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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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Detalles Bibliográficos
Autores principales:	Flynn, Rowan, Grundmann, Alexander, Renz, Peter, Hänseler, Walther, James, William S., Cowley, Sally A., Moore, Michael D.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier Science Inc 2015
Materias:	Gene Therapy
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4596252/ https://www.ncbi.nlm.nih.gov/pubmed/26101162 http://dx.doi.org/10.1016/j.exphem.2015.06.002

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