Gene Therapy: X-scid Transgene Leukaemogenicity.
From: Molecular Immunology Unit, Institute of Child Health, University College London, London WC1N 1EH, UK. a.thrasher@ich.ucl.ac.uk
Nature
- Publish Date: Sep 2006
- ISSN: 1476-4687
- Volume: 443
- Issue: 7109
- Pages: E5-6; discussion E6-7
- Medium: Internet
- Language: English
- Citation (JAMA): Thrasher Adrian J, Gaspar H Bobby, Baum Christopher, et al. Gene Therapy: X-scid Transgene Leukaemogenicity.. Nature Sep 2006;443:E5-6; discussion E6-7
Abstract
Gene therapy has been remarkably effective for the immunological reconstitution of patients with severe combined immune deficiency, but the occurrence of leukaemia in a few patients has stimulated debate about the safety of the procedure and the mechanisms of leukaemogenesis. Woods et al. forced high expression of the corrective therapeutic gene IL2RG, which encodes the gamma-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.
Mesh Headings (Keywords): Animals, Cell Transformation, Neoplastic, Clinical Trials as Topic, Gene Therapy, Humans, Leukemia, Mice, Mice, SCID, Oncogene Proteins, Receptors, Interleukin-2, Severe Combined Immunodeficiency, Transgenes
Check for Full Text / PubMed Unique Identifier (PMID): 16988659
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