MOLMED 504: Challenges in Gene Therapy

Spring 2008

	Course Chair: Andrew Scharenberg, SCHRI

Session 1 May 1, 2008	Prof. Alain Fischer INSERM Gene Therapy Laboratory, Necker Hospital, Paris "Results and Perspectives of Gene Therapy in Human SCID" Vector Integration is Nonrandom and Clustered... Multilineage Hematopoietic Reconstitution without Clonal Selection... Long-Term Immune Reconstitution in RAG-1-Deficient Mice... Gene Therapy of Human Severe Combined Immunodeficiency... Gene Therapy for Primary Immunodeficiencies

Session 2 May 8, 2008	Dr. Michael Holmes Sangamo Biosciences "Engineered Zinc Finger Nucleases: Editors of Genome Sequence and Function" The Zinc Finger Nuclease Monopoly An Improved Zinc-Finger Nuclease Architecture... Targeted Gene Addition into a Specified Location... Targeted Gene Knockout in Mammalian Cells..

Session 3 May 15, 2008	Prof. Phil Greenberg Professor of Medicine and Immunology, UW School of Medicine "Engineering T Cells for the Treatment of Human Disease" Adoptive Immunotherapy: Engineering T Cell Responses... Optimizing Adoptive Polyclonal T Cell Immunotherapy... Antigen Sensitivity of CD22-Specific Chimeric TCR... Facilitating Matched Pairing and Expression of TCR Chains... Transfection of RNA Encoding...

Session 4 May 22, 2008	Prof. Katherine High Professor of Pediatrics, University of Pennsylvania School of Medicine "AAV-Mediated Gene Transfer for Genetic Disease" Successful Transduction of Liver in Hemophilia... Immune Responses to AAV in Clinical Trials Effects of Transient Immunosuppression... Gene Therapy for Haemophilia

Session 5 May 29, 2008	Dr. Dusty Miller Fred Hutchinson Cancer Research Center and UW Department of Pathology "Lung Gene Therapy" Prevalence of Neutralizing Antibodies Against Adeno-Associated Virus... Jaagsiekte Sheep Retrovirus Envelope... Adeno-Associated Virus Type 6 Vectors... Oncogenic Transformation by the Jaagsiekte Sheep Retrovirus Envelope Protein High Efficiency Promoter-Dependent Transduction...

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Course List