Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

BACKGROUND: The outcomes of gene therapy to correct congenital immunodeficiencies are unknown. We reviewed long-term outcomes after gene therapy in nine patients with X-linked severe combined immunodeficiency (SCID-X1), which is characterized by the absence of the cytokine receptor common γ chain. M...

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Detalhes bibliográficos
Main Authors: Hacein-Bey-Abina, Salima, Hauer, Julia, Lim, Annick, Picard, Capucine, Wang, Gary P., Berry, Charles C., Martinache, Chantal, Rieux-Laucat, Frédéric, Latour, Sylvain, Belohradsky, Bernd H., Leiva, Lily, Sorensen, Ricardo, Debré, Marianne, Casanova, Jean Laurent, Blanche, Stephane, Durandy, Anne, Bushman, Frederic D., Fischer, Alain, Cavazzana-Calvo, Marina
Formato: Artigo
Idioma:Inglês
Publicado em: 2010
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2957288/
https://ncbi.nlm.nih.gov/pubmed/20660403
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa1000164
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