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Volume 348:193-194 January 16, 2003 Number 3
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Risks and Benefits of Gene Therapy
Philip Noguchi, M.D.

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Updated information on this topic from the Food and Drug Administration is available at http://www.fda.gov/bbs/topics/ANSWERS/2003/ANS01190.html.

Although most of today's gene-therapy trials are targeted to cancer, there is renewed interest in pursuing the goal for which gene therapy was invented: the cure of genetic disease. Recent studies from France, the United Kingdom, and Italy have provided encouraging results in the treatment of several forms of a rare, devastating disease of infancy, collectively called severe combined immunodeficiency. Each form of this disease is caused by a mutation in a single gene. In these studies, a modified retrovirus was used to insert, . . . [Full Text of this Article]


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From the Food and Drug Administration, Rockville, Md.


Related Letters:

A Serious Adverse Event after Successful Gene Therapy for X-Linked Severe Combined Immunodeficiency
Hacein-Bey-Abina S., von Kalle C., Schmidt M., Le Deist F., Wulffraat N., McIntyre E., Radford I., Villeval J.-L., Fraser C. C., Cavazzana-Calvo M., Fischer A.
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N Engl J Med 2003; 348:255-256, Jan 16, 2003. Correspondence

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