FINAL PROGRAM - American Society of Gene & Cell Therapy

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13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 51Program ScheduleKevin V. Morris, PhDUtilizing the Endogenous Long Non-Coding RNA Pathway in Human Cells to Transcriptionally Modulate Gene ExpressionObservations over the last few years have demonstrated that non-coding RNAs can transcriptionally modulate gene expression in human cells. Recent data hasinferred long non-coding RNAs as endogenous effector molecules regulating gene transcription in human cells. Importantly, this non-coding RNA based mechanism canbe taken advantage of to either transcriptionally silence gene expression in a specific long-term manner or activate gene transcription by the targeted degradation ofgene specific regulatory long antisense non-coding RNAs.Thursday, May 20 thBakhos A. Tannous, PhDEx-Vivo Monitoring of In Vivo Gene and Cell TherapySecreted reporters are useful tools for the detection and non-invasive monitoring of different biological processes in experimental models. We have characterized thenaturally secreted Gaussia luciferase (Gluc) as a highly sensitive reporter for quantitative assessment of cells in vivo by measuring its activity in few microliters ofblood. The Gluc blood level is linear with respect to cell number and therefore is a sensitive tool for monitoring tumor growth and response to therapy, gene transfer,viral infection and replication, transcription factor activation, as well as circulating cells survival and proliferation. We also applied the Gluc assay to monitor the effectof novel gene therapy based on the constitutively active mutant human brain sodium channel as well as tumor-specific nuclear factor kappa B inducible system drivingthe expression of a suicidal gene. The Gluc blood assay complements in vivo bioluminescence imaging which has the ability to localize the signal and provides amultifaceted assessment of biological processes in experimental disease and therapy models.Charles P. Venditti, MD, PhDGene Therapy for Methylmalonic Acidemia (MMA)The hereditary methylmalonic acidemias are inborn errors of metabolism and are excellent candidates for gene and cell therapy. We have generated new insights frommouse models and patient investigations to instruct successful pre-clinical experiments. To facilitate the implementation of clinical gene therapy for MMA, we havedeveloped and validated a sensitive isotope ratio mass spectrometry technique to non-invasively monitor whole-body metabolic effects after gene delivery.
52American Society of Gene & Cell TherapyProgram ScheduleExhibit Hall Open & Networking Reception6:15 pm – 8:15 pmRoom: Exhibit Hall CThursday, May 20 thPoster Session I6:15 pm – 8:15 pmRoom: Exhibit Hall B South, CAAV Vectors I(Abstracts 57 through 72; see pages 89-90)Adenovirus and Other DNA Virus Vectors I(Abstracts 73 through 86; see pages 90-91)Targeting Gene Modification and Integration(Abstracts 87 through 99; see pages 91-92)Physical Methods of Delivery(Abstracts 100 through 116; see page 92)Chemical and Molecular Conjugates I(Abstracts 117 through 132; see page 93)Genetic and Metabolic Diseases Gene & Cell Therapy I(Abstracts 133 through 147; see pages 93-94)Musculo-skeletal Gene & Cell Therapy I(Abstracts 148 through 160; see pages 94-95)Infectious Diseases and Vaccines: Vaccine Related Gene Transfer Research(Abstracts 161 through 174; see pages 95-96)Cancer – Immunotherapy I(Abstracts 175 through 190; see pages 96-97)Cancer-Oncolytic Viruses I(Abstracts 192 through 204; Abstract 191 moved to Oral Session 413; see pages 97-98)Cancer-Targeted Gene & Cell Therapy I(Abstracts 205 through 219; see pages 98-99)Hematologic and Immunologic Gene & Cell Therapy I(Abstracts 220 through 233 and Abstract 869 which has been moved from Poster Session III; see page 99)Gene Regulation(Abstracts 234 through 244; see page 100)Oligonucleotide & RNAi Therapeutics I(Abstracts 245 through 259; see pages 100-101)Stem Cell Biology(Abstracts 260 through 273; see pages 101-102)Cell Processing and Vector Production I(Abstracts 274 through 282; see pages 102)Late Breaking Science I(Abstracts 922 through 932; Abstract 926 withdrawn; see pages 102-103)EXHIBITOR PROSPECTUSfinal program
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