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

Gene therapy with genetically modified human CD34(+) hematopoietic stem and progenitor cells (HSPCs) may be safer using targeted integration (TI) of transgenes into a genomic 'safe harbor' site rather than random viral integration. We demonstrate that temporally optimized delivery of zinc finger nuclease mRNA via electroporation and adeno-associated virus (AAV) 6 delivery of donor constructs in human HSPCs approaches clinically relevant levels of TI into the AAVS1 safe harbor locus. Up to 58% Venus(+) HSPCs with 6-16% human cell marking were observed following engraftment into mice. In HSPCs from patients with X-linked chronic granulomatous disease (X-CGD), caused by mutations in the gp91phox subunit of the NADPH oxidase, TI of a gp91phox transgene into AAVS1 resulted in ~15% gp91phox expression and increased NADPH oxidase activity in ex vivo-derived neutrophils. In mice transplanted with corrected HSPCs, 4-11% of human cells in the bone marrow expressed gp91phox. This method for TI into AAVS1 may be broadly applicable to correction of other monogenic diseases.

Journal: Nature biotechnology
DOI: 10.1038/nbt.3513
Year: 2016

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