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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Bibliografische gegevens
Gepubliceerd in:Exp Hematol
Hoofdauteurs: Flynn, Rowan, Grundmann, Alexander, Renz, Peter, Hänseler, Walther, James, William S., Cowley, Sally A., Moore, Michael D.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Elsevier Science Inc 2015
Onderwerpen:
Gene Therapy
Online toegang: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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