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Nature. 2006 Apr 27;440(7088):1123.

Gene therapy: therapeutic gene causing lymphoma.

Author information

  • 1Laboratory of Genetics, The Salk Institute for Biological Studies, La Jolla, California 92037, USA.

Abstract

The development of T-cell leukaemia following the otherwise successful treatment of three patients with X-linked severe combined immune deficiency (X-SCID) in gene-therapy trials using haematopoietic stem cells has led to a re-evaluation of this approach. Using a mouse model for gene therapy of X-SCID, we find that the corrective therapeutic gene IL2RG itself can act as a contributor to the genesis of T-cell lymphomas, with one-third of animals being affected. Gene-therapy trials for X-SCID, which have been based on the assumption that IL2RG is minimally oncogenic, may therefore pose some risk to patients.

Comment in

  • Gene therapy: is IL2RG oncogenic in T-cell development? [Nature. 2006]
  • Gene therapy: X-SCID transgene leukaemogenicity. [Nature. 2006]
PMID:
16641981
[PubMed - indexed for MEDLINE]

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