Targeted Gene Addition to a Safe Harbor locus in human CD34+ Hematopoietic Stem Cells for Correction of X-linked Chronic Granulomatous Disease

Targeted Gene Addition to a Safe Harbor locus in human CD34+ Hematopoietic Stem Cells for Correction of X-linked Chronic Granulomatous Disease

Suk See De Ravin, Andreas Reik, Pei-Qi Liu, Linhong Li, Xiaolin Wu, Ling Su, Castle Raley, Narda Theobald, Uimook Choi, Alexander H. Song, Andy Chan, Jocelynn R. Pearl, David E. Paschon, Janet Lee, Hannah Newcombe, Sherry Koontz, Colin Sweeney, David A. Shivak, Kol A. Zarember, Madhusudan V. Peshwa, Philip D. Gregory, Fyodor D. Urnov, Harry L. Malech

Nat Biotechnol. Author manuscript; available in PMC 2016 Sep 7.

Published in final edited form as: Nat Biotechnol. 2016 Apr; 34(4): 424–429. Published online 2016 Mar 7. doi: 10.1038/nbt.3513

PMCID: PMC4824656