

Peterson, Denise 462 

Peterson, Laura J. 455, 456 

Petit, Caroline 92 

Petras, Ognjen 501 

Petravicz, Jeremy 164 

Petroni, Debora 121 

Petry, Harald 170, 172, 715, 

958, 960 

Pham, Phuong Lan 518 

Phelps, Michael E. 808 

Philippe, Moullier 429 

Philippe, Stephanie 92 

Pierce, Glenn 130, 603, 614, 658 

Pierimarchi, Pasquale 706 

Pike, Lisa 164 

Pilapil, Carmencita G. 835, 1120 

Pilewski, Joseph 817 

Pintel, David J. 870 

Pinto, Marta 158 

Pipe, Steven W. 598 

Pitt, Bruce R. 586 

Pittaluga, Stefania 387, 1133 

Pivirotto, Philip 942 

Piwnica-Worms, David 212, 783 

Plantinga, Josee 408 

Plinkert, Peter K. 665 

Podhajcer, Osvaldo 78 

Podjarny, Eduardo 342 

Poenaru, Livia 233 

Pohlschmidt, Marita 258, 784 

Poirier, Amy E. 134, 1077 

Poliseno, Laura 370 

Polisky, Barry 438 

Politi, Letterio S. 972 

Polito, Vinicia 250 

Pollak, Eleanor S. 606 

Pollok, Karen E. 2, 474 

Polyak, Boris 930 

Pomerantz, Roger J. 999 

Pommey, Sandra 268 

Ponder, Bruce A. J. 310 

Ponder, Katherine P. 32, 239, 247, 

421, 602 

Pondugula, Krishnakant 540 

Ponnazhagan, Selvarangan 119, 136, 

507, 542, 849 

Pope, Melissa A. 763 

Porada, Christopher D. 750 

Porter, Colin 484 

Porter, Forbes 232 

Portlock, Joylette L. 194, 622 

Porvasnik, Stacy L. 422, 1117 

Potter, Murray A. 220 

Poulard, Karine 794 

Poulsen, Hans S. 730 

Poulsen, Thomas T. 730 

Power, John 41 

Praetorius, Mark 60, 665 

Preece, Jon A. 197 

Prenovitz, Robert 357 

Preuss, Meredith A. 883, 1017 

Prieto, Jesus 320, 547, 555, 

759, 808, 876, 1134 

Pringle, Ian A. 366 

Printz, M. 950 

Printz, Marie A. 947 

Prinz, Claudia 33 

Przybylowski, Mark 558 

Puddy, Alan C. 893 

Pujol, Anna 248 

Pule, Martin 798, 801 

Puntel, Mariana 143 

Puttaraju, M. 293, 1113 

Putzer, Brigitte M. 310 

Qasim, Waseem 796 

Qian, Cheng 547, 722, 731, 

808, 876, 1134 

Qian, Hu Sheng 715, 768, 958, 

959, 960 

Qian, Jun 361 

Qian, Qi-Jun 722, 731 

Qian, Zhong 936 

Qiang, Mei 617 

Qiao, Chunping 398, 792, 916, 1125 

Qiao, Jian 271, 449, 723, 804 

Qijun, Qian 705 

Qin, Xiao-Feng 95 

Qing, Keyun 11, 16, 401 

Qiu, JianPing 975 

Quattrini, Angelo 426, 972 

Quax, Paul H. 1112 

Quenneville, Simon P. 257 

Quiambao, Alexander B. 668 

Quijano, Erlinda 749 

Quintero, Jesse 293 

Quinton, Paul M. 363 

Quiroga, Jorge 1134 

Qun, Li 595 

Raben, N. 245 

Rabinowitz, Adam 788 

Rabkin, Samuel D. 160, 761 

Radcliffe, Pippa A. 55, 395, 599 

Raf, Lemmens 174 

Rafii, Shahin 147



Rahim, Ahad 209, 484 

Rainaldi, Giuseppe 370 

Raizada, Mohan 627 

Rajagopal, Ramesh 809 

Rajecki, Maria 1010 

Rajnish, Dave S. 999 

Raki, Mari 299, 328, 1004, 

1026, 1028 

Rakoczy, Elizabeth P. 434 

Ralph, Gareth S. 55 

Ramakrishnan, Sriram 177, 181, 

182, 574 

Ramesh, Rajagopal 287, 290, 691 

Ramezani, Ali 89 

Ramirez, Genaro 381 

Ramos-Kuri, Manuel 850 

Ramsey, Angela 227 

Ramunas, John 79 

Rando, Thomas A. 262, 265, 622, 789 

Ranki, Tuuli 299, 328, 1004, 1026 

Raoul, Cedric 54 

Raphael, Yehoash 664 

Rapoza, Robert 928 

Rascoe, Philip 301 

Rasooly, Linda 806 

Ratnayake, Maheshika K. 800 

Raty, Jani K. 165 

Raupp, Sylvia 475, 836 

Raut, Sanj 779 

Rawlings, D. 341 

Rawlings, David J. 1048 

Raykin, Julia 483 

Raymond, Benoît 884 

Read, Martin L. 197 

Reay, Daniel P. 1086 

Rebar, Edward J. 43, 85, 379, 974, 1072 

Rebello, Tessio 990, 1075 

Rebollo, Patricia 20 

Recchia, Alessandra 819 

Recchia, Fabio 1087 

Reddick, Robert A. 617 

Reddy, P. Seshidhar 538, 545 

Reddy, Seshidhar P. 725 

Reed, Sharon E. 870 

Reid, Tony 311 

Reier, Paul J. 654 

Reik, Andreas 1072 

Reilly, Raymond M. 75 

Reimsnider, Sharon K. 1082 

Reineke, Theresa M. 782 

Reiser, Jakob 482, 493 

Remaley, Alan T. 1113 

Remaut, Katrien 774 

Reme, Thierry 739 

Remsey-Semmelweis, Ernoe 754 

Remy, Jean Serge 583 

Ren, Changchun 119, 849 

Ren, Chenghui 275, 695 

Ren, Chengzhen 275, 695 

Ren, Lufeng 108 

Rentsendorj, Altan 211, 1108 

Rettig, Garrett R. 223 

Rettig, Michael P. 709, 810 

Rex, Tonia S. 117 

Reyes, Beverley S. 659, 661 

Reyes, Morayma 1040, 1127 

Reynolds, Ann M. 811 

Reynolds, Paul N. 811, 1017 

Rhee, Byoung Doo 1060 

Rhee, Gyu Seek 827 

Rhee, Melanie 201 

Ribas, Antoni 61 

Ribeiro, Marcia G. 970 

Rice, Jennifer 279 

Rice, Karen 815, 906 

Rice, Kevin G. 198, 223, 1106 

Rice, Michael 651 

Richard, Robert E. 987 

Richter, Jose A. 808 

Ricketts, Melisa 63 

Riddle, David 704 

Rieger-Christ, Kimberly M. 689 

Riley, Jim 974 

Rinaldi, Monica 706 

Rip, Jaap 907 

Ristimaki, Ari 1004 

Ritchey, Julie K. 288, 709, 810 

Riu, Efren 563 

Rivella, Stefano 4 

Rivera, Angel A. 537, 540, 1007, 

1018, 1029 

Rivera, Victor M. 117, 466 

Riveros, María D. 696 

Rives, Christopher B. 769 

Rivière, Christel 794 

Riviere, Isabelle 277, 558, 707 

Robbins, Paul D. 66, 150, 294, 300, 

674, 675, 817, 891 

Roberts, Melanie A. 534 

Robertson, Jen 694 

Robicchaux, Myle 977 

Robinet, Eric 739 

Robison, Clinton S. 657 

Rock, Jeremy 85, 337, 379, 974 

Rockenstein, Edward 56, 57 

Rodarte, Charles 301 

Rodijk, Mark 543, 1005 

Rodriguez, Carlos 759 

Rodriguez, Edgardo 53, 952, 954 

Rodriguez, Ronald 289 

Rodriguez-Madoz, Juan R. 876 

Roehl, Holger 825 

Roelvink, Peter W. 437, 443, 975, 

997, 1000, 1088 

Roesler, Joachim 234 

Rogers, Faye 369 

Rojas-Martinez, Augusto 298, 553 

Rolland, Sandra 902 

Roncarolo, Maria Grazia 36, 330, 331, 

345, 468 

Rooney, Cliona M. 795, 798, 800, 

801, 979, 1074 

Roques, Caroline 229 

Rosal, Marta 432 

Rosen, Antony 672 

Rosencrance, Dana 561, 575 

Rosenqvist, Nina 91 

Rosenthal, Felicia 1070 

Rosenzweig, Anthony 40, 46, 50 

Rosic-Kablar, Suzana 1036 

Rosova, Ivana 927 

Ross, Colin J. 907 

Ross, David L. 839 

Rossi, Claudia 83 

Rossi, J. J. 8 

Rossi, John 440 

Rossi, John J. 436, 437, 743, 998 

Rothschild, Barbra Bluestone 1078 

Rothstein, Jeffrey D. 650, 652, 653 

Rots, Marianne G. 314, 408, 753, 1073 

Rottier, Peter J. M. 9 

Rousseau, Joël 102, 257, 690 

Rowe, Peter 485, 988 

Rowe, Thomas 1092 

Roy, Brigitte 1079 

Roy, Krishnendu 225 

Roy, Raynald 1079 

Roy, Soumitra 872 

Rozemuller, Henk 461 

Rubanyi, Gabor M. 170, 172, 715, 768, 

958, 959, 960 

Rubinchik, Semyon 1058 

Rueger, Maria A. 77 

Ruiz, Juan 1134 

Ruiz, Maria 808 

Rusch, Valerie W. 762

138 



Rusconi, Sandro 1070 

Russell, David W. 15, 386, 987 

Russell, Stephen J. 47, 73, 297, 303, 

719, 720, 721, 846 

Ruthe, Alaine 394 

Rutishauser, Maja 332 

Ruzicka, Thomas 309 

Ruzo, Albert 248 

Ryser, Martin F. 234 

Ryu, Byoung Y. 10 

Saamoah-Moffatt, Stanley 780 

Sabatino, Denise 609, 614 

Sachabhipulkij, Karishma 225 

Sachdeva, Geetanjali 476 

Sadaba, Belen 1134 

Sadelain, Michel 4, 277, 558, 

604, 707, 799 

Sadilek, Martin 409 

Safinya, Cyrus R. 771 

Saijo, Yasuo 69 

Sakae, Mitsuko 224 

Sakaguchi, Gaku 154 

Sakaguchi, Takemasa 107 

Sakai, Tetsuro 920 

Sakai, Yoshi 1117 

Sakata, Susumu 45 

Sakata, Yoichi 125, 860, 862 

Sakata, Yuri 45 

Sakiyama, Yukio 35 

Sakiyama-Elbert, Shelly E. 219 

Sakurai, Fuminori 525, 527, 888 

Salama, Salama A. 259, 264 

Salazar, Felix H. 441 

Salmon, André 229, 934 

Salo, Jarmo 1026 

