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

Por De Ravin, Suk See, Reik, Andreas, Liu, Pei-Qi, Li, Linhong, Wu, Xiaolin, Su, Ling, Raley, Castle, Theobald, Narda, Choi, Uimook, Song, Alexander H., Chan, Andy, Pearl, Jocelynn R., Paschon, David E., Lee, Janet, Newcombe, Hannah, Koontz, Sherry, Sweeney, Colin, Shivak, David A., Zarember, Kol A., Peshwa, Madhusudan V., Gregory, Philip D., Urnov, Fyodor D., Malech, Harry L.
Publicado no Nat Biotechnol (2016)