An AAVS1-Targeted Minigene Platform for Correction of iPSCs From All Five Types of Chronic Granulomatous Disease

There are five genetic forms of chronic granulomatous disease (CGD), resulting from mutations in any of five subunits of phagocyte oxidase, an enzyme complex in neutrophils, monocytes, and macrophages that produces microbicidal reactive oxygen species. We generated induced pluripotent stem cells (iP...

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Bibliografiska uppgifter
I publikationen:Mol Ther
Huvudupphovsmän: Merling, Randall K, Sweeney, Colin L, Chu, Jessica, Bodansky, Aaron, Choi, Uimook, Priel, Debra Long, Kuhns, Douglas B, Wang, Hongmei, Vasilevsky, Sam, De Ravin, Suk See, Winkler, Thomas, Dunbar, Cynthia E, Zou, Jizhong, Zarember, Kol A, Gallin, John I, Holland, Steven M, Malech, Harry L
Materialtyp: Artigo
Språk:Inglês
Publicerad: Nature Publishing Group 2015
Ämnen:
Original Article
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC4426805/
https://ncbi.nlm.nih.gov/pubmed/25288370
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2014.195
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