Gene therapy: therapeutic gene causing lymphoma.

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

doi:10.1038/4401123a

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Gene therapy: therapeutic gene causing lymphoma.

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

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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.

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Animals Cell Transformation, Neoplastic Clinical Trials as Topic Disease Models, Animal Dogs Genetic Therapy Hematopoietic Stem Cell Transplantation Humans Lymphoma, T-Cell Mice Mice, SCID Oncogenes Receptors, Interleukin-2 Severe Combined Immunodeficiency Time Factors Journal Article

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Niels-Bjarne Woods, Virginie Bottero, Manfred Schmidt, Christof von Kalle, Inder M Verma. 2006-04-27. Gene therapy: therapeutic gene causing lymphoma.. https://doi.org/10.1038/4401123a

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