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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.

Gene therapy: Therapeutic gene causing lymphoma

Niels-Bjarne Woods1, Virginie Bottero1, Manfred Schmidt2,3, Christof von Kalle2 & Inder M. Verma1

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Insight into risks posed by corrective gene therapy comes from an immunodeficient mouse model.

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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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