FINAL PROGRAM - American Society of Gene & Cell Therapy

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13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 53Program ScheduleFoundation Symposium 2507:00 pm - 10:00 pmRoom: Thurgood Marshall WestExpanding Frontiers in Spinal Muscular Atrophy Gene TherapyChairChristian L. Lorson, PhDSpeakersChristian L. Lorson, PhDIntroduction: Perspectives on SMA TherapeuticsC. Frank Bennett, PhDRNA Targeting Therapeutics for the Treatment of Motor Neuron DiseasesMarco Passini, PhDGene and Antisense Therapies in SMA MiceBrian K. Kaspar, PhDRescue of a Spinal Muscular Atrophy Mouse Model by Early Postnatal Delivery of SMN with scAAV9 and Clinical Development PlansPetra Kaufman, MDSMA Clinical Trials – NINDS PerspectiveThursday, May 20 thFoundation Symposium 250 is supported by the following ASGCT partners:
54American Society of Gene & Cell TherapyProgram ScheduleFriday, May 21Friday, May 21 stPlenary Abstract Session 3008:00 am - 10:00 amRoom: Marriott Ballroom Salons 1, 2 & 3Top Oral Abstracts Plenary SessionCo-ChairsStanton L. Gerson, MD & John J. Rossi, PhDSpeakersMervin C. Yoder, MD IntroducesEirini P. Papapetrou, MD, PhDTherapeutic Transgene Expression in Patient-Specific iPS Cells after Selection of “Safe Harbors” for Lentiviral Vector IntegrationJames Ellis, PhD IntroducesAxel Schambach, MD PhDA Novel Lentiviral Expression System Reveals Patchwork Colony Formation during Reprogramming to Induced Pluripotent Stem CellsDieter C. Gruenert, PhD IntroducesErica B. Schleifman, BSTargeted Genomic Modification and Inactivation of the CCR5 Gene Using PNA-Induced Homologous RecombinationMatthew H. Porteus, MD, PhD IntroducesRichard Gabriel, Dipl.-Biol.Lentiviral Microarrays for High-Throughput and Real-Time Monitoring of Gene Expression DynamicsBruce Sullenger, PhD IntroducesJiehua Zhou, PhDSystemic Administration of Sendrimer- and Aptamer-siRNA Conjugates Efficiently Suppresses HIV-1 InfectionChiara Bonini, MD IntroducesAntonio Di Stasi, MDCASPALLO: Phase I Clinical Trial of Allodepleted T Cells Transduced with Inducible Caspase 9 Suicide Gene after Haploidentical Stem Cell TransplantationBreak10:00 am – 10:30 amScientific Symposium 31010:30 am - 12:30 pmRoom: Virginia SuitePersonalized Cell and Gene TherapyChairJohn J. Nemunaitis, MDSpeakersJohn J. Nemunaitis, MDSuccess of Gene Therapy - A Personalized and Personal ApproachClinical use of molecular science is coming of age! Numerous targeted therapies, many related to gene based technology, in particular involving RNA interference platforms,are undergoing clinical track development. Mechanisms for cancer specific plasmid delivery are unfolding in the clinical arena. Furthermore, gene based vaccinesare showing improved promise. These dynamics combined with more efficient and economic quantitative proteogenomic technology development will enable a personalizedapproach to cancer therapy. Results of personalized gene and cell based therapies will be discussed.EXHIBITOR PROSPECTUSfinal program
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