Critical Variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency

Patients with X-linked severe combined immunodeficiency (SCID-X1) were successfully cured following gene therapy with a gamma-retroviral vector (gRV) expressing the common gamma chain of the interleukin-2 receptor (IL2RG). However, 5 of 20 patients developed leukemia from activation of cellular prot...

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Detalhes bibliográficos
Main Authors: van der Loo, JCM, Swaney, WP, Grassman, E, Terwilliger, A, Higashimoto, T, Schambach, A, Hacein-Bey-Abina, S, Nordling, DL, Cavazzana-Calvo, M, Thrasher, AJ, Williams, DA, Reeves, L, Malik, P
Formato: Artigo
Idioma:Inglês
Publicado em: 2012
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4174358/
https://ncbi.nlm.nih.gov/pubmed/22551777
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2012.37
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