Gene Transfer/Gene Therapy

The Gene Transfer/Gene Therapy Program is currently supported by a five-year Training Grant from the National Institutes of Health. The training program is designed to develop the next generation of scientific investigators in gene transfer/gene therapy since it has become increasingly clear that new scientific advances will undoubtedly influence the treatment and management of a number of human genetic diseases, both inherited and acquired. Although a number of clinical gene therapy trials are currently underway, the importance of basic research has repeatedly been emphasized. By focusing on cellular, molecular, and immunological aspects, a better understanding of the disease processes will be gained, ultimately leading to the potential cure of these diseases. It has also become abundantly clear that a multi-pronged approach is needed to address these complex issues since these disorders are no longer confined to a single domain or a single discipline. In addition to the fundamental research in basic sciences, an understanding of the clinical aspects of the disease is also of paramount importance. Ideally, continued progress in advancing treatment of disease will require investigators well-trained and knowledgeable in basic as well as clinical aspects of gene therapy. Several well-established preceptors provide extensive training in highly active research laboratories pursuing state of the art research on fundamental aspects of cellular and molecular biological aspects of a variety of human diseases, and the prospects of developing novel gene therapy approaches for some of these disorders.These preceptors form a cohesive, complementary and interactive group of investigators. All of the preceptors are situated in close proximity to each other.The joint departmental and research center appointments of the preceptors encourage collaborations, multi-disciplinary approaches to solving scientific problems, and enhance learning and productivity. Each of the preceptors has varied backgrounds and experiences. All of them are interested in the problems related to the cellular and molecular biology of human gene therapy. The diverse backgrounds of the investigators are viewed as a major strength of the program for it allows for a multi-disciplinary approach for providing optimal training experience to predoctoral students and postdoctoral fellows. Strong track-records of peer-reviewed publications and extramural funding of each of the preceptors are also a major strength of the training program. Ample opportunities for pursuing multi-disciplinary research exist due to active, ongoing scientific collaborations among various preceptors. A multi-pronged approach for comprehensive training encompassing close and direct contact with preceptors; frequent, informal interactions with staff members and visiting scientists; formal and informal courses and seminars; laboratory meetings; and participation in state, national and international scientific meetings, is in place and is emphasized. Collectively, the preceptors have trained (and are in the process of training) 36 predoctoral students and 140 postdoctoral fellows in the past ten years. Our trainees have been productive and have published extensively in peer-reviewed and respected scientific journals. 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., an NIH-designated Cancer Research Center, and state of the art infra-structure.

• Hal E. Broxmeyer, Ph.D.
• Wade Clapp, M.D.
• Ken Cornetta, M.D.
• Thomas Gardner, M.D.
• Laura Haneline, M.D.
• Chinghai Kao, Ph.D.
• Louis Pelus, Ph.D.
• Edward F. Srour, Ph.D.

If you wish to receive application material for our Departmental Graduate Program, please go to the How to Apply page.

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Hal Broxmeyer named distinguished professor at IUSM (click here for more details)

Janice Blum discusses Immunology & Infectious Diseases (click here for more details)