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Science. 2003 Oct 17;302(5644):400-1.

Medicine. Gene therapy--new challenges ahead.

Author information

  • 1Division of Experimental Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA. david.williams@cchmc.org

Abstract

The successful use of retroviral gene transfer to treat 10 patients with X-linked severe combined immunodeficiency (SCID-X1) has been welcomed as evidence of the therapeutic potential of gene therapy. However, as Williams and Baum suggest in their Perspective, the discovery that 2 of the 10 patients developed leukemia within 3 years of gene therapy (Hacein-Bey-Abina et al.) reinforces the need to develop even more specific gene therapy interventions.

PMID:
14563994
DOI:
10.1126/science.1091258
[PubMed - indexed for MEDLINE]
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