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Nat Rev Cancer. 2003 Jul;3(7):477-88.

Occurrence of leukaemia following gene therapy of X-linked SCID.

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1
Division of Research Immunology/BMT, Childrens Hospital Los Angeles, USC Keck School of Medicine, 4650 Sunset Boulevard, Los Angeles, California 90027, USA. dkohn@chla.usc.edu

Erratum in

  • Nat Rev Cancer. 2003 Nov;3(11):883.

Abstract

Recombinant viral vectors have allowed gene transfer to be developed as a promising approach to the treatment of genetic diseases. Recently, gene therapy of children with X-linked severe combined immune deficiency resulted in impressive levels of immune reconstitution--a triumph that was later overshadowed by the development of leukaemia in two patients. What were the causes of this cancer, and how can the therapeutic benefits of gene therapy be achieved while minimizing risk to the patient?

PMID:
12835668
DOI:
10.1038/nrc1122
[Indexed for MEDLINE]
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