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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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| Publicado no: | J Exp Med |
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| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Rockefeller University Press
2019
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| Assuntos: | |
| Acesso em linha: | 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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