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Targeted gene addition in human CD34(+) hematopoietic cells for correction of X-linked chronic granulomatous disease.
Nat Biotechnol. 2016 Apr;34(4):424-9. doi: 10.1038/nbt.3513. Epub 2016 Mar 7.
Nat Biotechnol. 2016.
PMID: 26950749
Free PMC article.
An AAVS1-targeted minigene platform for correction of iPSCs from all five types of chronic granulomatous disease.
Merling RK, Sweeney CL, Chu J, Bodansky A, Choi U, Priel DL, Kuhns DB, Wang H, Vasilevsky S, De Ravin SS, Winkler T, Dunbar CE, Zou J, Zarember KA, Gallin JI, Holland SM, Malech HL.
Merling RK, et al.
Mol Ther. 2015 Jan;23(1):147-57. doi: 10.1038/mt.2014.195. Epub 2014 Oct 7.
Mol Ther. 2015.
PMID: 25288370
Free PMC article.
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