X-SCID transgene leukaemogenicity (original) (raw)
- Brief Communications Arising
- Published: 20 September 2006
Gene therapy
- 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
Nature volume 443, pages E5–E6 (2006)Cite this article
- 1831 Accesses
- 116 Citations
- 6 Altmetric
- Metrics details
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 SpringerLink
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
Additional access options:
Similar content being viewed by others
References
- Cavazzana-Calvo, M. et al. Science 288, 669–672 (2000).
Article ADS CAS Google Scholar - Aiuti, A. et al. Science 296, 2410–2413 (2002).
Article ADS CAS Google Scholar - Gaspar, H. B. et al. Lancet 364, 2181–2187 (2004).
Article CAS Google Scholar - Hacein-Bey-Abina, S. et al. N. Engl. J. Med. 348, 255–256 (2003).
Article Google Scholar - Woods, N.-B., Bottero, V., Schmidt, M., von Kalle, C. & Verma, I. M. Nature 440, 1123 (2006).
Article ADS CAS Google Scholar - Hacein-Bey-Abina, S. et al. Science 302, 415–419 (2003).
Article ADS CAS Google Scholar - Dave, U. P., Jenkins, N. A. & Copeland, N. G. Science 303, 333 (2004).
Article Google Scholar - Modlich, U. et al. Blood 105, 4235–4246 (2005).
Article CAS Google Scholar - Soudais, C. et al. Blood 95, 3071–3077 (2000).
CAS PubMed Google Scholar - Otsu, M. et al. Mol. Ther. 1, 145–153 (2000).
Article CAS Google Scholar
Author information
Authors and Affiliations
- Molecular Immunology Unit, Institute of Child Health, University College London, London, WC1N 1EH, UK
Adrian J. Thrasher & H. Bobby Gaspar - Great Ormond Street Hospital for Children NHS Trust, London, WC1N 3JH, UK
Adrian J. Thrasher & H. Bobby Gaspar - Department of Experimental Hematology, Hannover Medical School, Hannover, 30625, Germany
Christopher Baum, Ute Modlich & Axel Schambach - Division of Experimental Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, 45229-3039, Ohio, USA
Christopher Baum - Genetics and Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, 20892, Maryland, USA
Fabio Candotti - Center for Experimental Medicine, University of Tokyo, Tokyo, 108-8639, Japan
Makoto Otsu - Department of Hematology/Oncology, Division of Experimental Hematology, St Jude Children's Research Hospital, Memphis, 38105, Tennessee, USA
Brian Sorrentino - Division of Pathological Sciences, Institute of Comparative Medicine, Faculty of Veterinary Medicine, University of Glasgow, Glasgow, G61 1QH, UK
Linda Scobie, Ewan Cameron, Karen Blyth & Jim Neil - INSERM Unit 768, Hôpital Necker, Assistance Publique–Hôpitaux de Paris, Paris, 75743, France
Salima Hacein-Bey Abina, Marina Cavazzana-Calvo & Alain Fischer - Département de Biothérapies, Assistance Publique–Hôpitaux de Paris, Paris, 75743, France
Salima Hacein-Bey Abina & Marina Cavazzana-Calvo - Faculté de Médecine, Université René Descartes, Paris, 75270, France
Marina Cavazzana-Calvo & Alain Fischer - Unité d'Immuno-Hématologie Pédiatrique, Assistance Publique–Hôpitaux de Paris, Paris, 75743, France
Alain Fischer
Authors
- Adrian J. Thrasher
You can also search for this author inPubMed Google Scholar - H. Bobby Gaspar
You can also search for this author inPubMed Google Scholar - Christopher Baum
You can also search for this author inPubMed Google Scholar - Ute Modlich
You can also search for this author inPubMed Google Scholar - Axel Schambach
You can also search for this author inPubMed Google Scholar - Fabio Candotti
You can also search for this author inPubMed Google Scholar - Makoto Otsu
You can also search for this author inPubMed Google Scholar - Brian Sorrentino
You can also search for this author inPubMed Google Scholar - Linda Scobie
You can also search for this author inPubMed Google Scholar - Ewan Cameron
You can also search for this author inPubMed Google Scholar - Karen Blyth
You can also search for this author inPubMed Google Scholar - Jim Neil
You can also search for this author inPubMed Google Scholar - Salima Hacein-Bey Abina
You can also search for this author inPubMed Google Scholar - Marina Cavazzana-Calvo
You can also search for this author inPubMed Google Scholar - Alain Fischer
You can also search for this author inPubMed Google Scholar
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: 20 September 2006
- Issue Date: 21 September 2006
- DOI: https://doi.org/10.1038/nature05219