Salonen, Jukka I. 1095 

Salonen, Tuula 97 

Salva, Maja Z. 266 

Salvatore, Francesco 683 

Salvetti, Anna 512 

Salvi, Alessandro 114 

Samali, Afshin 550 

Sambuceti, GianMario 121 

Sampaolesi, Maurilio 446 

Samuel, Charles E. 771 

Samulski, R. Jude 12, 16, 18, 343, 

403, 464, 854, 958, 959 

Sanchez, Andre R. 662 

Sanchez-Perez, Luis 267, 449, 804 

Sanders, Christine T. 660 

Sanders, Niek N. 580, 594 

Sandhu, Arbans 872 

Sands, Mark S. 237, 243, 424, 460 

Sanford, Jeremy R. 534 

Sanftner, Laura M. 658 

Sangiuolo, Federica 568, 651 

Sangro, Bruno 808, 1134 

Sanmiguel, Julio 500, 506, 1092 

Sano, Akihiko 199 

Sano, Akiko 742 

Santat, Leah 19 

Santegoets, Saskia J. A. M. 1130 

Santiago, Francisco 994 

Santoni de Sio, Francesca 1038 

Santos, Elmer 797 

Sanvito, Francesca 389 

Sanz, Laura 704, 802 

Sapinoro, Ramil E. 533 

Sapru, Mohan K. 944 

Sarkadi, Balazs 1045 

Sarkar, N. 924 

Sarkar, Rita 200, 779 

Särkioja, Merja 299, 328, 1004, 

1010, 1026 

Sarkis, Chamsy 92 

Sarsero, Joseph P. 957, 967 

Sartori, Daniela 819 

Sarukawa, Mutsuko 616, 938 

Sasahara, Yoichi 344 

Sasaki, Erika 472 

Sasaki, Kentaro 587 

Sasaki, Tomomi 525 

Sastry, Lakshmi 2, 494, 825 

Sati, Fumiaki 325 

Sato, Eigo 489 

Sato, Makoto 1008, 1051 

Sato, Naoyuki 945, 948 

Satyamurthy, Nagichettiar 808 

Sauer, Julie A. 496 

Savoldo, Barbara 798, 801 

Sawicki, Janet A. 777 

Saydam, Okay 724 

Scaldaferri, Lucia 568 

Scanga, Charles 349 

Scappaticci, Frank A. 445 

Scaramuzza, Samantha 330, 345 

Scaria, Puthupparampil V. 318 

Schaefer, Caralee 960 

Schaffer, David V. 488, 519, 

1002, 1085 

Schambach, Axel 829 

Scharenberg, Andrew M. 1048 

Scheffner, Martin 526 

Scheidel, Jason 561, 575 

Schenkwein, Diana 758 

Scheper, Rik J. 1130 

Scherr, Michaela 368 

Scheule, Ronald K. 531 

Schick, Bernhard 665 

Schierer, Stephan 1007 

Schiffelers, Raymond M. 318 

Schilz, Andrea 336, 495 

Schlachterman, Alexander 13, 14, 84, 

397, 608, 

612, 1114 

Schlemminger, Stefanie 502 

Schlesinger, Sondra 845 

Schmidt, Anke 310 

Schmidt, Erika 27 

Schmidt, M. 1055 

Schmidt, Manfred 33, 394, 1076 

Schmidt, Michael K. 396 

Schmidt, Sonja 33 

Schmidt-Wolf, Ingo G. H. 284 

Schmittner, Nicola 309 

Schmitz, Joern E. 63 

Schneider, Ayn 912, 1124 

Schneider, Richard M. 846 

Schnell, Matthias J. 999 

Schofield, Timothy L. 893 

Schorderet, Daniel F. 667 

Schorr, Joachim 565 

Schowalter, David B. 921 

Schroeder, A. Rosanna 443, 1000 

Schroeder, Annette R. 997 

Schroeder, Mark 720 

Schuchman, Ed H. 110, 249 

Schuele, Silke 7, 475, 836 

Schuettrumpf, Joerg 603, 1114 

Schuler, Gerold 1007 

Schult-Dietrich, Patricia 63 

Schultheis, Beate 368 

Schultz, Gregory S. 861 

Schumer, Gregory 141 

Schwantes, Astrid 846 

Schwartz, David A. 450 

Schwartz, Fletcher 914 

Schwartz, Timothy 384 

Schwarzwaelder, Kerstin 33, 1076 

Schweizer, Matthias 7, 475, 836 

Sciascia, Sandra A. 143 

Scollard, Deborah 75 

Scollenbergen, Lisa 776 

Scudiero, Dominic A. 716 

Scull, Samantha E. 531 

Seeliger, Mathias 434



Seethammagari, Mamatha R. 1132 

Segal, David J. 371 

Segawa, Hiroaki 107 

Seger, Reinhard 332, 336, 1076 

Seggewiss, Ruth 387 

Segovia, Jose Carlos 236 

Segura, Tatiana 217 

Seiler, Michael P. 885 

Seitz, William A. 588 

Sekiguchi, Keishi 624, 685 

Selby, Peter 271 

Seleme, Marie del Carmen 1093 

Selimovic, Dennis 309 

Senadheera, Dinithi 101, 463 

Sena-Esteves, Miguel 866, 961 

Senay, Hélène 1079 

Seok, Ji hyun 827 

Seol, Dai-Wu 294, 300 

Seregina, Tatiana 1133 

Sergi Sergi, Lucia 36, 446 

Serguera, Che 92 

Serose, Armelle 229, 934 

Serra, Delila 407, 1094 

Serriello, Rebecca G. 531 

Sevin, Caroline 429 

Seya, Tsukasa 68 

Seyment, Oktay 894 

Seymour, L. 1027 

Seymour, Leonard W. 197 

Sferra, Thomas J. 246 

Shabtay, Ariel 931 

Shackleton, Cedric 232 

Shafer, Jessica A. 892 

Shaffer, Chris S. 438 

Shaffer, James 276 

Shah, Maulik R. 285 

Shah, Neelima 779 

Shai, Ela 837 

Sham, Mai Har 305, 352 

Shani, Noam 342 

Shankar, Veijay 547 

Shanker, Manish 290 

Shansky, Janet 40 

Shaochun, Song 241 

Shaw, Denise R. 849 

Shaw, Janet R. 582 

Shaw, Kit L. 90 

Shaw, Lucinda 438 

Shaw, Paul A. 228 

Shayakhmetov, Dmitry M. 137, 138, 

348, 409, 

411, 880 

Shea, Lonnie D. 204, 769, 775, 1107 

Sheard, Victoria 1044 

Shelburne, Charles E. 677 

Shelley, W. Christopher 374 

Shellito, Judd E. 977 

Shen, Feng 220 

Shen, Xuan 405 

Shen, Y. 1027 

Shen, Zhenping 785 

Sheren, Jamie E. 1099 

Shewach, Donna S. 807 

Sheykholeslami, Kianoush 663 

Shi, Changxin 694 

Shi, Juan 305 

Shi, Wenfang 72, 74, 113, 

291, 509 

Shihabuddin, Lamya S. 110 

Shimada, Kazuyuki 616, 938 

Shimada, Takashi 3, 93, 242, 618, 

968, 1071 

Shimamura, Munehisa 945, 948 

Shimazaki, Kuniko 663, 938 

Shimozato, Osamu 282 

Shimpo, Masahisa 616, 938 

Shin, Duckhyang 1003 

Shin, Hyun-chul 992 

Shin, In-Soon 625, 1052 

Shine, H. David 964, 647 

Shinkai, Seiji 214 

Shirakawa, Toshiro 546 

Shivakumar, Rama 192 

Shizuru, Judith A. 467 

Shklayev, Stanislav 400, 418 

Shoemaker, Robert H. 716 

Shorrock, C. A. 1135 

Shou, Yan 454, 1030 

Shroff, Geeta Barthakur 971 

Shu, Chih-Chien 280, 281 

Shungu, Dikoma C. 969 

Shurman, Shepherd H. 333 

Shuto, Tusyoshi 913 

Shyur, Lie-Fen 280, 281 

Si, Yue 470 

Sibley, Don A. 25 

Siddiqui, M. A. Q. 754 

Siegal, Gene P. 537, 1029 

Siegal, Tali 806 

Siegfried, Jill 729 

Sieler, Ulrich 1076 

Sierts, Jeroen A. 907 

Sievers, Richard E. 933 

Sigg, Daniel C. 864 

Signori, Emanuela 706 

Siler, Ulrich 332, 336 

Sillevis Smitt, Peter A. E. 543, 1005 

Silva-Platas, Christian I. 553 

Simões, Sérgio 222, 302 

Simon, Greg G. 914 

Simonenko, Vera 30 

Simpson, Ann M. 899 

Simpson, J. 1135 

Sinagra, Gianfranco 114 

Sinclair, Jo 31 

Sinclair, Molly E. 198 

Singer, Oded 56 

Singh, Charanjit 356 

Singh, Nalini 711 

Singh, Surjeet 197 

Sinn, Patrick L. 1, 816 

Sipler, Matthew 605 

Skaggs, Jeff 668 

Skelton, Dianne 805 

Skuk, Daniel 1079 

Sleat, David E. 427 

Slepushkin, Vladimir 477, 990, 1075 

Slepushkina, Tatiana 477 

Sloane, Evan M. 644, 645 

Slobodkin, Gregory 227, 279 

Sluiter, Wim J. 1112 

Smart, Larry 274 

Smerdou, Cristian 876 

Smirnow, Irina 840 

Smit, Andries J. 1112 

Smith, Alexander J. 391 

Smith, Elisheva 1121 

Smith, Jeffrey S. 144 

Smith, Julianne 417 

Smith, Peter 130 

Smith, Peter H. 658 

Smith, Rebecca L. 362, 366 

Smith, Richard 747 

Smith-Powell, Leslie 402 

Smyth, Christine 839 

Smythe, W. Roy 301 

Snoeys, Jan 413, 530, 548, 

636, 1115 

Snyder, Richard O. 400, 1077 

So, Po-Wah 356 

Soda, Yasushi 103, 472 

Sohn, Joo Hyuk 551, 1019 

Soifer, Harris S. 1093 

Sokolic, Robert A. 458 

Sola, Josu 1134 

Soleimani, Masoud 1049

140 



Soloff, Adam 66 

Somanathan, Sury 872 

Somerton, Luci 356, 842 

Somia, Nikunj V. 826, 838 

Sommer, Jurg M. 658 

Sondhi, Dolan 427, 660, 969 

Sone, Tomomichi 322 

Song, Anthony 110, 497 

Song, In-Ho 556, 728 

Song, Sihong 511, 514, 1077 

Songsungthong, Warangkhana 106 

Sonne Holm, Per 532 

Sood, Vandana 582 

Soong, NayWei 984 

Sorace, Oreste 121 

Sorba, Tyler 85 

