Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair

Colin L. Sweeney(National Institutes of Health), Mara Pavel-Dinu(Stanford University), Uimook Choi(National Institutes of Health), Julie Brault(National Institutes of Health), Taylor Liu(National Institutes of Health), Sherry Koontz(National Institutes of Health), Linhong Li(MaxCyte (United States)), Narda Theobald(National Institutes of Health), Janet S. Lee(National Institutes of Health), Ezekiel Bello(National Institutes of Health), Xiaolin Wu(Leidos (United States)), Ronald J. Meis, Gary A. Dahl, Matthew H. Porteus(Stanford Medicine), Harry L. Malech(National Institutes of Health), Suk See De Ravin(National Institutes of Health)
Gene Therapy
March 12, 2021
Cited by 52Open Access
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