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January 4, 1985

Clinical stage draws nearer in ongoing studies of gene therapy

JAMA. 1985;253(1):13-15. doi:10.1001/jama.1985.03350250013001

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It may be a matter of three years or less before clinical trials are undertaken for the study of gene therapy of inherited disease.

That is the estimation of C. Thomas Caskey, MD, director of the Robert J. Kleberg, Jr, Center for Human Genetics at Baylor College of Medicine, Houston. It is an opinion shared by numerous other investigators.

(Two unsuccessful attempts to undertake gene transfer involving women with β0-thalassemia were performed abroad in 1980 by Martin J. Cline, MD, of the University of California, Los Angeles, School of Medicine. Although Cline had permission of the local hospitals abroad to perform the experiments, the UCLA institutional review board rejected his proposal on the grounds that further animal studies were needed, and he lost his National Institutes of Health grants.)

Caskey, who spoke at the annual Short Course in Medical and Experimental Mammalian Genetics in Bar Harbor, Me, which