Sorrentino, Brian P. 387, 454, 1030 

Sorscher, Eric J. 507 

Southerland, Hilary A. 431 

Southgate, Thomas D. 1044, 1047 

Souweidane, Mark 969 

Souza, D. 245 

Sova, Pavel 1006 

Sowers, Anastasia L. 471 

Soykan, Orhan 864 

Soylemez, Mehment Ali 894 

Spaapen, Robbert 461 

Spencer, Brian J. 428 

Spencer, Carolyn T. 1117 

Spencer, David M. 1132 

Spencer, Jacqueline F. 1021, 1022, 1023 

Spitalieri, Paola 568 

Splinter, Patrick 721 

Spratt, S. Kaye 43 

Springer, Matthew L. 933 

Springfeld, Christoph 106, 732, 843 

Srinivas, Sai 402 

Srinivasan, Rahul 641 

Srivastava, Arun 11, 16, 401 

Srour, Edward F. 470 

Staber, Patrick D. 52 

Stabile, Laura 729 

Stachelek, Stanley J. 919 

Stachler, Matthew 113, 848 

Staecker, Hinrich 60, 665, 875 

Stagg, John 268 

Staley, Elizabeth M. 870 

Stamatoyannopoulos, George 81, 348, 

388, 824 

Stan, LeRoy 858 

Stantz, Keith M. 1009 

Stargel, Amanda 448 

Stauss, Hans 796 

Stayton, Patrick 1111 

Stedman, Hansell H. 42, 603 

Steel, Jason C. 26 

Stefani, Anna-Lisa 536 

Stefaniak, Maureen E. 989 

Stefanski, Jolanta 707 

Steidl, Stefanie 475 

Stein, Colleen S. 601 

Stein, Stefan 332, 336, 1076 

Steiner, J. 1135 

Stenman, Ulf-Hakan 299, 1010 

Stenzel, Werner 77 

Stepanova, Lilia 454 

Stephan, Matthias 604, 797 

Stephan, Sharon A. 496 

Sterman, Dan H. 1129 

Steven, N. M. 1135 

Stevenson, Mark 197 

Stewart, Gregory R. 110, 427, 497 

Stice, Steven 892 

Stiefelhagen, Marius 865 

St-Laurent, Gilles 518 

Stocker, Alice G. 360 

Stoddard, Barry L. 1048 

Stoff, Alexander 1018 

Stoff-Khalili, Mariam A. 1018, 1029 

Stone, Daniel 411, 1091 

Storb, Rainer J. 791 

Storm, Theresa A. 87, 405, 504, 516 

Straathof, Karin C. 801 

Strappe, Padraig 490 

Strassheim, Mary L. 675 

Stratford, Ian J. 327 

Stratton, Jamie A. 660 

Strauss, Eric 674 

Strauss, Robert 980, 1006 

Strayer, David S. 175, 659, 661, 813, 

965, 985, 1043, 1062 

Streetz, Konrad L. 87, 467, 857 

Strehl, John W. 284 

Stricker, Hans 153 

Stripecke, Renata 61, 982, 1054 

Strom, T. 341 

Strong, Theresa V. 849 

Stuass, Hans J. 171 

Studeny, Matus 447, 727 

Su, Hua 619, 926 

Su, Leonard T. 42 

Subramanian, Thyagarajan 1050 

Subtil, Jose 1134 

Suda, Takeshi 1096 

Sueishi, Katsuo 833 

Sugiura, Wataru 489 

Suh, Beum-Seok 702 

Suh, Wonhee 625, 1052 

Suhy, David A. 437, 443, 997, 1000 

Sukonnik, Tanya 79 

Sullenger, Bruce A. 377, 378 

Sullivan, Mark A. 966 

Sumimoto, Hideki 103 

Summerhayes, Ian C. 689 

Summers, James B. 1100 

Sumners, Colin 627 

Sun, Baodong 912, 1124 

Sun, Fei 817 

Sun, Huijie 891 

Sun, Jin 1129 

Sun, Minli 230 

Sun, Yuansheng 846 

Sunyog, April M. 760 

Surace, Enrico M. 435, 669 

Suter, Steven E. 803 

Sutjipto, Suganto 414 

Suwanmanee, Thipparat 343 

Suzanne, Greiner 721 

Suzuki, Brian M. 658 

Suzuki, Eiji 1129 

Suzuki, Misao 251 

Swan, Christina H. 973, 1001 

Swiderski, Donald L. 664 

Sylven, C. 924 

Sylvester, Karl 467 

Szulc, Zdislaw 1011 

Szymanski, Paul 768, 958, 959, 960 

Szyroki, Alexander 63 

Taba Jr, Mario 677 

Tabata, Toshiaki 833 

Tabata, Yasuhiko 49, 216 

Tabron, Dorothy 232 

Tabucchi, Antonella 331 

Tacchetti, Carlo 435 

Tada, Yuji 282 

Tafuro, Sabrina 126 

Tagawa, Akihiro 841 

Tagawa, Masatoshi 282 

Tahir, Salahaldin A. 275 

Tai, Ming-Hong 549 

Tai, Phillip 266 

Taira, Kazunari 592 

Tak, Paul P. 1122 

Takahashi, Masuhiro 270 

Takahashi, Tomoyuki 58, 473, 929



Takahashi, Yuki 71 

Takaki, Miyako 45 

Takakura, Yoshinobu 71, 701 

Takakusaki, Yukiko 968 

Takashima, Sachio 58 

Takayama, Koichi 154, 322, 364 

Takayama, Kozo 107 

Takeda, Shin’ichi 253 

Takemura, Genzou 929 

Takeshima, Yasuhiro 252 

Takeshita, Fumitaka 199 

Takiguchi, Yuichi 282 

Taksir, T. 245 

Taksir, Tatyana V. 110 

Tam, Paul 123, 305, 352 

Tam, Sidney 123 

Tamai, Katsuto 216, 452, 1119 

Tamas, Laszlo 124 

Tan, Charles Y. 893 

Tan, Langzhu 479 

Tan, Peng H. 171, 773, 981, 1042 

Tan, Siyuan 646 

Tan, Wenjie 393 

Tanaka, Naoko Iida 224 

Tanaka, Ryuichi 270 

Tang, Jin Bo 680, 684, 689 

Tang, Nan 75 

Tang, Susan 984 

Tang, Waixing 135 

Tang, Ying 254 

Tang, Yucheng 1128 

Tani, Kenzaburo 103, 154, 472, 886 

Tanioka, Yoshikuni 472 

Tao, Chang 899 

Tao, Lucy 881 

Tapscott, Stephen J. 791 

Tarantal, Alice F. 462, 577, 743 

Tasciotti, Ennio 121 

Tatsumi, Koichiro 282 

Tatsuno, Gwen 130 

Tatsuzawa, Osamu 35 

Tavares, Isaura 158 

Tavassoli, Mahvash 307 

Taylor, Jason 987 

Taylor, Kristin M. 531 

Taylor, Sarah L. 484 

Tazelaar, Henry D. 47 

Tedford, Nathan C. 205 

Teh, Bin S. 286, 695 

Tei, Kumiko 618 

Tellez, Joe 750 

TenBroek, Erica 864 

Tenenholz, Todd 1100 

Teng, Qingshan 433, 482, 855, 1050 

Tennant, Lucinda 1133 

Teodósio, Cristina 222 

Teraishi, Fuminori 539 

Teramoto, Kenichi 489 

Terao, Keiji 833 

Terwilliger, Ernest F. 120, 522, 859 

Tessitore, Alessandra 240, 669 

Thanarajasingam, Uma 802, 804 

Thanou, Maya 215 

Themis, Matthew 394 

Themis, Michael 238, 394 

Therrien, Jean-Philippe 1118 

Theys, Jan 329 

Thomas, Clare 946 

Thomas, Gail D. 251 

Thomas, Maria A. 1021, 1022, 1023 

Thomas, Stuart P. 839 

Thompson, E. Brad 588 

Thompson, Jill 267, 271, 449, 

704, 802 

Thompson, Timothy C. 275, 695 

Thony, Beat 898 

Thorne, Stephen 311 

Thorne, Steve H. 167, 168, 169, 756 

Thorpe, Robin 105 

Thorrez, Lieven 40 

Thrasher, Adrian J. 31, 33, 330, 

345, 391, 394, 796 

Thresher, Randy J. 478 

Thurberg, B. L. 245 

Thurn, Tanja 120, 522 

Thyagarajan, Bhaskar 194 

Tian, Chaorui 469, 1033 

Tian, Jie 144 

Tian, Mengnan 792 

Tian, Weihua 275 

Tiberghien, Pierre 739 

Tidona, Christian A. 843 

Tigges, Michael 614 

Tilapur, Uday 197 

Timiryasova, Tatyana 155 

Timme, Terry L. 275, 695 

Timmers, Adrian M. 1080 

Tio, Rene A. 1112 

Tirapu, Inigo 1134 

Tisdale, John F. 1041 

Tiziana, Cervelli 17 

To, Richard 1111 

Tobita, Kimimasa 920, 923 

Tock, Christine L. 1118 

Toietta, Gabriele 535 

Toita, Nariaki 35 

Toivanen, Pyry I. 758 

Tojo, Arinobu 103 

Tollefson, Ann E. 1021, 1022, 1023 

Tolmachov, Oleg 209 

Tolstrup Nielsen, Troels 91 

Tomita, Naruya 624, 631, 685 

Tomita, Tetsuya 685 

Tomizuka, Kazuma 742 

Tomoshige, Ryuji 216 

Ton-Nu, Thanh-Thao 48 

Tonon, Giancarlo 706 

Torbett, Bruce E. 973, 1001 

Tordo, Noel 482 

Toriseva, Mervi 1095 

Toro, Ana 339 

Torrero, Marina N. 700, 703 

Torres, Raquel 44, 1117 

Toth, Karoly 1021, 1022, 1023 

Totterman, Thomas H. 1024 

Traas, Anne 240 

Traister, Russell S. 678 

Tran, Diane Thi 601 

Trecki, Jordan 159 

Treiber, Uwe 532 

Treleaven, Chris 628 

Tremblay, Jacques P. 102, 257, 

690, 884, 1079 

Trevisan, Marta 536 

Triche, Timothy J. 70 

Trifari, Sara 330, 345 

Triozzi, Pierre L. 849 

Trippel, Stephen B. 522, 676 

Trivin, Francois 895 

Trobridge, Grant D. 15, 386, 440 

Trono, Didier 895, 896 

Trost, Urska 536 

Trout, M. 245 

Tschan, Mario P. 973, 1001 

Tse, Lai-Yin 713 

Tsuchiya, Hiroyuki 376 

Tsuchiya, Shigeru 344, 345 

Tsuda, Naotake 691 

Tsuruta, Yuko 528, 886, 1018 

Tubb, Julie 824 

Tuch, Bernard E. 899 

Tuchman, Mendel 908 

Tuli, Sonal S. 861 

Tullis, Gregory E. 434, 870 

Tumanova, Irina 561, 575

142 



