Final Program - American Society of Gene & Cell Therapy
Final Program - American Society of Gene & Cell Therapy
Final Program - American Society of Gene & Cell Therapy
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Abstract Directory, Friday, May 17, 2013<br />
Oral Abstract Session 325<br />
<strong>Gene</strong> <strong>Therapy</strong> for Hematologic Diseases: From<br />
Models to Clinical Results<br />
1:00 PM - 3:00 PM<br />
Room: 151 ABCG<br />
306 1:00 PM<br />
Lentiviral Vector-Based <strong>Gene</strong> <strong>Therapy</strong> for Wiskott-<br />
Aldrich Syndrome: Preliminary Results from the<br />
French Center<br />
Salima Hacein-Bey-Abina, Johanna Blondeau, Laure Caccavelli,<br />
Sabine Charrier, Capucine Picard, Liliane Dal-Cortivo, Guillem<br />
Cross, Stephane Blanche, Frederic Bushman, Nirav Malani, Alain<br />
Fischer, Adrian Thrasher, Anne Galy, Marina Cavazzana-Calvo.<br />
307 1:15 PM<br />
Phase II Clinical <strong>Gene</strong> <strong>Therapy</strong> Trial for Adenosine<br />
Deaminase-Deicient Severe Combined Immune<br />
Deiciency (ADA-SCID)<br />
Kit L. Shaw, Robert Sokolic, Suparna Mishra, Satiro de Oliveira,<br />
Christopher Silvin, Geiger-Schredelseker Sabine, Brown<br />
Berkley, Garabedian Elizabeth, Monika Smogorzewska, Denise<br />
A. Carbonaro, Pei-yu Fu, Alejandra Davila, Jayashree Jagadeesh,<br />
Michael Hershield, Alan Wayne, Neena Kapoor, Theodore Moore,<br />
Fabio Candotti, Donald B. Kohn.<br />
311 2:15 PM<br />
Expansion <strong>of</strong> <strong>Gene</strong>-Modiied T <strong>Cell</strong>s Following<br />
Direct Intravenous Injection <strong>of</strong> Therapeutic Foamy<br />
Virus Vector in a Canine Model <strong>of</strong> Severe Combined<br />
Immunodeiciency<br />
Christopher R. Burtner, Brian C. Beard, Douglas R. Kennedy, Troy<br />
R. Torgerson, Andrew M. Scharenberg, David J. Rawlings, Grant D.<br />
Trobridge, Peter J. Felsburg, Hans-Peter Kiem.<br />
312 2:30 PM<br />
Adeno-Associated Virus-Mediated Expression <strong>of</strong> a<br />
C-Terminal Truncated ADAMTS13 Variant Prevents<br />
Shigatoxin-Induced Thrombotic Thrombocytopenic<br />
Purpura<br />
Sheng-Yu Jin, Juan Xiao, Jialing Bao, Shangzhen Zhou, Fraser J.<br />
Wright, X. Long Zheng.<br />
313 2:45 PM<br />
Self-Inactivating Lentiviral Vectors for Correction <strong>of</strong><br />
Rag1 Severe Combined Immunodeiciency<br />
Karin Pike-Overzet, Anna-Sophia Wiekmeijer, Miranda R. M. Baert,<br />
Chantal Lagresle-Peyrou, Axel Schambach, Fang Zhang, Rob C.<br />
Hoeben, Salima Hacein-Bey-Abina, Arjen C. Lankester, Robbert G.<br />
M. Bredius, Gertjan J. A. Driessen, Adrian J. Thrasher, Christopher<br />
Baum, Marina Cavazzana-Calvo, Jacques J. M. van Dongen, Frank J.<br />
T. Staal.<br />
308 1:30 PM<br />
Lentiviral <strong>Gene</strong> Transfer for Treatment <strong>of</strong> Children<br />
>2 Years Old with X-Linked Severe Combined<br />
Immunodeiciency<br />
Suk See De Ravin, Uimook Choi, Narda Theobald, Janet Lee,<br />
Hongmei Wang, Xiaolin Wu, Geraldine O’Connor, Daniel W.<br />
McVicar, Elizabeth Kang, John T. Gray, Brian P. Sorrentino, Harry L.<br />
Malech.<br />
309 1:45 PM<br />
Integration Site Analysis in a Clinical Trial <strong>of</strong> Lentiviral<br />
Vector Based Hematopoietic Stem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong><br />
for Metachromatic Leukodystrophy. An 18 Months<br />
Follow-Up<br />
Eugenio Montini, Alessandra Bifi, Andrea Calabria, Luca Biasco,<br />
Martina Cesani, Fabrizio Benedicenti, Tiziana Plati, Simone Leo,<br />
Victor Neduva, Gianluigi Zanetti, Alessandro Aiuti, David Dow,<br />
Christ<strong>of</strong> von Kalle, Manfred Schmidt, Maria Sessa, Luigi Naldini.<br />
310 2:00 PM<br />
Comparison <strong>of</strong> Integration Site Proiles between<br />
SCID-X1 <strong>Gene</strong> <strong>Therapy</strong> Trials Using Gammaretroviral<br />
Vectors with Intact or Deleted (SIN) LTRs<br />
Salima Hacein-Bey-Abina, Laure Caccavelli, Nirav Malani, Charles<br />
Berry, Frances Male, Frederic Bushman, Alain Fischer, Marina<br />
Cavazzana-Calvo.<br />
Oral Abstract Session 326<br />
<strong>Gene</strong> Regulation and Animal Models<br />
1:00 PM - 3:00 PM<br />
Room: Ballroom D<br />
314 1:00 PM<br />
Pristimerin Enhances Recombinant Adeno-Associated<br />
Virus Serotype 2 Vector-Mediated Transgene<br />
Expression Both In Vitro and In Vivo<br />
Lina Wang, Yuan Wang, George V. Aslanidi, Arun Srivastava,<br />
Changquan Ling, Chen Ling.<br />
315 1:15 PM<br />
Transcriptional and Post-Transcriptional Regulation To<br />
Overcome Immunological Responses to <strong>Gene</strong> <strong>Therapy</strong><br />
for Hemophilia A<br />
Stefania E. Cannizzo, Simone Merlin, Antonia Follenzi.<br />
316 1:30 PM<br />
Selective Expansion <strong>of</strong> Transduced Hepatocytes: A<br />
Mechanism for Modulating Transgene Levels In Vivo<br />
Sean C. Nygaard, Annelise Haft, Milton Finegold, Markus Grompe.<br />
317 1:45 PM<br />
Multiplex Bi-Functional Short-Hairpin RNA (shRNA)<br />
Targeting Single-Nucleotide KRAS Mutations in<br />
Pancreatic Cancer<br />
Donald D. Rao, Zhaohui Wang, Padmasini Kumar, Neil Senzer, John<br />
Nemnaitis.<br />
Friday, May 17, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
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