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December 11, 1981

Gene Therapy

Author Affiliations

From the Laboratory of Molecular Hematology, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Md.

JAMA. 1981;246(23):2737-2739. doi:10.1001/jama.1981.03320230059029

Can Gene Therapy Help Me?  This article is designed to help the practicing physician answer the patient with a genetic disease who asks, "Can gene therapy help me?" The answer depends on the patient's disease, but for some genetic conditions, gene therapy should be a beneficial therapeutic procedure in the coming years. This brief review examines the present state of the art, points out what still needs to be accomplished before gene therapy will be practical, and concludes by suggesting the potential and the limitations of this promising, but not yet available, technology.Progress in the area of "genetic engineering" has been spectular in recent years. Not only are bacteria being programmed to produce medically useful compounds (insulin, growth hormone, etc), but genes have been transferred into mammalian cells successfully. Individual cells in tissue culture have been cured of genetic defects by insertion of a normal gene.1-3 Purified genes