Turbant, Sabrina 790 

Turgeman, Gadi 1053 

Turkeri, Gulen 724 

Turunen, Anna-Mari 98 

Turunen, Mikko P. 38, 98 

Tuschong, Laura M. 458 

Tvrdik, Wayne 565 

Twisk, Jaap 907 

Uchino, Junji 322, 364 

Uchiyama, Toru 344 

Uckert, Wolfgang 796 

Ueda, Yasuji 355, 833 

Ueno, Hiroe 251 

Ueno, Masaharu 587 

Ueoka, Ryuichi 252 

Ugen, Kenneth E. 693 

Uil, Taco G. 878 

Ujhelly, Olga 1045 

Ulasov, Ilya V. 537 

Umeda, Naoyasu 96 

Uney, James B. 656, 955 

Unwalla, Hoshang J. 998 

Urabe, Masashi 517, 900 

Urban, Nicole 1006 

Urbinati, Fabrizia 819 

Urish, Kenneth L. 920 

Urnov, Fyodor D. 85, 337, 346, 

379, 974 

Ushikoshi, Hiroaki 929 

Vähäkangas, Elisa 97 

Vaags, Andrea K. 1036 

Vacante, D. 950 

Vachani, Anil 1129 

Vahanian, Nicholas 1133 

Vallanti, Giuliana 64 

Vally, Maragatha 1100 

Van Asperen, Peter 485 

van Beusechem, Victor W. 9, 526 

Van Bockstaele, Elizabeth J. 659, 661 

van der Zaal, Bert J. 1073 

van Eert, Simone J. 314 

van Rooijen, Nico 547 

Vandenberghe, Luk H. 503 

Vandenbroucke, Roosmarijn 594, 774 

Vandenburgh, Herman 40 

VandenDriessche, Thierry 40 

VanDyke, Teryy 822 

Vanhook, Miriam K. 359 

Vanier, Marie-T 429 

Vanin, Elio F. 387 

Vankov, Alexander 770 

VanRoey, Melinda 311 

Varathalingam, Anusha 362 

Varella, Rafael B. 970 

Varga, Steven M. 816 

Vargas, Joseph 658 

Vargeese, Chandra 438 

Varghese, Susan 160 

Vassallo, Elizabeth 660 

Vatanen, Tero 38 

Vaughan, Erin E. 767 

Vaysse, Laurence 209 

Veldwijk, Marlon R. 131, 865 

Vellinga, Jort 878 

Vengco, Isabelita 445 

Venneri, Mary Anna 446 

Ventura, Jose Luis 995 

Venturi, Consuelo 435 

Vera, Maria 759 

Veres, Gabor 794 

Vergona, Ron 715 

Verheije, Hélène M. 9 

Verma, Inder M. 56, 57, 428, 1081 

Vetu, Christelle 92 

Veys, Paul A. 31 

Viale, Diego L. 78 

Vidal, Francisco 432 

Vierling, Pierre 591 

Vigant, Frédéric 524 

Vigna, Elisa 373 

Vigouroux, Sthephane 801 

Vile, Richard 267, 271, 449, 704, 

723, 802, 804 

Vincent, Karen A. 628 

Vincent, Loic 147 

Visigalli, Ilaria 972 

Viswanathan, Mohan N. 933 

Vitale, Aniello 669 

Vitolo, Joseph M. 877 

Vlachaki, Maria T. 286, 695 

Vockley, Jerry 921 

Voelkel-Johnson, Christina 324 

Vogel, Jonathan C. 1118 

Vogel, Roland 1059 

Vogler, Carole 237, 424 

Vojtech, Lucia 987 

Volk, Andrea L. 1007 

Vollmer, Regis 1096 

von Kalle, C. 1055 

von Kalle, Christof 6, 33, 394, 1076 

von Laer, Dorothee M. 63, 77, 836, 987 

von Messling, Veronika A. 106, 732, 

803, 843, 844 

von Smitten, Karl 328 

Vongpunsawad, Sompong 720 

Voullaire, Lucille 967 

Voutetakis, Antonis 466, 471, 520 

Vrahas, Mark S. 889, 1120 

Vulin, Adeline 261, 820 

Vyas, Y. 341 

Wada, Akihiko 282 

Wada, Takahito 618 

Waddington, Simon N. 238, 394 

Wade-Martins, Richard 956 

Wadhwa, Meenu 711 

Wadman, Shannon A. 192, 347 

Waerzeggers, Yannic 77 

Wagner, Denisa 335 

Wagner, Ralf 764 

Wahlfors, Jarmo 97, 308, 323, 487, 845 

Wahlfors, Tiina 308 

Wait, Richard B. 754 

Wakimoto, Hiroaki 283 

Waldon, Lanaya 459 

Walker, Henry J. 496 

Walker, Jon 288, 810 

Wally, Verena 1065 

Walter, Claire P. 642 

Walter, Glenn A. 44, 418, 1117 

Wan, Song 620 

Wang, Alfred Y. 915 

Wang, Bin 239, 247, 421 

Wang, Bing 398, 792 

Wang, Cao Yang 166 

Wang, Chi-Hsien 304 

Wang, Chiou-Miin 246 

Wang, Daowen 916 

Wang, G. 8 

Wang, Grace Ying 163 

Wang, Haibin 643 

Wang, Hongjie 348, 880 

Wang, Hongli 605 

Wang, Hongyu 275 

Wang, Hui Q. 264 

Wang, Jeng-Hwan 280, 281 

Wang, Jianghua 466, 471, 672, 1122 

Wang, Jin-hui 731 

Wang, Jun 1113 

Wang, L. 419, 615 

Wang, Li 539, 576 

Wang, Lili 111, 500, 1081 

Wang, Lina 988 

Wang, Linlin 465 

Wang, Lixin 687 

Wang, Minghui 537, 1029



Wang, Peiyin 768, 958, 959, 960 

Wang, Ping 128, 240, 247, 421 

Wang, Qing 571, 925, 928 

Wang, Sam 164 

Wang, Shiyang 147 

Wang, Shu 166 

Wang, Su-Jane 280, 281 

Wang, Wenzheng 160 

Wang, Xiao Tian 680, 684, 689 

Wang, Xiaohong 991 

Wang, Xin 1068 

Wang, Xinzhong 887 

Wang, Xuli 779 

Wang, Yaming 1126 

Wang, Yi-gang 722, 731 

Wang, Yoahe 394 

Wang, Yun 133 

Wang, Zejing 791 

Wang, Zheng 639 

Wang, Zhong 398, 916 

Wang, Zhonglin 42 

Wang, Zhongyan 667 

Wangensteen, Kirk J. 1068 

Ward, Peter 20 

Warfe, Lyndon 1072 

Warrington, Kenneth H. 400 

Washizawa, Kentaro 451, 841 

Wasserfall, Clive 914 

Watanabe, Atsushi 618, 968 

Watanabe, Yoshiteru 527 

Watchko, Jon 1125 

Watkins, Jeff 132 

Watkins, Linda R. 644, 645 

Watson, Gordon 232 

Watt, Suzanne M. 238 

Waud, William R. 453 

Webb, Donna J. 649 

Wechuck, James 757, 760 

Wechuck, Jim 156 

Weel, Vincent 1112 

Wegman, Troy R. 496 

Wei, Dongguang 666 

Wei, Du 344 

Wei, Jun-Ping 407 

Wei, Lisa L. 875 

Wei, Wen-Chi 280, 281 

Wei, Zhanyong 135 

Weigek-Kelley, Kirsten A. 11 

Weiner, David B. 575, 693 

Weiss, Daniel J. 357 

Weissig, Volkmar 1105 

Weissleder, Ralph 283 

Weitzman, Matthew D. 371 

Wellman, Jennifer A. 130, 850 

Wen, Jih-Chun 280 

Wen, Shu Fen 749 

Wendell, Steven K. 156 

Wenger, David 972 

Wenk, Corinne 332 

Wenz, Frederik 131, 326, 865 

Wenzel, Andreas 667 

Werner, Petra 606 

Wesley, Bob 1041 

West, Jennifer L. 681, 781, 892 

Westover, Jeffrey 693, 699 

Wetzler, Lee M. 406 

Whalen, Robert 984 

Wheeler, Christopher J. 698 

Wheeler, Matthew 179 

Wheeler, Thurman M. 265 

White, April F. 507, 849 

White, Julie 575 

White, Kathy 959 

White, Stephen J. 534 

Whiting-Theobald, Narda 234 

Whitley, Chester B. 244, 1098 

Wickham, Thomas J. 975, 1013, 1088 

Wickremasinghe, R. G. 292 

Wiehle, Sandra 780 

Wielgosz, Matthew M. 831 

Wiik, A. 924 

Wilber, Andy 193, 338, 908, 1068 

Wilcox, David A. 340 

Wild, Jens 764 

Wilderman, Michael J. 1129 

Wildhaber, Barbara 895 

Wilfond, Benjamin S. 1078 

Wilkes, Fraser J. 481, 599 

Wilkinson, Miles 272 

Will, Elke 829 

Willer, Mette 306 

Williams, Briana J. 316 

Williams, Darryl N. 918, 919 

Williams, David A. 6, 829 

Williams, James A. 180 

Williams, Tiffany L. 321 

Wilson, A. 950 

Wilson, Alistair 947 

Wilson, Annette S. 586 

Wilson, Brian C. 75 

Wilson, C. 950 

Wilson, David C. 682 

Wilson, James M. 111, 112, 139, 141, 

146, 404, 406, 500, 

503, 506, 508, 660, 

812, 847, 853, 872, 

1081, 1089, 1092 

Wilson, Steven 158 

Winkler, Christian 941 

Wirth, Thomas 165, 758 

Wirthlin, Louisa 460, 1031 

Wisse, Eddie 548 

Wissler, Manuela 33 

Wivel, Nelson 506 

Wohlford-Lenane, Christine L. 816 

Wold, William S. M. 1021, 1022, 1023 

Wolf, Christian 495 

Wolf, Hans 764 

Wolf, Jayanthi J. 893 

Wolfe, Darren 156, 641, 646, 

671, 757 

Wolfe, Darren P. 640, 642, 760 

Wolfe, John H. 129, 157, 510, 856 

Wolfer, David P. 939 

Wolfrum, Nina 7 

Wolfsberg, Tyra 333 

Won, Young Hyuk 827 

Wong, Christie 19 

Wong, K. K. 19, 402 

Wong, Liang-Fong 648 

Wonganan, Piyanuch 142, 414 

Woo, Savio L. C. 1097 

Wood, Brent 465 

Wood, Kathryn 238 

Woodcock, Denise 628 

Woodle, Martin C. 318 

Woolf, Clifford J. 643 

Woraratanadharm, Jan 1058 

Worden, Andrew 477 

Worgall, Stefan 969, 975, 1088 

Work, Lorraine M. 851 

Working, Peter 538, 545 

Worsham, D. Nicole 6 

Wouters, Brad G. 329 

Wright, Fraser 132 

Wu, Di 112, 1089 

