13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010107Abstract DirectoryFriday, May 21, 2010Oral Abstract Session 324 — continuedPhysical Methods <strong>of</strong> Delivery2:00 PM - 4:00 PMRoom: Wilson ABC327 3:30 PMA Novel Prototype Device for Electroporation-Enhanced DNA VaccineDelivery to Both Skin and MuscleFeng Lin, Jay McCoy, Xuefei Shen, Janess Mendoza, Kate E. Broderick, Jian Yan, SteveKemmerrer, David B. Weiner, Niranjan Y. Sardesai.328 3:45 PMSafety and Feasibility <strong>of</strong> Transvenous Limb Perfusion with Saline inHuman Muscular DystrophyWilliam Powers, Zheng Fan, Keith Kocis, Robert Valley, Manisha Chopra, James Howard,Jr, Joseph Muenzer.Oral Abstract Session 325Cancer-Targeted <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>2:00 PM - 4:00 PMRoom: Thurgood Marshall W334 3:15 PMTumor-Selectivity <strong>of</strong> Replication-Competent Retrovirus (RCR) VectorsRegulated by Prostate-Specific Promoters and Cytotoxic Effect <strong>of</strong>Suicide <strong>Gene</strong> <strong>Therapy</strong> Mediated by RCR Vectors In VitroShuichi Kamijima, Takahiro Kimura, Kazunori Haga, Kei Hiraoka, Akihito Inagaki,Christopher R. Logg, Chinghai Kao, Bernard H. Bochner, Noriyuki Kasahara.335 3:30 PMTranslational Phase I Study in Advanced Cancer Patients <strong>of</strong> Dual-Function Autologous (FANG) Vaccine Incorporating BifunctionalshRNA furin and GMCSF Transgene ExpressionNeil Senzer, Padmasini Kumar, Joseph Kuhn, Minal Barve, Cynthia Bedell, Yang Yu, AlexW. Tong, Phillip B. Maples, John Nemunaitis.336 3:45 PMEfficient <strong>Gene</strong> Transfer in Quiescent Healthy and Cancer B <strong>Cell</strong>s by aMeasles Virus gp Pseudotyped LVCecilia Frecha, Caroline Costa, Camille Levy, Didier Negre, Andrea Maisner, Stephen J.Russell, Nicholas Rachinel, Gilles Salles, Francois-Loic Cosset, Els Verhoeyen.Oral Abstract Session 326Hematologic/Immunologic: Basic2:00 PM - 4:00 PMRoom: Maryland Suite329 2:00 PMA <strong>Gene</strong>tically Engineered Multifunctional Vector for HER2 TargetedCancer Suicide <strong>Gene</strong> <strong>Therapy</strong>Yuhua Wang, Arash Hatefi.330 2:15 PMComparative Analysis <strong>of</strong> Enzyme and Pathway Engineering Strategiesfor Suicide <strong>Gene</strong> <strong>Therapy</strong> ApplicationsAdam J. Johnson, Andressa Ardiani, Kinta Serve, Marilyn Sanchez-Bonilla, Margaret E.Black.331 2:30 PMConditional and Effective <strong>Gene</strong> Knock-Down in ProangiogenicMacrophages To Disrupt Tumor AngiogenesisFerdinando Pucci, Roberta Mazzieri, Bernhard Gentner, Luigi Naldini, Michele De Palma.332 2:45 PM<strong>Cell</strong> Fusion between Mesenchymal Stem <strong>Cell</strong>s and Cancer <strong>Cell</strong>s: AMechanism <strong>of</strong> Cancer ProgressionYan Xiao, Dirk H. Hondmann, Carla J. Aldrich, Chenzhong Kuang.333 3:00 PMNext-<strong>Gene</strong>ration Lentiviral <strong>Cell</strong> Fate Control <strong>Gene</strong> <strong>Therapy</strong> Based onEngineered Variants <strong>of</strong> Human Deoxycytidine KinaseAnton Neschadim, Arnon Lavie, Takeya Sato, Daniel H. Fowler, Vladimir Ponomarev,Jeffrey A. Medin.337 2:00 PMSafely Exploiting Hematopoeitic Stem and Progenitor <strong>Cell</strong> SpecificmicroRNAs