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Brief Communications
Nature 440, 1123 (27 April 2006) | doi:10.1038/4401123a; Received 18 October 2005; Accepted 28 March 2006; Published online 26 April 2006
There is a Brief Communications Arising (21 September 2006) associated with this document.
There is a Brief Communications Arising (21 September 2006) associated with this document.
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Research Fellows in Pluripotent Stem Cell Technology
- The University of Nottingham
- Nottingham, UK
Laboratory Manager / Principal Research Assistant
- Wellcome Trust Sanger Institute
- Hinxton, Cambridge, CB10 1SA, UK
Gene therapy: Therapeutic gene causing lymphoma
Niels-Bjarne Woods1, Virginie Bottero1, Manfred Schmidt2,3, Christof von Kalle2 & Inder M. Verma1
Abstract
Insight into risks posed by corrective gene therapy comes from an immunodeficient mouse model.
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 cells1 has led to a re-evaluation of this approach2. 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 oncogenic3, 4, 5, 6, 7, may therefore pose some risk to patients.
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RESEARCH
Gene therapy Is IL2RG oncogenic in T-cell development?: X-SCID transgene leukaemogenicity (reply)Nature Brief Communication (21 Sep 2006)
Gene therapy X-SCID transgene leukaemogenicityNature Brief Communication (21 Sep 2006)
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