Wu, Hongju 299, 328, 448, 1026 

Wu, Jerry 442, 832 

Wu, Jian 99, 596 

Wu, Jiangxue 589 

Wu, Jianqing 11 

Wu, Lily 1008, 1051 

Wu, Qi 140, 991, 1090 

Wu, Wenman 605

144 



Wu, Xiao Bing 127 

Wu, Xiaobing 108, 621, 637, 935 

Wu, Xiaolin 516 

Wu, Zhijian 12 

Wuertzer, Charles A. 966 

Würdinger, Tom 9 

Xenariou, Stefania 356 

Xia, Danny 43, 379 

Xia, Xueqing 272 

Xiao, Shuifang 663 

Xiao, Weidong 123, 352, 505, 605 

Xiao, Wei-Hong 96 

Xiao, Xiao 254, 304, 398, 

678, 751, 792, 916, 

1086, 1125 

Xie, Jiansong 211, 1108 

Xie, Jin 937 

Xie, Jing 505, 605 

Xie, Litao 601 

Xie, Xiaoming 82 

Xin, Hong 69 

Xinghua, Wang 705 

Xinyuan, Liu 705 

Xiong, Anming 554 

Xiong, Weidong 143, 269, 273, 

430, 946 

Xu, Benling 589 

Xu, Fang 1094 

Xu, Hui 405, 441, 557, 

600, 821, 915 

Xu, Jenny 893 

Xu, Jianfeng 859 

Xu, Kai 539 

Xu, Li 984 

Xu, Lihong 272 

Xu, Lingfei 32, 239, 247, 602 

Xu, Ruian 123, 305, 352, 605, 713 

Xu, Xin 140, 1090 

Xu, Yi 825 

Xue, Gang 589 

Xue, Shao-An 796 

Xue, Shoa-An 171 

Yakubov, Leonid A. 740 

Yam, Priscilla 442, 832 

Yamada, Masanori 949 

Yamaguchi, Teruhide 525, 888 

Yamaguchi, Tomoyuki 838 

Yamamoto, Keiji 616, 938 

Yamamoto, Masato 314, 679, 1020 

Yamamoto, Naoki 99, 489 

Yamamoto, Seiji 216, 452, 1020 

Yamamura, Kenichi 255 

Yamanaka, Ryuya 270 

Yamaoka, Tetsuji 210 

Yamashita, Misa 226 

Yan, Hua 489, 621 

Yan, Ziying 498, 499, 521 

Yanay, Ofer 459 

Yáñez-Muñoz, Rafael J. 391 

Yang, Chen 718 

Yang, Guang 275, 695 

Yang, Jun 433, 482, 855, 1050 

Yang, Linda 716 

Yang, Michael H. 503 

Yang, Ning-Sun 280, 281, 978 

Yang, PingAr 140, 991, 1090 

Yang, Qing 823 

Yang, Shuting 755 

Yang, Wendy 427 

Yang, Xiaoming 200 

Yang, Yanping 293 

Yang, Yu 108 

Yang, ZhenFan 712 

Yankelson, Lior 931 

Yant, Stephen R. 22, 191, 573, 

600, 821, 1046, 1101 

Yates, Jonathan W. 944 

Ybarra, Cathy 1075 

Ye, Jianqin 926 

Ye, P. 341 

Ye, Peiqing 611, 613 

Ye, Tao 444 

Yee, Jiing-Kuan 440, 442, 832 

Yee, Michael S. 933 

Yeghiazarians, Yerem 933 

Yeh, Yi-Chun 978 

Yellen, Benjamin B. 918, 919 

Yen, Yi-Fu 280 

Yeng, Looi Chung 344 

Yi, So-Yong 717 

Yigit, Gunnur 894 

Yilmaz, Cigdem E. 274 

Yim, Anthony P. C. 620 

Yin, HaiFang 788 

Yin, Jing 325 

Yin, Xiaoqing 42 

Ying, Baoling 1021, 1022, 1023 

Ying, Chuyan 694 

Yip, Ping K. 648 

Ylä-Herttuala, Seppo 38, 98, 165, 

758, 1070, 1073 

Yockman, James W. 207, 585, 218 

Yoder, Mervin C. 374, 401 

Yokoo, Tomoko 472 

Yokosawa, Hideyoshi 68 

Yokoyama, Takashi 597 

Yonemitsu, Yoshikazu 833 

Yoneyama, Ryuichi 48, 49 

Yong, Liu 128 

Yoo, Ji-young 1019 

Yoon, A-Rum 1019 

Yoon, Jin-Han 168 

Yoon, Ji-Won 416 

Yoon, Nam Kyung 104 

Yoshida, Kimiko 708 

Yoshida, Teruhiko 708 

Yoshida, Tetsuya 107 

Yoshikawa, Hideki 685 

Yoshikawa, Hiroyuki 125 

Yoshikawa, Kenichi 221 

Yoshimoto, Takayuki 692 

Yoshimura, Madoka 253 

Yoshio, Naoko 679 

Yoshioka, Akira 597 

Yoshioka, Kowashi 251 

Yoshioka, Toru 616, 938 

Yoshizaki, Mariko 841 

Yotvat, H. 766 

Yotvat, Hagit 735 

You, Hong 128 

You, Ying Jian 357 

Young, James W. 277 

Young, Lauren M. 147 

Young, Sarah P. 1124 

Yu, De-Chao 311, 538, 545, 881 

Yu, Guann-yi 982 

Yu, Hong 1058 

Yu, Jie 155 

Yu, John S. 692, 698 

Yu, Julie H. 488 

Yu, Seung Shin 104, 827 

Yu, XiaoJin 463 

Yu, Ying 442 

Yu, Ze-Yan 485 

Yu, Zhenkun 762 

Yuan, Jianda 277 

Yuan, Jin 470 

Yuan, Xiangpeng 692, 698 

Yuasa, Katsutoshi 253 

Yue, Yongping 515, 688, 793 

Yun, Chae-Ok 541, 551, 702, 1019 

Yun, Sung-Tae 295 

Yurek, David M. 655 

Yvon, Eric 801 

Zabala, Maider 320, 547



Zabner, Joseph 747, 814 

Zacchigna, Serena 114, 121, 126, 1087 

Zaia, John A. 8, 442, 832 

Zajac, Allan J. 140, 991, 1090 

Zak, Roman 521 

Zakay-Rones, Zichria 806 

Zala, Diana 953 

Zamir, Gideon 744 

Zanetti, Maurizio 179 

Zanjani, Esmail D. 750 

Zaratiegui, Mikel 759 

Zeira, Evelyne 735, 744, 766 

Zeller, W. Jens 131, 326, 865, 1034 

Zeltner, Nadja 20 

Zemgulis, Vitas 630 

Zeng, Guisheng 1068 

Zenri-Aihara, Katsuko 242 

Zentella-D, Alejandro 995 

Zentilin, Lorena 17, 114, 126, 1087 

Zepeda, Monica L. 749 

Zern, Mark A. 99 

Zhang, Baigen 230, 562, 635, 639 

Zhang, Fan 147 

Zhang, Fang 31 

Zhang, Guisheng 595 

Zhang, H. Steven 43, 646 

Zhang, Haiping 364 

Zhang, Hao 230, 635 

Zhang, Jian 398 

Zhang, Jianchao 986 

Zhang, JianXin 377 

Zhang, Jin-he 722 

Zhang, Jiwei 562, 635, 639 

Zhang, Jun 296 

Zhang, Lan 562, 798 

Zhang, Lei 43, 337, 379, 1072 

Zhang, Liang 116, 399 

Zhang, Liang N. 521 

Zhang, Lidong 539 

Zhang, Maolin 988 

Zhang, Ming 361 

Zhang, Peng 621 

Zhang, Shangming 2 

Zhang, Tingguo 761 

Zhang, W. 341 

Zhang, Wei Wei 1128 

Zhang, Weitan 877 

Zhang, Wenge 984 

Zhang, Xiao-Bing 456 

Zhang, Xichen 1069 

Zhang, Yan-hong 722, 731 

Zhang, YanPing 1009 

Zhang, YuanYuan 860, 862 

Zhang, Yuhua 893 

Zhang, Zhe 406 

Zhang, Zi-lai 722 

Zhao, Ai Zhi 127 

Zhao, Li-li 722, 731 

Zhao, Qi 110 

Zhao, Weihong 11 

Zhao, Yuan 105 

Zhenfu, Cui 705 

Zheng, Changyu 877 

Zheng, Dexian 305 

Zheng, Maolin 485 

Zheng, Ming 287 

Zheng, Mingquan 977 

Zheng, Mingzhong 809 

Zheng, Yi 982 

Zhi, Yan 112, 141, 406, 1089 

Zhong, Li 11, 16, 401 

Zhong, Xiaohong 43, 646 

Zhou, Chun 943 

Zhou, Jiemin 571 

Zhou, Lijun 647 

Zhou, Liqiao 398, 792, 916 

Zhou, Qin 318 

Zhou, Shang-Zhen 130, 849, 850 

Zhou, Xiaohuai 16 

Zhou, Xiaoyang 404 

Zhou, Yuanyue 1072 

Zhou, Zhaoxiong 635 

Zhu, Julie X. 286, 695 

Zhu, Q. 341 

Zhu, Tong 398, 792, 916, 1125 

Zhu, Zeng B. 537 

Ziady, Assem G. 1104 

Ziegler, R. J. 245, 249 

Ziesling, Rivka 233 

Zilberman, Y. 766 

Zilberman, Yoram 184, 1053 

Zimmer, Catherine 1078 

Zinn, Kurt R. 448 

Zinnen, Shawn 438 

Zirger, Jeffrey M. 143, 430, 745, 946 

Ziske, Carsten 284 

Zolotukhin, Irene 514, 909, 911 

Zolotukhin, Sergei 400, 418 

Zompetta, Claudia 447, 727 

Zou, Gang-Ming 374 

Zou, Jianxiang 1114 

Zou, Shaomin 579, 584 

Zou, Wei-guo 731 

Zsebo, Krisztina 41 

Zu, Lingyun 621, 637 

Zugates, Gregory T. 786 

Zumbuehl, Andreas 777 

Zumstein, Louis A. 1021, 1022, 1023 

Zurakowski, David 522, 676 

Zurn, Anne D. 953 

Zwick, Michael 984 

Zwierzina, Heinz 1070

146 


EXHIBIT HALL FLOORPLAN 

AISLE 200 

AISLE 300 

AISLE 400 

AISLE 500 

AISLE 600


EXHIBITOR INFORMATION 

GENERAL INFORMATION 

The Exhibit Hall is located in Exhibit Halls 1 & 2 of the 

America’s Center. Please allow adequate time in your 

daily schedule to visit the exhibits. The companies and 

organizations exhibiting at the ASGT Annual Meeting 

will provide you with the latest information on the 

products and services directly related to your professional 

needs. 



(Welcome Reception begins at 4:00 pm) 





EXHIBITOR REGISTRATION DESK 

The Exhibitor Registration Desk is located in the 

Washington Lobby of the America’s Center. 

EXHIBITOR REGISTRATION DESK 

HOURS 

Wednesday, June 1 Noon – 6:00 pm 




Thank You 

Exhibitors! 

EXHIBITOR BADGE POLICY 

Admission to the Exhibit Hall will be by name badge 

only. Security guards will monitor exhibit hall entrances 

for proper I.D. Exhibit booth personnel must show an 

