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Gene therapy for severe combined immunodeficiencies and beyond

Ex vivo retrovirally mediated gene therapy has been shown within the last 20 yr to correct the T cell immunodeficiency caused by γc-deficiency (SCID X1) and adenosine deaminase (ADA) deficiency. The rationale was brought up by the observation of the revertant of SCIDX1 and ADA deficiency as a kind o...

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Publicat a:J Exp Med
Autors principals: Fischer, Alain, Hacein-Bey-Abina, Salima
Format: Artigo
Idioma:Inglês
Publicat: Rockefeller University Press 2019
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7041706/
https://ncbi.nlm.nih.gov/pubmed/31826240
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1084/jem.20190607
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