The Core Center for Gene Therapy at the University of Washington School of Medicine (UWSM) will focus on three main areas of research: 1) development of adeno-associated virus and retrovirus vectors for human applications in cystic fibrosis, 2) development of retroviral vectors for gene replacement in primary immunodeficiency syndromes, as well as lysomal storage disorders, 3) retroviral and adenoviral vectors for human application in blood clotting factor deficiencies. A total of 23 investigators who receive over $10 million in independent external support (NIH and CFF) will -regularly use the core facilities and collaborate in development of gene therapy protocols. This work will be supported by an Administrative Component and five other core facilities: Human Applications Core, Vector Development Core, Immunohistochemistry Core, Hematopoiesis Cell Transduction Core, and Animal Core. In addition, the pilot and feasibility program will consist of 5 projects which are related to CF gene therapy and 3 projects which are directed towards gene therapy in other genetic disorders. The 5 CF-related projects include the following: 1) Safety and biologic effect of AAV2/CFTR intranasal and intrabronchial administration in individuals with CF-Preclinical studies, 2) Blockade of Tumor Necrosis Factor and Interleukin-1 to enhance gene therapy in CF, 3) Adeno- associated virus for efficient gene transfer and expression in rabbit airway epithelial cells, 4) Identification of key virulence determinants of P. aeruginosa expressed in vivo and their role in lung infection in CF, 5) CF gene transfer: biliary epithelial cell pathobiology in cystic fibrosis. The 3 non CF-related projects include 1) Transduction of CD 18 gene using retroviral vectors into peripheral blood and bone marrow from children with LAD, 2) Retrovirus-mediated transfer of the human glucocerebrosidase gene into canine long-term hematopoietic stem cells, Retroviral and adenoviral mediated transfer of Factor VIII cDNA transfer into rodent hepatocytes.
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