official ASGT exhibitor name badge in order to gain 

access to the exhibit hall during installation, show, or 

dismantlement hours. Independent contractor personnel, 

hired by an exhibitor to install and dismantle their display, 

do not need to show exhibitor badges, but should possess 

identification that they are employed by the independent 

contractor. If independent contractors do not possess 

proper identification, they must obtain a name badge at 

the on-site exhibitor registration area. 

ENDORSEMENT DISCLAIMER 

Products and services displayed in the Exhibit Hall or 

advertised in this program occur by contractual business 

arrangements between the ASGT and participating 

companies and organizations. These arrangements do not 

constitute or imply an endorsement by the ASGT of 

these products and services.

148 


EXHIBITOR DESCRIPTIONS 

Aldevron, LLC 

3233 15th Street South 

Fargo, ND 58104 

Phone: 701-297-9256 

Fax: 701-280-1642 

E-Mail: vchambers@aldevron.com 

www.aldevron.com 

Booth Number: 405 

Aldevron continues to set new plasmid manufacturing 

standards. We offer research, pre clinical and GMP levels 

of production services. Our Genetic Immunization and 

Antibody (GIA) production technology can be used to 

rapidly screen new vaccine candidates and/or to generate 

antibodies to any gene-specific antigen. Through our 

partnership with GENOVAC we are also able to provide 

researchers a source for custom monoclonal antibody 

production. Visit our website for a catalog of services and 

products. 

AllCells, LLC 

2001 Dwight Way, Suite 1360 

Berkeley, CA 94704 

Phone: 510-548-8908 

Fax: 510-548-8327 

E-Mail: jtong@allcells.com 

www.allcells.com 


AllCells, LLC sells cells for your research needs in vitro: 

human hematopoeitic stem/progenitor cells, immunological 

cells, endothelial cells, hepatocytes, astrocytes, and 

RNA/cDNA purified from these cells. 

Alzet Osmotic Pumps®, Durect Corporation 

10240 Bubb Rd. 

Cupertino, CA 95014 

Phone: 800-692-2990 

FAX: 408-865-1406 

E-Mail: alzet@durect.com 

www.alzet.com 


Implantable Infusion Pumps 

ALZET ® Osmotic Pumps deliver solutions continuously 

for 1 - 28 days, in animals as small as mice, without 

external connections. These implantable infusion pumps 

have been used in nearly 8,000 studies for delivery of 

agents such as gene vectors, oligonucleotides, cytokines, 

growth factors and more. 

amaxa Inc. 

205 Perry Parkway, Suite 7 

Gaithersburg, MD 20877 

Phone: 240-632-9110 

Fax: 240-632-9112 

E-Mail: info@amaxa.com 

www.amaxa.com 


Established worldwide in leading labs amaxa’s 

Nucleofector technology provides a highly efficient and 

reproducible non-viral transfection method for cell lines 

and primary cells. Using a unique combination of 

electrical parameters and cell type-specific solutions 

DNA and siRNA can be delivered into cells with top 

efficiencies. 

American Society of Gene Therapy 

555 East Wells Street, Suite 1100 

Milwaukee, WI 53202 

Phone: 414-278-1341 

Fax: 414-276-3349 

E-Mail: info@asgt.org 

www.asgt.org 

Booth: 514-516 

Stop by the ASGT membership booth to pick-up 

meeting related items and membership applications. Find 

out how to easily access the Members’ Only Section of 

the website, including the ASGT on-line membership 

directory. You can also purchase an attractive ASGT shirt! 

Arkios BioDevelopment International 

2700 International Parkway, Suite 300 

Virginia Beach, VA 23452 

Phone: 757-631-2114 

Fax: 757-631-2115 

E-Mail: info@arkios.com 

www.arkios.com 

Booth: 410 

Arkios is dedicated within this field of innovation and 

prepared to provide service to strengthen the development 

path for emerging technologies. We are currently 

managing the first Gene Therapy trial for Parkinson’s 

Disease and have done programs and research in Cystic 

Fibrosis as well as anti-aging. Our services include 

regulatory planning, IND preparation, clinical trial 

project management, investigator selection, site monitoring, 

medical writing, data management and biostatistics.



Avanti Polar Lipids, Inc. 

700 Industrial Park Drive 

Alabaster, AL 35007 

Phone: 205-663-2494 

Fax: 205-663-0756 

E-Mail: trevor@avantilipids.com 

www.avantilipids.com 


Avanti Polar Lipids, with an unparalleled reputation for 

purity, is clearly established as the world leader in lipid 

production. Avanti is well known to the pharmaceutical 

industry with a cGMP production facility that is regulated 

by the FDA. Recent developments include Glycosylated 

and Phosphorylated Sphingosines and Ceramides 

including fluorescent derivatives. 

Baltimore Area Convention & Visitors Association 

Convention Services Department 

100 Light Street, 12 th Floor 

Baltimore, MD 21202 

Phone: 410-659-7300 

Fax: 410-727-2308 

www.baltimore.org 


Mission: The Baltimore Area Convention and Visitors 

Association (BACVA), a membership-based organization, 

is the official destination development and marketing 

leader for Greater Baltimore, responsible for generating 

incremental economic benefits to the community 

through the attraction of convention and leisure visits. 

BioProcess International 

One Research Drive, Suite 400A 

Westborough, MA 01581 

Phone: 508-616-5550 

Fax: 508-616-0476 

E-Mail: bcaine@bioprocessintl.com 

www.bioprocessintl.com 


BioProcess International provides the biotechnology 

industry with monthly, peer-reviewed information 

necessary to successfully drive products through the 

biopharmaceutical, biovaccine and biodiagnostic development 

and manufacture process. BPI’s circulation consists 

of 30,000 mid-to-upper managers working in all phases 

of the biotherapeutic development and manufacturing 

process in the United States, Europe and Canada. 

BioSpherix, Ltd. 

6678 Route 17 

Redfield, NY 13437 

Phone: 315-387-3414 

Fax: 315-387-3415 

E-Mail: ryerden@biospherix.com 

www.biospherix.com 


Advanced new GMP-like incubation concept for conventional 

bag, plate, flask, multi-well plate, and petri dish 

cultures. Flexible system integrates multiple independent 

high performance O2/CO2 incubators that open into 

sterile gloved clean hood with identical O2/CO2 atmospheres 

to provide uninterrupted conditions during 

handling. GMP cell culture facility in a self-contained, 

bench-top unit. 

CellGenix, US Operations 

602 Hillside Avenue 

Antioch, IL 60002 

Phone: 847-395-7277 

Fax: 847-395-0808 

E-Mail: cellgenixusa@aol.com 

www.cellgenix.com 


CellGenix manufactures various high quality cGMP 

produced cytokines and cell culture medium for ex-vivo 

dendritic, stem, and T cell expansion and maturation 

protocols. cGMP contract services are also available. 

Corning Incorporated 

45 Nagog Park 

Acton, MA 01720 

Phone: 978-635-2200 

Fax: 978-635-2476 

E-Mail: clswebmail@corning.com 

www.corning.com/lifesciences 

Booth Number: 206-208 

Corning Incorporated, a diversified technology company, 

manufactures high quality, high performance research 

products for life sciences. Corning will exhibit reliable 

and fully-tested cell culture products for scale up. 

