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Joan Stephenson, PhD

JAMA. 2000;283:589-590.

	Since this article does not have an abstract, we have provided the first 150 words of the full text and any section headings.

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.

BURSTING SCID'S BUBBLE?

In one report, a team of French investigators described encouraging results in a trial for X-linked severe combined immunodeficiency disease (SCID), the most common form of human SCID. This disorder, which occurs in about 1 in 50,000 births, is caused by a mutation in a component (the . . . [Full Text of this Article]

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