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Medical News & Perspectives
February 2, 2000

Gene Therapy Trials Show Clinical Efficacy

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JAMA. 2000;283(5):589-590. doi:10.1001/jama.283.5.589

New Orleans—In the wake of the highly publicized and tragic death of a patient who participated in a gene therapy trial at the University of Pennsylvania comes some good news: encouraging early results from two studies reported here at the annual meeting of the American Society of Hematology, one aimed at a lethal form of inherited immunodeficiency and the other targeting hemophilia B.

If the promising but preliminary results from the two phase 1 trials hold up, the two studies may be the first to provide evidence of clinical benefit for patients treated with gene therapy.

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