Nature. 2006 Apr 27;440(7088):1123.
Gene therapy: therapeutic gene causing lymphoma.
Woods NB, Bottero V, Schmidt M, von Kalle C, Verma IM.
3 children who underwent gene therapy for X-linked Severe Combined Immunodeficiency (X-SCID),using therapeutic administration of the IL2RG gene, developped T-cell leukaemia.
In this article, the investigators conducted long term studies in a murine moodel of X-SCID using a similar treatment. One third of the mice developped T-cell lymphomas. This implicates IL2RG in the lymphomagenesis in this model. It underscores the requirement for long-term studies in animals before conducting human gene therapy trials.
For more information on the treatement of genetic disease and on X-SCID, see chapters 5 and 185 of OMMBID, respectively.
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Philippe Campeau, MD
Resident in Medical Genetics at McGill University
OMMBID Blog Administrator