FINAL PROGR AM - American Society of Gene & Cell Therapy
FINAL PROGR AM - American Society of Gene & Cell Therapy
FINAL PROGR AM - American Society of Gene & Cell Therapy
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68<br />
8th Annual Meeting • June 1 - 5, 2005 • St. Louis<br />
ABSTRACTS<br />
THURSDAY, JUNE 2<br />
367<br />
Safety and Immunological Responses Following Intrapleural<br />
Administration <strong>of</strong> an AAV5 Vector Encoding<br />
Human α1-Antitrypsin to Non-Human Primates<br />
Ben-Gary Harvey, Bishnu P. De, John P. Ayala, Neil R.<br />
Hackett, Adriana Heguy, Ronald Crystal<br />
<strong>Gene</strong> Regulation: <strong>Gene</strong> Targeting and RNA<br />
368<br />
Differential Sensitivity <strong>of</strong> Normal and CML-Derived<br />
CD34+ <strong>Cell</strong>s to Inhibition <strong>of</strong> SHP2, Gab2 and Stat5<br />
<strong>Gene</strong> Expression by RNA Interference (RNAi)<br />
Michaela Scherr, Karin Battmer, Anuhar Chaturvedi, Beate<br />
Schultheis, Arnold Ganser, Matthias Eder<br />
369<br />
Targeted <strong>Gene</strong> Modification Via Triple Helix-Forming<br />
Oligonucleotides<br />
Peter M. Glazer, Faye Rogers, Janice Lloyd<br />
370<br />
Multi-Copy, Multi-siRNA Vectors as Versatile Tools for<br />
Multiple <strong>Gene</strong> Knock-Down Applications<br />
Laura Poliseno, Monica Evangelista, Mauro Giacca, Giuseppe<br />
Rainaldi<br />
371<br />
Optimizing Custom Zinc-Finger Nucleases for Use in<br />
Human <strong>Cell</strong>s<br />
Stephen Alwin, Maja B. Gere, Eva Guhl, Karin Effertz, Carlos<br />
F. Barbas III, David J. Segal, Matthew D. Weitzman, Toni<br />
Cathomen<br />
372<br />
Targeting <strong>of</strong> Non-Viral Vectors to Specific Subnuclear<br />
Domains<br />
Joshua Z. Gasiorowski, David A. Dean<br />
373<br />
Use <strong>of</strong> Lentiviral and Adeno-Associated Vectors for<br />
Targeted <strong>Gene</strong> Inactivation in Human Cancer <strong>Cell</strong>s<br />
Sabrina Arena, Elisa Vigna, Luigi Naldini, Alberto Bardelli<br />
374<br />
Knockdown <strong>of</strong> Shp-2 Expression by siRNA in Mouse<br />
Embryonic Stem <strong>Cell</strong> Derived EB <strong>Cell</strong>s Affects Their<br />
Hematopoietic Development<br />
Gang-Ming Zou, Rebecca J. Chan, W. Christopher Shelley,<br />
Mervin C. Yoder<br />
375<br />
Targeted <strong>Gene</strong> Repair Induces the DNA Damage<br />
Response Pathways through the Activation <strong>of</strong> ATM, and<br />
the Stalling <strong>of</strong> Replication Forks Leading to a Blockage <strong>of</strong><br />
Inherited Correction<br />
Luciana Ferrara, Julia Engstrom, Erin Brachman, Miya Drury,<br />
Eric B. Kmiec<br />
376<br />
Various Aspects <strong>of</strong> the Single-Stranded DNA Fragment-<br />
Mediated <strong>Gene</strong> Correction<br />
Hiroyuki Tsuchiya, Hideyoshi Harashima, Hiroyuki Kamiya<br />
377<br />
Developing siRNAs to the E2Fs to Control Vascular<br />
Growth and Remodeling<br />
Paloma H. Giangrande, JianXin Zhang, Juliana Layzer, Otto<br />
Hagen, Joseph R. Nevins, Bruce A. Sullenger<br />
378<br />
Retargeting Mobile Group II Introns to Repair Mutant<br />
<strong>Gene</strong>s<br />
Monique N. Kierlin, John P. Jones III, Rob G. Coon, Jiri<br />
Perutka, Alan M. Lambowitz, Bruce A. Sullenger<br />
379<br />
New Zinc Finger Protein Nuclease Architectures for<br />
More Efficient <strong>Gene</strong> Modification Therapies<br />
Jeffrey C. Miller, Lei Zhang, Fyodor D. Urnov, Sarah Patterson,<br />
Danny Xia, Jeremy Rock, Sheldon Augustus, Carl O. Pabo,<br />
Philip D. Gregory, Edward J. Rebar Michael C. Holmes<br />
380<br />
Studies on the Mechanism <strong>of</strong> <strong>Gene</strong> Repair Using<br />
Saccharomyces cerevisae as a Model System<br />
Katie Maguire, Hetal Parekh-Olmedo, Eric B. Kmiec<br />
381<br />
Methods for Optimizing Lentiviral Vector-Mediated<br />
<strong>Gene</strong> Suppression by RNA Interference<br />
Eddy Kizana, Genaro Ramirez, Eugenio Cingolani, Roselle<br />
Abraham, Eduardo Marban<br />
382<br />
Expanding the Repertoire <strong>of</strong> Heterospecific LoxP Sites<br />
for Cre-Mediated Site-Specific Recombination<br />
Stephen J. Langer, Jarred Keith, Leslie A. Leinwand<br />
383<br />
Zinc Finger Nuclease-Boosted <strong>Gene</strong> Targeting &<br />
Synergistic Transient Regenerative Stem <strong>Cell</strong> <strong>Gene</strong><br />
<strong>Therapy</strong>: Toward Clinical <strong>Gene</strong> Repair/Alteration &<br />
Custom Site-Specific Integrative <strong>Gene</strong> <strong>Therapy</strong><br />
Roger Bertolotti<br />
384<br />
Double-Stranded DNA Breaks Stimulate the Frequency<br />
<strong>of</strong> Targeted <strong>Gene</strong> Repair in Mammalian <strong>Cell</strong>s<br />
Timothy Schwartz, Luciana Ferrara, Hetal Parekh-Olmedo,<br />
Eric B. Kmiec<br />
385<br />
<strong>Gene</strong> Correction in the Absence <strong>of</strong> DNA Repair: A<br />
Universal Model Involving Homologous Pairing and<br />
DNA Replication<br />
Hetal Parekh-Olmedo, Erin Brachman, Eric B. Kmiec
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