Gene therapy: X-SCID transgene leukaemogenicity

Thrasher, A.J. et al. (2006) Gene therapy: X-SCID transgene leukaemogenicity. Nature, 443(7109), E5-E6. (doi: 10.1038/nature05219)

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Gene therapy has been remarkably effective for the immunological reconstitution of patients with severe combined immune deficiency1, 2, 3, but the occurrence of leukaemia in a few patients has stimulated debate about the safety of the procedure and the mechanisms of leukaemogenesis4. Woods et al.5 forced high expression of the corrective therapeutic gene IL2RG, which encodes the -chain of the interleukin-2 receptor, in a mouse model of the disease and found that tumours appeared in a proportion of cases. Here we show that transgenic IL2RG does not necessarily have potent intrinsic oncogenic properties, and argue that the interpretation of this observation with respect to human trials is overstated.

Item Type:Articles
Glasgow Author(s) Enlighten ID:Blyth, Professor Karen and Cameron, Professor Ewan and Scobie, Dr Linda and Neil, Professor James
Authors: Thrasher, A.J., Gaspar, H. B., Baum, C., Modlich, U., Schambach, A., Candotti, F., Otsu, M., Sorrentino, B., Scobie, L., Cameron, E.R., Blyth, K., Neil, J.C., Abina, S.H.-B., Cavazzana-Calvo, M., and Fischer, A.
College/School:College of Medical Veterinary and Life Sciences > School of Cancer Sciences
College of Medical Veterinary and Life Sciences > School of Infection & Immunity
College of Medical Veterinary and Life Sciences > School of Biodiversity, One Health & Veterinary Medicine
Journal Name:Nature

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