13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 99Abstract DirectoryThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-South219Angiotensin II Type 2 Receptor <strong>Gene</strong> Mediated Apoptosis <strong>of</strong> HumanLung Adenocarcinoma A549 <strong>Cell</strong>sYanling Zhang, Ling Zhang, Yongxin Gao, Baihong Chen, Hongwei Li.Hematologic and Immunologic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I220Modeling X-Linked Chronic Granulomatous Disease Using NeutrophilsDifferentiated from Patient-Derived Induced Pluripotent Stem <strong>Cell</strong>sColin L. Sweeney, Jizhong Zou, Uimook Choi, Bin-Kuan Chou, Linzhao Cheng, Harry L.Malech.221Use <strong>of</strong> the Natural Human Artemis Promoter To Regulate HumanArtemis Expression Prevents Toxicity and Corrects Artemis DeficientSCID after Ex Vivo Lentiviral Transduction in a Murine Model <strong>of</strong> theDiseaseMegan M. Multhaup, Kelly Podetz-Pedersen, Sweta Gurram, Andrea Karlen, DebraSwanson, Nikunj V. Somia, Bruce R. Blazar, Morton J. Cowan, R. Scott McIvor, McIvorLab.222Lack <strong>of</strong> Enhancer-Blocking Activity by 400bp cHS4 Insulator in aHuman T- Lymphoid <strong>Cell</strong> lineDisha A. Mody, Taihe Lu, Sheng Zhou, John Gray, Brian P. Sorrentino.223Liver-Directed Intravascular Fetal Delivery <strong>of</strong> scAAV FacilitatesSustained Transgene Expression with Evidence <strong>of</strong> TransplacentalTrafficking and Possible Vector Integration EventsC. N. Mattar, A. C. Nathwani, N. Dighe, S. Waddington, J. McIntosh, A. Biswas, N. Fisk,A. M. David<strong>of</strong>f, A. David, D. Peebles, A. Nowrouzi, C. Kappel, M. Schmidt, M. Choolani,J. Chan.224Helper-Dependent Adenoviral Vector <strong>Gene</strong> <strong>Therapy</strong> Mediates Long-Term Low-Level Correction <strong>of</strong> Hemophilia A in the Murine Model afterNeonatal <strong>Gene</strong> TransferChuhong Hu, Fides D. Lay, Racel G. Cela, Masataka Suzuki, Joseph Springer, Ronald W.Busuttil, Brendan Lee, Gerald S. Lipshutz.225Lentiviral Vector Mediated <strong>Gene</strong> <strong>Therapy</strong> for the Treatment <strong>of</strong>Wiskott-Aldrich Syndrome: In Vitro and In Vivo Preclinical Studieswith Human CD34 + <strong>Cell</strong>sSamantha Scaramuzza, Marita Bosticardo, Anna Ripamonti, Maria C. Castiello, LucaBiasco, Giuliana Vallanti, Marina Radrizzani, Anne Galy, Robbert Bredius, BarbaraCappelli, Maria G. Roncarolo, Luigi Naldini, Anna Villa, Alessandro Aiuti.226Development <strong>of</strong> B-Lineage Specific Lentiviral Vectors for Use in <strong>Gene</strong><strong>Therapy</strong> for B-<strong>Cell</strong> DisordersBlythe D. Sather, Brigid V. Sterling, Byoung Y. Ryu, Stéphanie Baron, David J. Rawlings.227Development <strong>of</strong> a <strong>Gene</strong> Transfer System for Wiskott-AldrichSyndrome Using Foamy Virus VectorsToru Uchiyama, Masaki Shimizu, Fabio Candotti.228Integration Site Distribution in Mice Following Therapeutic <strong>Gene</strong>Transfer To Treat Beta-ThalassemiaKeshet Ronen, Olivier Negre, Nirav Malani, Maria Denaro, Beatrix Gillet-Legrand, PhilippeLeboulch, Julian D. Down, Emmanuel Payen, Frederic D. Bushman.229Validation <strong>of</strong> the Tranduction <strong>of</strong> Patients Hematopoietic Progenitor<strong>Cell</strong>s with a Clinical-Grade Lentiviral Vector Developed for Wiskott-Aldrich Syndrome <strong>Gene</strong> <strong>Therapy</strong>Salima Hacein-Bey Abina, Elena De Falco, Johanna Blondeau, Sabine Charrier, LaureCaccavelli, Mike Blundell, Alain Fischer, Alessandro Aiuti, Adrian J. Thrasher, MarinaCavazzana-Calvo, Anne Galy.230<strong>Gene</strong>ration <strong>of</strong> an Optimized Lentiviral Vector Encoding a High-Expression fVIII Transgene for <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> Hemophilia AJennifer M. Johnston, Robby Moot, Bagirath Gangadharan, Christopher B. Doering, H.Trent B. Spencer.231Biodistribution <strong>of</strong> a High Dose <strong>of</strong> Intraarticularly Administered AAV-5Vectors to Lewis RatsScott A. Loiler, Karin de Cortie, Paul P. Tak, Margriet J. Vervoordeldonk.232Ex Vivo Lentiviral Transduction <strong>of</strong> the Human Purine NucleosidePhosphorylase (PNP) <strong>Gene</strong> in Bone Marrow Derived HematopoieticStem <strong>Cell</strong>s: An In Vivo Model <strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong> for PNP DeficientT-<strong>Cell</strong> ImmunodeficiencyKelly M. Podetz-Pedersen, Megan M. Multhaup, Andrea D. Karlen, Debra L. Swanson, K.Scott Baker, Nikunj V. Somia, R. Scott McIvor.233Signed Outside: A Surface Marker System for Transgenic CytoplasmicProteinsVital Wohlgensinger, Reinhard A. Seger, Martin D. Ryan, Janine Reichenbach, Ulrich Siler.869 (Moved from Poster Session III)Myelospecific Self-Inactivating Gammaretroviral Vectors for p47 phoxCGD <strong>Gene</strong> <strong>Therapy</strong>Vital Wohlgensinger, Christian Brendel, Axel Schambach, Manuel Grez, JanineReichenbach, Reinhard A. Seger, Ulrich Siler.Thursday, May 20 th
100<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryThursday, May 20 thThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-South<strong>Gene</strong> Regulation234Low Levels <strong>of</strong> Expression <strong>of</strong> CD18 Using the pgk Promoter in aFoamy Viral Vector To Treat Animals with Canine Leukocyte AdhesionDeficiencyThomas R. Bauer, Jr., Erik M. Olson, Laura M. Tuschong, James M. Allen, Yi Li, Tanya H.Burkholder, Dennis D. Hickstein, David W. Russell.235A Dual Self-Inactivating Lentiviral Vector System for Mifepristone-Inducible Bicistronic Transgene Expression in Primary NeuralProgenitor <strong>Cell</strong>sMichael A. Mooney, Kenneth H. Fischbeck, Tyler Mark Pierson.236A Novel Human Artificial Chromosome Vector Derived fromChromosome 14 (14HAC): Higher Transgene Expression SystemUsing Sub-Telomere SequencesMinoru Kakeda, Keiko Nagata, Akiko Sano, Kanako Osawa, Akiyo Okazaki, ShingoShitara, Yasuhiro Kazuki, Mitsuo Oshimura, Kazuma Tomizuka.237Effective <strong>Gene</strong> Delivery to Wounds by Transplantation <strong>of</strong> Minced SkinParticles Transfected with Adenoviral-VEGFTaro Koyama, Florian Hackl, Pejman Aflaki, Juri Bergmann, Baraa Zuhaili, EmilyWaisbren, Feng Yao, El<strong>of</strong> Eriksson.238MicroRNA-122 Regulation Prevents Liver Toxicity <strong>of</strong> Suicide <strong>Gene</strong><strong>Therapy</strong>Gang Wang, Wenhong Tian, Yunfan Liu, Wenjie Tan, Xiaobing Wu.239Regulation <strong>of</strong> Endogenous GAD67 Expression Using ArtificialTranscription FactorsErika Elgstrand Wettergren, Fredrik Gussing, Maike Sest, Torbjörn Gräslund, CeciliaLundberg.240Enhanced In Vivo Transgene Expression by Two-Step TranscriptionAmplificationGenki Kanda, Hiroshi Ochiai, Hideyoshi Harashima, Hiroyuki Kamiya.241Targeted Knockdown <strong>of</strong> Serum Response Factor in Hearts <strong>of</strong> AdultSRF Conditional Knock-Out Mice by AAV9-Mediated Transfer <strong>of</strong> Cre-RecombinaseStanislas M. Werfel, Andreas Jungmann, Rolf Sprengel, Jürgen A. Kleinschmidt, AlfredNordheim, Hugo A. Katus, Oliver J. Müller.242Pro-Proliferative Functions <strong>of</strong> Drosophila Small Mitochondrial HeatShock Protein 22 in Human <strong>Cell</strong>sJihoon Ryu, Renu Wadhwa, Ran Gao, Il-Kyu Choi, <strong>Gene</strong>vieve Morrow, Kamal Rajput,Inwook Kim, Sunil C. Kaul, Robert M. Tanguay, Chae-Ok Yun.243Interleukin-(IL)-24 Regulates Epithelial to Mesenchymal Transition(EMT) Transcription FactorsCorbin Goerlich, Manish Shanker, Jiankang Jin, Sendurai Mani, Rajagopal Ramesh.244Transient Epigenetic <strong>Gene</strong> <strong>Therapy</strong> as a Rising Arm <strong>of</strong> a UniversalStem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong> Platform both for Ex Vivo and In VivoApplicationsRoger Bertolotti.Oligonucleotide & RNAi Therapeutics I245The Application <strong>of</strong> “Bi-Functional” Short Hairpin RNA in A FurinshRNAand GM-CSF (FANG) Autologous Vaccine for Cancer <strong>Therapy</strong>Zhaohui Wang, Connor Phalon, Padmasini Kumar, Chris M. Jay, Donald D. Rao, Alex W.Tong, Phillip B. Maples, Neil Senzer, John Nemunaitis.246A pH-Sensitive Fusogenic Peptide Modified siRNA-EncapsulatingNanoparticles Exerts the <strong>Gene</strong> Silencing Synergistically with Tumor-Specific Cleavable PEG-LipidYu Sakurai, Hiroto Hatakeyama, Hidetaka Akita, Motoi Oishi, Yukio Nagasaki, ShirohFutaki, Hideyoshi Harashima.247Systemic Administration <strong>of</strong> Bifunctional shRNA Lipoplex TargetingPDX-1 for Murine Model <strong>of</strong> Insulinoma and Pancreatic CancerShihe Liu, Zhaohui Wang, Chris M. Jay, Donald D. Rao, Nancy S. Templeton, Phillip B.Maples, Neil Senzer, F. C. Brunicardi, John Nemunaitis.248Augmentation <strong>of</strong> Docetaxel Anti-tumor Activity by a Stathmin-Specific Bi-Functional shRNAAlex W. Tong, Yang Yu, Donald D. Rao, Phillip B. Maples, Neil Senzer, John Nemunaitis.249RNAi- and U1i-Mediated <strong>Gene</strong> <strong>Therapy</strong>Annemart Koornneef, Pavlina Konstantinova, Bas Blits, Richard van Logtenstein, TitaRitsema, Puri Fortes, Harald Petry.250Lentiviral Delivery <strong>of</strong> EzH2 shRNA Inhibits Tumor Growth <strong>of</strong>Epithelial Ovarian CancerRamesh B. Batchu, Aamer M. Qazi, Assaad Semaan, Shelly M. Seward, SreedharChamala, Christopher S. Bryant, Robert Morris, Sanjeev Kumar, Christopher Steffes,Masood A. Shammas, Donald W. Weaver.251Prostate Tumor Radiosensitization through PSMA-Aptamer-TargetedsiRNA Knock-Down <strong>of</strong> DNA Repair PathwaysXiahoua Ni, Yonggong Zhang, Theodore L. DeWeese, Shawn E. Lupold.EXHIBITOR PROSPECTUSfinal program
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