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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Thursday, May 16, 2013<br />
Abstract Directory, Thursday, May 16, 2013<br />
Oral Abstract Session 230<br />
Clinical <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> Oral Abstract<br />
Session<br />
2:00 PM - 4:00 PM<br />
Room: Ballroom A<br />
57 2:00 PM<br />
Acid alpha-Glucosidase <strong>Gene</strong> Replacement <strong>Therapy</strong><br />
to the Diaphragm in Ventilator-Dependent Pompe<br />
Disease: One-Year Respiratory Motor Outcomes<br />
Barbara K. Smith, Daniel Martin, Lee Ann Lawson, Cathryn S. Mah,<br />
Saleem Islam, Thomas Conlon J. Conlon, Dawn Phillips, Shelley<br />
Collins, Barry J. Byrne.<br />
58 2:15 PM<br />
Long Term CD4 Reconstitution in HIV Subjects<br />
Receiving ZFN CCR5 Modiied CD4 T-<strong>Cell</strong>s (SB-728-T)<br />
May Be Attributed to the Sustained Durability <strong>of</strong> the<br />
Central Memory T-<strong>Cell</strong> Subset<br />
Gary K. Lee, Joumana Zeidan, Jay Lalezari, Ronald Mitsuyasu,<br />
Shelley Wang, Marty Giedlin, Ge<strong>of</strong>f Nichol, Winson Tang, Dale<br />
Ando, Raick-Pierre Sekaly.<br />
59 2:30 PM<br />
Cardiovascular Toxicity and Titin Cross-Reactivity <strong>of</strong><br />
Afinity-Enhanced TCR-Engineered T <strong>Cell</strong>s Against<br />
HLA-A1 Restricted MAGE-A3 Antigen<br />
Marcela V. Maus, Gerald P. Linette, Edward A. Stadtmauer, Aaron<br />
P. Rapoport, Bruce L. Levine, Lyndsey A. Emery, Leslie Litzky,<br />
Gwendolyn K. Binder-Scholl, Dominic P. Smethurst, Andrew B.<br />
Gerry, Nick J. Pumphrey, Alan Bennet, Joanna Brewer, Jane Harper,<br />
Namir Hassan, Bent K. Jakobsen, Michael Kalos, Carl H. June.<br />
60 2:45 PM<br />
<strong>Gene</strong> <strong>Therapy</strong> for Aromatic L-Amino Acid<br />
Decarboxylase Deiciency: Report <strong>of</strong> Treatment for 8<br />
Patients<br />
Wuh-Liang Hwu, Shin-ichi Muramatsu, Sheng-Hong Tseng, Kai-<br />
Yuan Tzen, Ni-Chung Lee, Yin-Hsiu Chien, Richard O. Snyder, Barry<br />
Byrne, Chun-Hwei Tai, Ruey-Meei Wu.<br />
61 3:00 PM<br />
Safe G-CSF Mobilization <strong>of</strong> Thalassemia Patient CD34+<br />
<strong>Cell</strong>s and Effective Globin <strong>Gene</strong> Transduction for<br />
Therapeutic Infusion<br />
Farid Boulad, Xiuyan Wang, Clare Taylor, Jinrong Qu, Leda Ferro,<br />
Garie Karponi, Shirley Bartido, Patricia Giardina, Glenn Heller,<br />
Susan E. Prockop, Aurelio Maggio, Michel Sadelain, Isabelle<br />
Riviere.<br />
62 3:15 PM<br />
Hematopoietic Stem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong> with<br />
Lentiviral Vector in 4 Patients with Cerebral X-Linked<br />
Adrenoleukodystrophy: Long-Term Outcome and<br />
Comparison <strong>of</strong> Eficacy with Allogeneic Hematopoietic<br />
Stem <strong>Cell</strong> Transplantation<br />
Paul Orchard, Nathalie Cartier, Salima Hacein-Bey-Abina,<br />
Joanne Kurtzberg, Maria Escolar, Jakub Tolar, André Baruchel,<br />
Jean-Hughes Dalle, Gérard Michel, Stéphane Blanche, Cynthia<br />
Bartholomä, Manfred Schmidt, Pierre Bougnères, Christ<strong>of</strong> van<br />
Kalle, Marina Cavazzana-Calvo, Alain Fischer, Patrick Aubourg.<br />
676 3:30 PM<br />
Update on Toca 511 - a retroviral replicating vector in<br />
clinical trials for High Grade Glioma<br />
Douglas J Jolly, Joan M. Robbins, Derek Ostertag, Amy Lin, Carlos<br />
Ibanez, Harry E. Gruber, Noriyuki Kasahara, Timothy F. Cloughesy,<br />
Manish K. Aghi, Susan M. Chang, Michael A. Vogelbaum, Santosh<br />
Kesari, Tom Mikkelsen, Joseph Landoli, E. Antonio Chiocca, James<br />
Elder, Greg Foltz, Dan Pertschuk<br />
299 3:45 PM<br />
Clinical Responses in Patients Infused with<br />
T Lymphocytes Redirected To Target -Light<br />
Immunoglobulin Chain<br />
Carlos A. Ramos, Barbara Savoldo, Enli Liu, Adrian P. Gee, Zhuyong<br />
Mei, Bambi Grilley, Cliona M. Rooney, Helen E. Heslop, Malcolm K.<br />
Brenner, Gianpietro Dotti.<br />
Oral Abstract Session 231<br />
RNA Virus Vectors<br />
2:00 PM - 4:00 PM<br />
Room: Ballroom B<br />
63 2:00 PM<br />
Comprehensive Study <strong>of</strong> Lentiviral “Integrome” in WAS<br />
<strong>Gene</strong> <strong>Therapy</strong> Patients<br />
Luca Biasco, Cristina Baricordi, Francesca Dionisio, Andrea<br />
Calabria, Samantha Scaramuzza, Danilo Pellin, Clelia Di Serio,<br />
Christ<strong>of</strong> Von Kalle, Manfred Schmidt, Eugenio Montini, Luigi<br />
Naldini, Alessandro Aiuti.<br />
64 2:15 PM<br />
In Vivo Safety Evaluation <strong>of</strong> a Novel Insulated SIN LV<br />
Reveals Genotoxicity Escape-Mechanisms<br />
Monica Volpin, Daniela Cesana, Alexandre Artus, Caroline Duros,<br />
Fabrizio Benedicenti, Lucia Sergi Sergi, Luigi Naldini, Odile Cohen-<br />
Haguenauer, Eugenio Montini.<br />
65 2:30 PM<br />
No Impact <strong>of</strong> Lentiviral Transduction on Hematopoietic<br />
Stem and Progenitor <strong>Cell</strong> Telomere Length or <strong>Gene</strong><br />
Expression Proile in the Rhesus Macaque<br />
Stephanie E. Sellers, Bogden Dumitriu, Mary Morgan, Colin O. Wu,<br />
Nalini Raghavarchari, Yanqin Yang, Naoya Uchida, John F. Tisdale,<br />
Robert E. Donahue, Andre Larochelle, Cynthia E. Dunbar.<br />
92<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
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