Functional Restoration of gp91phox-Oxidase Activity by BAC Transgenesis and Gene Targeting in X-linked Chronic Granulomatous Disease iPSCs

Chronic granulomatous disease (CGD) is an inherited immunodeficiency, caused by the inability of neutrophils to produce functional NADPH oxidase required for fighting microbial infections. The X-linked form of CGD (X-CGD), which is due to mutations in the CYBB (gp91phox) gene, a component of NADPH o...

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Veröffentlicht in:Mol Ther
Hauptverfasser: Laugsch, Magdalena, Rostovskaya, Maria, Velychko, Sergiy, Richter, Cornelia, Zimmer, Ariane, Klink, Barbara, Schröck, Evelin, Haase, Michael, Neumann, Katrin, Thieme, Sebastian, Roesler, Joachim, Brenner, Sebastian, Anastassiadis, Konstantinos
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2016
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Original Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4886927/
https://ncbi.nlm.nih.gov/pubmed/26316390
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.154
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