|
|
|||
| |||||||||||||||||||||||||||||||||||||||||||||
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,
Source Information
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.
Extract |
Full Text |
PDF
N Engl J Med 2003;
348:255-256, Jan 16, 2003.
Correspondence
This article has been cited by other articles:
HOME | SUBSCRIBE | SEARCH | CURRENT ISSUE | PAST ISSUES | COLLECTIONS | PRIVACY | HELP | beta.nejm.org Comments and questions? Please contact us. The New England Journal of Medicine is owned, published, and copyrighted © 2009 Massachusetts Medical Society. All rights reserved. |