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Brief Communications Arising
Nature 443, E5-E6 (21 September 2006) | doi:10.1038/nature05219; Published online 20 September 2006
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Gene therapy: X-SCID transgene leukaemogenicity
Adrian J. Thrasher1,2, H. Bobby Gaspar1,2, Christopher Baum3,4, Ute Modlich3, Axel Schambach3, Fabio Candotti5, Makoto Otsu6, Brian Sorrentino7, Linda Scobie8, Ewan Cameron8, Karen Blyth8, Jim Neil8, Salima Hacein-Bey Abina9,10, Marina Cavazzana-Calvo9,10,11 & Alain Fischer9,11,12
Abstract
Arising from: Woods, N.-B., Bottero, V., Schmidt, M., von Kalle, C. & Verma, I. M. Nature 440, 1123 (2006); see also communication from Pike-Overzet et al.; Woods et al. reply
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.
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