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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Dades bibliogràfiques
Publicat a:Exp Hematol
Autors principals: Flynn, Rowan, Grundmann, Alexander, Renz, Peter, Hänseler, Walther, James, William S., Cowley, Sally A., Moore, Michael D.
Format: Artigo
Idioma:Inglês
Publicat: Elsevier Science Inc 2015
Matèries:
Gene Therapy
Accés en línia: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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