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 γ-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.
This is a preview of subscription content, access via your institution
Access options
Subscribe to this journal
Receive 51 print issues and online access
$199.00 per year
only $3.90 per issue
Buy this article
- Purchase on Springer Link
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
Similar content being viewed by others
References
Cavazzana-Calvo, M. et al. Science 288, 669–672 (2000).
Aiuti, A. et al. Science 296, 2410–2413 (2002).
Gaspar, H. B. et al. Lancet 364, 2181–2187 (2004).
Hacein-Bey-Abina, S. et al. N. Engl. J. Med. 348, 255–256 (2003).
Woods, N.-B., Bottero, V., Schmidt, M., von Kalle, C. & Verma, I. M. Nature 440, 1123 (2006).
Hacein-Bey-Abina, S. et al. Science 302, 415–419 (2003).
Dave, U. P., Jenkins, N. A. & Copeland, N. G. Science 303, 333 (2004).
Modlich, U. et al. Blood 105, 4235–4246 (2005).
Soudais, C. et al. Blood 95, 3071–3077 (2000).
Otsu, M. et al. Mol. Ther. 1, 145–153 (2000).
Author information
Authors and Affiliations
Rights and permissions
About this article
Cite this article
Thrasher, A., Gaspar, H., Baum, C. et al. X-SCID transgene leukaemogenicity. Nature 443, E5–E6 (2006). https://doi.org/10.1038/nature05219
Published:
Issue Date:
DOI: https://doi.org/10.1038/nature05219
This article is cited by
-
Thymocyte self-renewal and oncogenic risk in immunodeficient mouse models: relevance for human gene therapy clinical trials targeting haematopoietic stem cell populations?
Mammalian Genome (2018)
-
T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency
Genome Medicine (2018)
-
Genome-wide Profiling Reveals Remarkable Parallels Between Insertion Site Selection Properties of the MLV Retrovirus and the piggyBac Transposon in Primary Human CD4+ T Cells
Molecular Therapy (2016)
-
Gene Therapy for SCID
Current Pediatrics Reports (2015)
-
Pigtailed macaques as a model to study long-term safety of lentivirus vector-mediated gene therapy for hemoglobinopathies
Molecular Therapy - Methods & Clinical Development (2014)
Comments
By submitting a comment you agree to abide by our Terms and Community Guidelines. If you find something abusive or that does not comply with our terms or guidelines please flag it as inappropriate.
