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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Bibliografski detalji
Izdano u:Exp Hematol
Glavni autori: Flynn, Rowan, Grundmann, Alexander, Renz, Peter, Hänseler, Walther, James, William S., Cowley, Sally A., Moore, Michael D.
Format: Artigo
Jezik:Inglês
Izdano: Elsevier Science Inc 2015
Teme:
Gene Therapy
Online pristup: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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