Gene therapy: X-SCID transgene leukaemogenicity

Details

Serval ID
serval:BIB_5306D04B7EF3
Type
Article: article from journal or magazin.
Collection
Publications
Title
Gene therapy: X-SCID transgene leukaemogenicity
Journal
Nature
Author(s)
Thrasher A. J., Gaspar H. B., Baum C., Modlich U., Schambach A., Candotti F., Otsu M., Sorrentino B., Scobie L., Cameron E., Blyth K., Neil J., Abina S. H., Cavazzana-Calvo M., Fischer A.
ISSN
1476-4687 (Electronic)
ISSN-L
0028-0836
Publication state
Published
Issued date
2006
Volume
443
Number
7109
Pages
E5-6; discussion E6-7
Language
english
Notes
Thrasher, Adrian J
Gaspar, H Bobby
Baum, Christopher
Modlich, Ute
Schambach, Axel
Candotti, Fabio
Otsu, Makoto
Sorrentino, Brian
Scobie, Linda
Cameron, Ewan
Blyth, Karen
Neil, Jim
Abina, Salima Hacein-Bey
Cavazzana-Calvo, Marina
Fischer, Alain
eng
Comment
England
Nature. 2006 Sep 21;443(7109):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.
Keywords
Animals, *Cell Transformation, Neoplastic, Clinical Trials as Topic/adverse effects, Genetic Therapy/*adverse effects, Humans, Leukemia/*etiology/genetics, Mice, Mice, SCID, Oncogene Proteins/genetics/metabolism, Receptors, Interleukin-2/genetics/*metabolism, Severe Combined Immunodeficiency/complications/*genetics/*therapy, Transgenes/*genetics
Pubmed
Create date
01/11/2017 10:29
Last modification date
20/08/2019 14:08
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