To Target Transgene Expression to the DifferentiatedProgenyBernhard Gentner, Alice Giustacchini, Francesco Boccalatte, Massimo Saini, Giulia Schira,Luigi Naldini.338 2:15 PMTherapeutic Fetal Hemoglobin Production in Erythroid Progeny <strong>of</strong>Normal and b-Thalassemic Human CD34 + <strong>Cell</strong>s Using LentiviralVectors Encoding g-Globin or shRNA To Down Modulate theTranscriptional Repressor BCL11AAndrew Wilber, Vijay G. Sankaran, Phillip W. Hargrove, Yoon-Sang Kim, EleniPapanikolaou, Nicholas P. Anagnou, Stuart H. Orkin, Arthur W. Nienhuis, Derek A.Persons.339 2:30 PMPotentially Therapeutic Levels <strong>of</strong> Fetal Hemoglobin in ErythroidProgeny <strong>of</strong> Normal and b-Thalassemic CD34 + <strong>Cell</strong>s FollowingLentivirus-Mediated Delivery <strong>of</strong> a Zinc-Finger TranscriptionalActivator Targeting the g-Globin <strong>Gene</strong> PromotersAndrew Wilber, Ulrich Tschulena, Phillip W. Hargrove, Yoon-Sang Kim, Carlos F. Barbas,III, Derek A. Persons, Arthur W. Nienhuis.340 2:45 PMDevelopment <strong>of</strong> an S/MAR Based Episomal Vector <strong>of</strong> a Specific Zinc-Finger Activator That Mediates Gamma-Globin <strong>Gene</strong> Activation, forthe <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> HemoglobinopathiesEleana Stavrou, Eleni Lagadinou, Nikolas Zoumbos, Alexandros Spyridonidis, CarlosBarbas, III, Kenneth Peterson, Aglaia Athanassiadou.Friday, May 21 st
108<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, 2010Oral Abstract Session 326— continuedHematologic/Immunologic: Basic2:00 PM - 4:00 PMRoom: Maryland Suite341 3:00 PMEngineered Human Regulatory T <strong>Cell</strong>s Expressing Lentiviral PDL1under <strong>Cell</strong> Fate Control Prevent Lethal Xenogeneic GVHDShoba Amarnath, James Wang, Courtney Mangus, James Riley, Bruce Levine, Carl June,Jeffrey Medin, Daniel Fowler.342 3:15 PMCorrection <strong>of</strong> Murine Hemophilia A Following NonmyeloablativeTransplantation <strong>of</strong> Hematopoietic Stem <strong>Cell</strong>s Engineered To Expressa Bioimproved Human FVIII Using a Safety-Augmented, B-LymphoidLineage-Restricted Lentiviral VectorAli Ramezani, Sara Karandish, Lynnsey A. Zweier-Renn, Teresa S. Hawley, Robert G.Hawley.343 3:30 PMLentiviral Vectors Containing an Endogenous Btk Promoter RestoreB and Myeloid Lineage Expression and Function in a Murine Model <strong>of</strong>X-Linked AgammaglobulinemiaHannah M. Kerns, Byoung Y. Ryu, Brigid V. Stirling, Blythe D. Sather, AlexanderAstrakhan, Stephanie Humblet-Baron, Denny Liggitt, David J. Rawlings.344 3:45 PMEarly Gestational <strong>Gene</strong> Transfer <strong>of</strong> IL-10 by Lentiviral Vector CanPrevent Arthritis in a Murine ModelJessica L. Roybal, Masayuki Endo, Antoneta Radu, Carlyn Todorow, Philip W. Zoltick,Alan W. Flake.Poster Session II6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-SouthRNA Virus Vectors I345Expression Pr<strong>of</strong>ile and Epigenetic Landscape Influence in a <strong>Cell</strong>-Specific Fashion Integration Site Selection and In Vivo Distribution <strong>of</strong>MLV-Vector in PatientsLuca Biasco, Alessandro Ambrosi, Danilo Pellin, Cynthia Bartholomae, ImmacolataBrigida, Maria