Gene therapy: X-SCID transgene leukaemogenicity

Détails

ID Serval
serval:BIB_5306D04B7EF3
Type
Article: article d'un périodique ou d'un magazine.
Collection
Publications
Titre
Gene therapy: X-SCID transgene leukaemogenicity
Périodique
Nature
Auteur⸱e⸱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
Statut éditorial
Publié
Date de publication
2006
Volume
443
Numéro
7109
Pages
E5-6; discussion E6-7
Langue
anglais
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.
Résumé
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.
Mots-clé
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
Création de la notice
01/11/2017 11:29
Dernière modification de la notice
20/08/2019 15:08
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