
First human gene therapy trial was conducted
On Sept. 14, 1990, W. French Anderson and his colleagues at the National Institutes of Health (NIH) performed the first approved gene therapy procedure on a four-year-old girl, Ashanti DeSilva, who became the first gene therapy patient. She had adenosine deaminase (ADA) deficiency, a genetic disease which left her defenseless against infections.
The NIH team took white blood cells from her, and the normal genes for making adenosine deaminase were inserted into them. The corrected cells were reinjected into her. Dr. Anderson helped develop this landmark clinical trial when he worked at the National Heart, Lung, and Blood Institute. Gene therapy is defined as the treatment of disease by transfer of genetic material into cells.
Despite the viral horror stories written by the popular media, this initial trial was largely a success, and the most recent report on this individual in 2004 noted that she is thriving as an 18-year-old teenager in suburban Cleveland.
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Source: National Institutes of Health
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