Included will be CellSTACKCulture Chambers and E- 

CubeCulture System. Key products from first culture 

to high throughput screening will also be on display.

150 



DNAVEC Corporation 

Toranomon 40MT Bldg. 9F 

5-13-1 Toranomon, Minato-ku, Tokyo 105-0001 

JAPAN 

Phone: +81-3-5403-7061 

Fax: +81-3-5403-7062 

E-Mail: info@dnavec-corp.com 

www.dnavec.co.jp 


DNAVEC has its own proprietary technology, Sendai 

Virus Vector (SeV). SeV is a novel “cytoplasmic RNA 

vector”, which is genetoxicity free and expresses a foreign 

gene quite efficiently. SeV has a wide application of gene 

therapy and gene vaccine mainly at cardiovascular disease 

and oncology. 

Elsevier 

525 B Street, Suite 1900 

San Diego, CA 92101 

Phone: 619-231-6616 

Fax: 619-699-6320 

www.elsevier.com 


Come to the Elsevier booth to browse our books and 

journals. Become an active member and receive a 50% 

discount on one of our selected books. 

GE Healthcare 

(formally known as Amersham Biosciences Corp) 

800 Centennial Ave. 

P.O. Box 1327 

Piscataway, NJ 08855-1327 

Phone: 800-526-3593 or 732-457-8000 

Fax: 778-295-8102 

E-Mail: cs-us@ge.com 

www.gehealthcare.com 


GE Healthcare provides transformational medical 

technologies that are shaping a new age of patient care. 

GE Healthcare’s expertise in medical imaging and 

information technologies, medical diagnostics, patient 

monitoring systems, disease research, drug discovery and 

biopharmaceuticals is dedicated to detecting disease 

earlier and tailoring treatment for individual patients. 

GENEART North America 

28 Kirk Bradden Road East 

M8Y 2E6 Etobicoke, Ontario 

CANADA 

Phone: 252-975-3417 

Fax: 252-975-1366 

E-Mail: patricia.nick@geneart.com 

www.genart.com 


Based on the technological platform gene synthesis, 

GENEART offers integrated system solutions for 

pharmaceutical and biotech industries for developing 

DNA and protein-based therapeutic agents and vaccines. 

The spectrum of performance ranges from rational gene 

design and synthesis over generation of combinatorial 

gene libraries to the production of plasmid DNA in any 

amount including GMP quality. 

Invitrogen Corporation 

1620 Faraday Avenue 

Carlsbad, CA 92008 

Phone: 760-603-7200 

www.invivogen.com 


Invitrogen provides products and services that support 

academic and government research institutions and 

pharmaceutical and biotech companies worldwide in 

their efforts to improve the human condition. Our life 

science technologies improve and accelerate all areas of 

research, drug discovery, and commercial bioproduction. 

InvivoGen 

3950 Sorrento Valley Boulevard, Suite A 


Phone: 858-457-5873 

Fax: 858-457-5843 

E-Mail: info@invivogen.com 

www.invivogen.com 


InvivoGen provides the research community with highquality 

products designed to accelerate discoveries in 

gene therapy research. InvivoGen has become a leader in 

RNAi research with our psiRNA family of silencing 

vectors. We also offer a large choice of therapeutic genes 

cloned into various mammalian expression vectors.



Kemp Biotechnologies, Inc. 

7307 Governors Way 

Frederick, MD 21704 

Phone: 301-620-7100 

Fax: 301-620-0162 

E-Mail: b.miller@kempbiotech.com 

www.kempbiotech.com 


Kemp Biotechnologies, Inc. will present a proprietary 

nontoxic transfection reagent and a proprietary transfection 

process, which enables AAV viral vector production 

via cotransfection of suspension 293 cells. This service is 

a scalable, cost-effective alternative to the time-consuming 

process of transfecting large quantities of plates of 

adherent 293 cell cultures to generate AAV vector stocks. 

Lark Technologies 

9441 W. Sam Houston Parkway South, Suite 103 

Houston, TX 77099 

Phone: 713-779-3663 

Fax: 713-779-1661 

E-Mail: sales@lark.com 

www.lark.com 


Lark, A Genaissance Company is a leading contract 

research organization providing research grade and 

regulatory submission services including DNA sequencing, 

quantitative PCR analysis, and genetic stability 

testing. All of Lark’s regulatory submission services 

comply with Good Laboratory Practices as specified by 

the FDA. 

Mary Ann Liebert, Inc. 

2 Madison Ave. 

Larchmont, NY 10538 

Phone: 914-834-3100 

Fax: 914-834-3771 

E-Mail: info@liebertpub.com 

www.liebertpub.com 


Mary Ann Liebert, Inc. Publishers invites you to come by 

our booth #112, and take a complementary copy of 

Human Gene Therapy, Genetic Testing, Genetic Engineering 

News and other related publications. 

MDS Pharma Services 

2350 Cohen Street 

Saint-Laurent (Montreal) 

Quebec, H4R 2N6 

Canada 

Phone: 514-333-0033 

Fax: 514-333-8861 

E-Mail: info@mdsps.com 

www.mdsps.com 


MDS Pharma Services’ biopharmaceuticals group 

executes rapid and rigorous GLP/cGMP safety and 

analytical testing. Our state-of-the-art facility in Bothell, 

Washington, features restricted-access laboratories and 

FDA and ICH compliance. Suites consist of BSL-2/BSL- 

3 virology labs, cell culture and cell-banking (cGMP) 

facilities, protein chemistry and PCR rooms. For more 

information, visit www.mdsps.com. 

Mirus Bio Corporation 

505 S. Rosa Road, Suite 104 

Madison, WI 53705 

Phone: 608-441-2824 

Fax: 608-441-2849 

E-Mail: Claire.ruzicka@mirusbio.org 

www.mirusbio.com 


Mirus Bio provides state-of-the-art nucleic acid delivery 

and labeling reagents for in vitro and in vivo research. 

TransIT â reagents achieve high efficiency/low toxicity 

transfections in a variety of cell lines and applications 

including siRNA delivery. Label IT â non-enzymatic 

nucleic acid labeling kits are optimized for hybridization 

and tracking applications. 

Molecular Medicine Bioservices, Inc. 

6219 el Camino Real 

Carlsbad, CA 92009 

Phone: 760-918-0007 

Fax: 760-918-0076 

E-Mail: dbacker@molecularmed.com 

www.molecularmed.com 


Molecular Medicine BioServices, Inc. provides enabling 

technologies for viral-based therapeutics. Located in 

Carlsbad, CA, the company is a leading contract manufacturing 

and clinical production resource for companies 

engaged in vaccine and gene therapy development. 

Molecular Medicine is committed to supporting clients 

from development through production and into clinical 

approval.

152 



National Gene Vector Laboratories 

650 Barnhill Drive, MS 205 

Indianapolis, IN 46202-5120 

Phone: 317-274-0448 

Fax: 317-274-4518 

E-Mail: kwicker@iupui.edu 

www.ngvl.org 


The NGVL consist of three vector production facilities 

located at Indiana University, Baylor College of Medicine, 

and the City of Hope/Beckman Research Institute. 

Two pharmacology/toxicology centers have been established 

at the University of Florida and Southern Research 

Institute. The production sites will generate clinical grade 

adenoviral, adeno-associated virus, DNA plasmid, 

lentivirus, herpes simplex virus and retrovirus vectors for 

Phase I/II gene therapy protocols, while the pharmacology/toxicology 

centers will provide support for investigators 

needing such studies for their clinical protocols. 

Requests for vector production and/or pharmacology/ 

toxicology support will be reviewed by the Scientific 

Review Board and Steering Committee of the NGVL 

with selection based upon scientific merit, feasibility and 

availability of NGVL resources. If the application is 

approved through the Steering Committee, clinical grade 

materials and pharmacology/toxicology studies will be 

provided without charge for human gene therapy trials. 

An application packet can be downloaded from the 

NGVL website at http://www.ngvl.org or obtained 

through the Coordinating Center. 

Nature Publishing Group 

345 Park Avenue 

New York, NY 10010 

Phone: 212-726-9200 

E-Mail: institutions@natureny.com 

www.nature.com 


Nature Publishing Group publishes quality research and 

review material; timely news; and essential career and 

recruitment information in science and clinical medicine. 

NPG combines the excellence of Nature, its associated 

primary research journals, the new Nature Clinical 

Practice review series, Nature Methods plus over 30 

leading international scientific, clinical, and medical 

journals. 

Nature Technology Corporation 

4701 Innovation Drive 

Lincoln, NE 68521 

Phone: 402-472-6530 

Fax: 402-472-6532 

E-Mail: natx@natx.com 

www.natx.com 


Nature Technology Corporation (NTC) is a full-service 

vector development company, specializing in the production 

of plasmid DNA and recombinant proteins for 

research and preclinical use. NTC’s pDNA fermentation 

process currently produces DNA at >1g/L, leading the 

industry in quality and economy: http://www.natx.com. 

Office of Biotechnology Activities, National Institutes 

of Health 

6705 Rockledge Drive, Suite 750 

Bethesda, MD 20892 

Phone: 301-496-9838 

Fax: 301-496-9839 

E-Mail: bm128w@nih.gov 

www4.od.nih.gov/oba 


The NIH Office of Biotechnology Activities (OBA) will 

be exhibiting its initiatives, and staff will be available to 

answer questions about the oversight of recombinant 

DNA research, including human gene transfer research. 

Meeting participants will be given “hands-on” demonstrations 

of OBA’s Genetic Modification Clinical Research 

Information System (GeMCRIS), as well as an 

extensive, Web-based resource on informed consent in 

gene transfer research. 

Olympus America Inc. 

Two Corporate Center Drive 

Melville, NY 11747 

Phone: 631-844-5051 

Fax: 631-844-5112 

E-Mail: Catherine.coll@olympus.com 

www.olympusamerica.com 


Olympus will feature the IX81 inverted motorized 

research microscope along with our innovative spinning 

disk confocal technology. When combined with our latest 

digital camera and SlideBook imaging software, Olympus 

can help you customize a complete system suited for your 

application needs and budget.



