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Gene Therapy

W. French Anderson, MD
JAMA. 1981;246(23):2737-2739. doi:10.1001/jama.1981.03320230059029.
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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


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The American Medical Association is accredited by the Accreditation Council for Continuing Medical Education to provide continuing medical education for physicians. The AMA designates this journal-based CME activity for a maximum of 1 AMA PRA Category 1 CreditTM per course. Physicians should claim only the credit commensurate with the extent of their participation in the activity. Physicians who complete the CME course and score at least 80% correct on the quiz are eligible for AMA PRA Category 1 CreditTM.
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