CRISPR-mediated genotypic and phenotypic correction of a chronic granulomatous disease mutation in human iPS cells

Chronic granulomatous disease (CGD) is a rare genetic disease characterized by severe and persistent childhood infections. It is caused by the lack of an antipathogen oxidative burst, normally performed by phagocytic cells to contain and clear bacterial and fungal growth. Restoration of immune funct...

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Detalhes bibliográficos
Publicado no:Exp Hematol
Main Authors: Flynn, Rowan, Grundmann, Alexander, Renz, Peter, Hänseler, Walther, James, William S., Cowley, Sally A., Moore, Michael D.
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier Science Inc 2015
Assuntos:
Gene Therapy
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4596252/
https://ncbi.nlm.nih.gov/pubmed/26101162
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.exphem.2015.06.002
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