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FINAL PROGRAM - American Society of Gene & Cell Therapy

FINAL PROGRAM - American Society of Gene & Cell Therapy

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

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