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Therapeutic gene causing lymphoma

2006; Nature Portfolio; Volume: 440; Issue: 7088 Linguagem: Inglês

10.1038/4401123a

1476-4687

Niels-Bjarne Woods, Virginie Bottero, Manfred Schmidt, Christof von Kalle, Inder M. Verma,

Viral gastroenteritis research and epidemiology

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.