Abstract
The state of the art in research on human gene therapy is summarized. Clinical trials are estimated to be at most three years away for some single gene defects, with current research focused on somatic cell rather than germline therapy. A gene therapy subcommittee of the National Institutes of Health's Recombinant DNA Advisory Committee is drafting guidelines for investigators who are planning to submit their research protocols to the committee for review.
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More From: JAMA: The Journal of the American Medical Association
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