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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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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2015
Assuntos:
Acesso em linha: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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