The Core Center for Gene Therapy at the University of Washington School of Medicine (UWSM) is under the direction of A. Dusty Miller, Ph.D. (Program Director), Affiliate Professor of Pathology and a Member of the Fred Hutchinson Cancer Research Center, and Associate Program Direction Bonnie W. Ramsey, M.D., Professor of Pediatrics. The Center brings together gene therapy research efforts at UWSM, the Fred Hutchinson Cancer Research Center, Children's Hospital and Medical Center, and the Veteran's Administration Medical Center in Seattle, and includes close ties with the Cystic Fibrosis Research Program headed by Dr. Bonnie Ramsey, and the General Clinical Research Center (GCRC) headed by John Brunzell, M.D. The Center will focus on three main areas of research: 1) development of viral vectors and procedures for the treatment of cystic fibrosis, 2) development of retroviral vectors for gene transfer and expression of therapeutic genes in hematopoietic and lymphoid cells in humans, and 3) development of retroviral vectors of gene transfer and expression of therapeutic genes in hematopoietic and lymphoid cells in humans, and 3) development of methods to deliver circulating proteins such as erythropoietin and clotting factors fro treatment of human disease. A total of 35 investigators who receive over $10 million in independent external support will regulatory 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, Hematopoietic Cell Transduction Core, and Animal Core. In addition, the pilot and feasibility program will consist of 8 projects which are related to CF gene therapy and 2 projects which are directed towards gene therapy in other genetic disorders. The CF-related projects include the following: 1) The Biology of Adeno-Associated Virus and Vector (J. Allen and D. Miller), 2) Recruitment of the Adenoviral Pre-terminal Protein (pTP) for nuclear Import of LV-DNA in Non-Dividing Cells (A. Lieber), 3) Identification of P. aeruginosa Virulence Determinants using an Invertebrate Model (C. Manoil), 4) Organ immunosuppression for donor lungs: Optimizing a safe approach using non-viral gene therapy (M. Allen), 5) Evaluation of lentiviral vectors for airway gene therapy (C. Halbert), 6) Refining non-viral gene therapy approaches for CF (M. Horowitz), 7) CFTR gene targeting by adeno-associated virus vectors (D. Russell), 8) Improving transgene expression by adenoviral vectors modified to co-express murine CD8 molecules (P. Fink). The 2 non CF-related projects include 1) Development of a coumermycin-responsive proliferation switch (C.A. Blau) and 2) A Novel approach for the modulation of host immune responses to gene modified cells (H.P. Kiem).
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