Grazia Roncarolo, Clelia Di Serio, Christ<strong>of</strong> Von Kalle, Manfred Schmidt,Alessandro Aiuti.346High-Definition Mapping <strong>of</strong> Retroviral Integration Sites Defines theFate <strong>of</strong> Allogeneic T <strong>Cell</strong>s after Donor Lymphocyte InfusionAlessandra Recchia, Claudia Cattoglio, Danilo Pellin, Giulietta Maruggi, Serena KimiPerna, Alessandro Ambrosi, Clelia Di Serio, Nirav Malani, Frederic D. Bushman, CynthiaBartholomae, Manfred Schmidt, Chiara Bonini, Fulvio Mavilio.347Genome Wide Characterization <strong>of</strong> Insertion Pr<strong>of</strong>iles <strong>of</strong> IntegrationDeficient Lentiviral Vectors Using Two-Sided Non-Restrictive andStandard LAM-PCRWei Wang, Cynthia C. Bartholomae, Anne Arens, Richard Gabriel, Hanno Glimm, Christ<strong>of</strong>von Kalle, Manfred Schmidt.348Multiple Pathways Facilitate Transduction <strong>of</strong> Quiescent HumanCD34+ <strong>Cell</strong>s by HIV-1 VectorsCathy X. Wang, Charles DeRossi, Bruce E. Torbett.349Targeted Lentiviral <strong>Gene</strong> Delivery to <strong>Cell</strong>s Expressing c-fms or IL-2ReceptorAnil Chandrashekran, Giedre Milinkeviciute, Rosana Cardoso, Fouzia Abdulrasul, Shao-AnXue, Nick Dibb, Carol Readhead, Colin Casimir.350Regulation <strong>of</strong> Transgene Expression In Vivo by ProteasomalDegradationKhalid Tai, Fredrik Gussing, Christina Isaksson, Luis Quintino, Mari Iwamoto, Tom J.Wandless, Cecilia Lundberg.351Clonal Analysis Allows Characterization and Risk Assessment<strong>of</strong> Viral-<strong>Cell</strong>ular Fusion Transcripts <strong>Gene</strong>rated by TranscriptionalTermination Read-Through in Keratinocytes Transduced with Self-Inactivating Retroviral VectorsDavid Almarza, Fernando Larcher, Rodolfo Murillas, Fulvio Mavilio.352Evaluation <strong>of</strong> Cytokine Effects on Lentiviral Transduction <strong>of</strong> HumanCD34 + <strong>Cell</strong>sNaoya Uchida, Kareem N. Washington, Matthew M. Hsieh, John F. Tisdale.353Towards Quantitative High Throughput Clonality Screening:Redefining the Frontier <strong>of</strong> Retroviral Integration AnalysisTroy B. Hawkins, Aparna Jasti, Jessica Dantzer, Nate Van Dusen, Brandon Downing,Sean Mooney, Mary C. Dinauer, Keithanne Mockaitis, Kenneth G. Cornetta.354Development <strong>of</strong> a pCL20c-Backbone Based HIV Lentivirus Vector ThatAchieves Efficient Functional Correction <strong>of</strong> Neutrophils Arising fromTransduced CD34+ Hematopoietic Progenitor <strong>Cell</strong>s <strong>of</strong> Patients withX-Linked Chronic Granulomatous DiseaseUimook Choi, Robert E. Throm, Elizabeth M. Kang, Suk See DeRavin, Narda Theobald,Gilda M. Linton, Brian P. Sorrentino, Mary C. Dinauer, John T. Gray, Harry L. Malech.355Packaging Heterologous Retroviral Vectors into HIV-1 Viral ParticlesAdam S. Cockrell, Henriette van Praag, Nicholas Santistevan, Thomas J. McCown, BonitaBlake, Hong Ma, John Olsen, Tal Kafri.356High Throughput Vector Integration Analysis with SeqMap 2.0Troy B. Hawkins, Jessica Dantzer, Brandon Peters, Mary Dinauer, Keithanne Mockaitis,Sean Mooney, Kenneth G. Cornetta.EXHIBITOR PROSPECTUSfinal program
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