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First gene therapy in humans proposed.

First gene therapy in humans proposed

Researchers at the National Institutes of Health (NIH) in Bethesda, Md., have filed the first formal request to inject into humans genetically engineered cells custom-designed to treat an inherited disease. The cells will produce an enzyme -- adenosine deaminase, or ADA -- missing in children suffering from a hereditary disorder called severe combined immunodeficiency. Because these children are incapable of mounting a proper immune response against disease-causing micro-organisms, they must live in sterile environments and rarely live beyond their first few years.

R. Michael Blaese, Kenneth Culver and W. French Anderson submitted their proposal to the NIH Feb. 23. The experimental protocol builds upon ongoing experiments by some of the same researchers, in which genetically engineered cells are being used not to cure patient but to learn more about naturally occurring cancer-fighting cells (SN: 5/27/89, p. 324).

Federal officials will review the proposal at a series of meetings during the month of March. Approval or rejection isn't expected until at least June.
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Publication:Science News
Date:Mar 10, 1990
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