Article
An AAVS1-targeted minigene platform for correction of iPSCs from all five types of chronic granulomatous disease.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Jan 2015
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
Abstract excerpt
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 (iPSCs) from peripheral blood CD34(+) hematopoietic stem cells of patients with each of five CGD genotypes. We used...
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