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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Publisher's URL: http://dx.doi.org/10.1038/nature05219

Abstract

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
Status:Published
Refereed:Yes
Glasgow Author(s) Enlighten ID:Blyth, Professor Karen and Cameron, Professor Ewan and Neil, Professor James and Scobie, Dr Linda
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 > Institute of Cancer Sciences
College of Medical Veterinary and Life Sciences > Institute of Infection Immunity and Inflammation
College of Medical Veterinary and Life Sciences > School of Veterinary Medicine
Journal Name:Nature
ISSN:0028-0836

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