Open Biosystems 

6705 Odyssey Dr. 

Huntsville, AL 35806 

Phone: 888-412-2225 

Fax: 256-704-4849 

E-Mail: info@openbiosystems.com 

www.openbiosystems.com 


Open Biosystems is dedicated to distributing high-quality 

tools to unlock the functions of human genes and their 

relationships to disease development. We offer products 

for genomic and proteomic research from shRNA 

libraries and cDNA clones to custom peptide and 

antibody production services. 

Pall Life Sciences 

2200 Northern Blvd. 

East Hills, NY 11548 

Phone: 516-484-3600 

Fax: 516-484-3649 

E-Mail: cdellicicchi@pall.com 

www.pall.com 


Pall offers process filtration, separation and purification 

development, pilot and manufacturing equipment and 

validation services together with pre-inspection reviews, 

troubleshooting consultancy, training, contamination 

analysis and other technical assistance. We offer protein 

expression monitoring and multi-step chromatography 

purification development, target and impurity tracking 

and host cell protein assay development, purification 

optimization and custom affinity sorbents. 

PolyPlus - Transfection 

Bioparc BP 90018 

Illkirch 67401 

FRANCE 

Phone: 33-03-90-40-61-85 

Fax: 33-03-90-40-61-81 

E-Mail: pbelguise@polyplus-transfection.com 

www.polyplus-transfection.com 


Polyplus-transfection is a French Life Science company 

dedicated to the development and marketing of transfection 

reagents. Polyplus key transfection reagents are 

jetPEI for DNA and oligonucleotides in vitro and in vivo 

delivery (cGMP available), jetSI-ENDO for siRNA 

delivery and Fecturin for protein production in synthetic 

media. 

Puresyn, Inc. 

87 Great Valley Parkway 

Malvern, PA 19355 

Phone: 610-640-0800 

Fax: 610-640-0808 

E-Mail: leslie@puresyn.com 

www.puresyn.com 


Puresyn, Inc. is a Separations and Purification Technology 

Company that develops, 

manufactures and markets state-of-the-art products and 

services for the separation and purification of nucleic 

acids and other biological molecules utilizing its proprietary 

polymer technology, PolyFlo ® , an inert, irregularly 

shaped, non-porous polymer resin. The Company is a 

leading supplier of contract manufacturing services for 

production and purification of plasmid and viral vectorbased 

gene therapies at scales and specifications suitable 

for research, development and pre-clinical requirements. 

Puresyn has also introduced two (2) new Adenovirus 

purification kits, ADENOPURE Ò and ADENOPURE 

LS ä . They replace time and labor intensive double cesium 

chloride gradient and generate adenovirus preparations 

with high purity and potency in a convenient kit format. 

RheoGene Inc. 

2650 Eisenhower Avenue 

Norristown, PA 19403 

Phone: 610-650-8734 

Fax: 610-650-8755 

www.rheogene.com 


RheoGene is developing its proprietary RheoSwitch® 

Therapeutic System for safe, targeted and regulated cell 

and gene therapies that deliver the right dose- to the right 

place- at the right time- to improve the lives of patients 

with serious medical conditions for which there are no 

effective treatments or cures. 

Sangamo BioSciences, Inc. 

501 Canal Boulevard, Suite A100 

Richmond, CA 94804 

Phone: 510-970-6000 

Fax: 510-236-8951 

E-Mail: erhodes@sangamo.com 

www.sangamo.com 


Sangamo Biosciences, Inc is focused on the research and 

development of zinc finger DNA binding proteins 

(ZFPs) for therapeutic gene regulation and repair. The 

company’s most advanced clinical program is for treatment 

of diabetic neuropathy. Sangamo has additional 

programs in cardiovascular disease, neuropathic pain, 

AMD, and HIV.

154 



SeqWright, Inc. 

2575 W. BellFort, S-2001 

Houston, TX 77054 

Phone: 713-528-4363 

Fax: 713-528-6232 

E-Mail: erhodes@sangamo.com 

www.seqwright.com 


SeqWright is a molecular biology research support 

company specializing in DNA discovery products and 

customized services designed for scientific researchers 

from the pharmaceutical, agricultural, biotechnology, 

government, and academic communities. We offer 

outsourcing options including de novo DNA sequencing 

and re-sequencing, DNA library construction, 

genotyping, quantitative PCR, and DNA synthesis. 

Sigma-Aldrich 

3050 Spruce Street 

St. Louis, MO 63103 

Phone: 314-286-8367 

Fax: 314-286-7817 

E-Mail: Kraymo@sial.com 

www.sial.com 


Sigma-Aldrich provides a broad range of products for use 

in the field of regenerative medicine and gene therapy. 

These products are suitable for research in genomics, 

proteomics, cell signaling and cell culture. Featured 

products include several media for cell culture as well as a 

full range of comprehensive products offerings critical to 

success in this area. Information on all of these products 

can be found by visiting our web site at www.sigmaaldrich.com 

or in our full range of applications-based 

catalogs. 

Southern Research Institute 

2000 9th Avenue South 

Birmingham, AL 35205 

Phone: 205-581-2688 

Fax: 205-322-7473 

E-Mail: Jefferson@SRI.org 

www.southernresearch.org 


Southern Research, based in Birmingham, Alabama, is an 

independent center for scientific research and development 

providing contract research services in preclinical 

drug discovery and development, while focusing on 

cancer, infectious diseases, emerging pathogens, and 

neurological diseases and disorders. For more 

information, visit www.southernresearch.org. 


332 N. Lauderdale, MS 340 

Memphis, TN 38105 

Phone: 901-495-4007 

Fax: 901-495-5376 

E-Mail: fellowships@stjude.org 

www.stjude.org 


St Jude Children’s Research Hospital is a non-profit 

research institution in Memphis, TN, dedicated to 

finding prevention and cures for catastrophic diseases of 

childhood. PhD, MD, PharmD students are needed 

immediately to fill open Postdoctoral fellowships. Bring 

along your CV or ask for our current positions available 

list. 

Virapur 

10461 Roselle Street 


Phone: 858-824-9000 

Fax: 858-824-9408 

E-Mail: virapur@sbcglobal.net 

www.virapur.com 


Virapur is a leader in developing virus purification kits 

and providing services to research and pharmaceutical 

communities. We directly market our Adenovirus Mini- 

Purification kits and the first ever AAV Purification kits 

for rapid virus purification without ultracentrifugation, 

while BD Clontech distributes our larger adenovirus 

purification kits. We also specialize in virus production, 

assays, process development, and consulting services. 

VisualSonics 

3080 Yonge Street, Suite 6100, Box 66 

Toronto, Canada M4N 3N1 

Phone: 416-484-5000 

Fax: 416-484-5001 

E-Mail: info@visualsonics.com 

www.visualsonics.com 


VisualSonics’ Vevo 770imaging system enables realtime 

in vivo visualization and measurement of anatomical 

structures and hemodynamic functional in longitudinal 

studies of mice, rats, chick embryos and other small 

animal models with resolution down to 30 microns. The 

system also enables real-time guidance of micro-injection 

and extraction procedures including targeted injections of 

stem cells, DNA, and other materials in vivo into mouse 

and mice embryos.



Vivascience, Inc. 

131 Heartland Blvd. 

Edgewood, NY 11717 

Phone: 877-452-0345 

Fax: 631-253-5445 

www.vivascience.com 


Vivascience, a member of the Sartorius Group, provides 

membrane-based devices for virus purification and 

concentration that save time and simplify laboratory 

procedures. We also will be displaying a range of syringe 

and capsule membranes for cell culture media sterilization 

or supernatant clarification. 

Waisman Clinical Biomanufacturing Facility 

1500 Highland Ave. 

Madison, WI 53705 

Phone: 608-263-8940 

Fax: 608-263-5725 

www.gmpbiomanufacturing.org 


The Waisman Clinical Biomanufacturing Facility at the 

University of Wisconsin-Madison is a state-of-the-art 

cleanroom facility for biologics and gene therapeutics 

production. The WCBF is comprised of segregated 

production suites for viral, non-viral, and cell-based 

biologics. Supporting features include a full quality 

system and industry experienced staff. Visit us at 

www.GMPbiomanufacturing.org 

The Williamsburg BioProcessing Foundation 

1206 Laskin Road, Suite 201 

Virginia Beach, VA 23451 

Phone: 757-423-8823 

Fax: 757-423-2065 

E-Mail: cburba@wilbio.com 

www.wilbio.com 


The Williamsburg BioProcessing Foundation (WilBio), 

The Most Trusted Source of BioProcess Technology TM, 

was established in 1994 to advance the development of 

safe and effective biological products by providing venues 

for the exchange of bioprocess technology. Now in our 

11th year, we have grown from six to 13 conferences and 

have expanded into Europe and Asia. The BioProcessing 

Journal TM, now in its fourth year, complements our 

conferences by providing a medium through which 

selected presentations are covered in more detail. 

Wiley 

111 River Street 

Hoboken, NJ 07030 

Phone: 201-748-6000 

Fax: 201-748-6088 

E-Mail: rbenner@wiley.com 

www.wiley.com 


Wiley is an independent, global publisher of print and 

electronic products. Visit the Wiley booth to learn more 

about developments to The Journal of Gene Medicine. Our 

latest book titles are available to browse, and purchase at a 

special convention discount of 20%. For details of Wiley 

publications, visit: www.wiley.com.

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Looking forward to Baltimore in 2006! 

Baltimore’s strategic location on the Eastern Seaboard 

places it within easy driving distance of Washington DC, 

Philadelphia and New York. Baltimore/Washington 

Airport (BWI) is the second fastest growing airport in the 

country. BWI is a short ride from downtown and is served 

by over a dozen major airlines. Baltimore is also served by 

Amtrak, making it extremely accessible from anywhere. 

Inner Harbor is the place to see in Baltimore! The world 

renowned National Aquarium, Maryland Science Center 

(with IMAX Theatre), the U.S.S. Constellation, the Power 

Plant, (featuring Hard Rock Café and ESPN Zone), and 

Port Discovery Children’s Museum are just a few of the 

incredible places to visit. 

Baltimore is famous for its succulent crabs and seafood. 

Baltimore offers every kind of ethnic fare imaginable, but 

no visit is complete without sampling the world-famous 

Chesapeake Bay cuisine 

In 1873, merchant Johns Hopkins bequeathed funds to start 

a hospital and university in his name. Today, these 

interconnected institutions are among the greatest for 

higher education and patient care. 

May 31 - June 4, 2006

FINAL PROGR AM - American Society of Gene & Cell Therapy

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