BASIC SCIENCE STUDIES ON GENE THERAPY OF BLOOD DISEASES

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This is a new application for a training program to support 6 postdoctoral trainees (M.D. and/or Ph.D.) and 6 predoctoral students (M.D./Ph.D.) per year with a strong emphasis on cell and molecular biology/immunology of gene therapy. The average training period for postdoctoral and predoctoral students will be 3 years and 4 years, respectively. Thirteen well-established preceptors will provide extensive training in highly active as well as interactive research laboratories pursuing state of the art research on fundamental aspects of cellular, molecular biological, and immunological aspects of a variety of human diseases, and the prospects of developing noel gene therapy approaches for some of these disorders. Most of the preceptors are housed in newly built or renovated spacious laboratories in the Department of Microbiology and Immunology; Division of Hematology/Oncology, Department of Medicine; Department of Medicinal and Molecular Genetics; Herman B Wells Center for Pediatric Research, Department of Pediatrics; and the Walther Oncology Center, in three adjoining buildings. Strong track-records of peer-reviewed publications and extramural funding of each of the preceptors a major strength of the proposed training program. Ample opportunities for pursuing multi- disciplinary research exist due to active, ongoing scientific collaborations among various preceptors. A significant emphasis is placed to promote and foster the development of the next generation of biomedical researchers committed to scientific careers in academic medicine to become independent investigators who, in turn, will themselves be productive and competitive. A multi-pronged approach for staff members and visiting scientists; formal and informal courses and seminars; laboratory meetings; and participation in state, national and international scientific meetings, is already in place and will be further emphasized. The decision to associate with a laboratory will be made by mutual consent of the trainee and the preceptor, and the scientific development of the trainee will be facilitated by a research committee composed of preceptors with multi-disciplinary research interests. Major resources that are available include internationally recognized clinical and basic science research programs, one of only three NIH-designated National Gene Vector Laboratories in the U.S., state of the art infrastructure, and frequent contacts with active investigators at Indiana University in Bloomington, Purdue University in West Lafayette, all in close proximity, and other investigators nationally as well as internationally.