Fig. 3.

IDUA-HSPCs maintain long-term repopulation capacity. a Schematic and representative FACS plots showing phenotyping by flow of human, myeloid, B-cell, and targeted cells after engraftment. b Percent human cell chimerism in bone marrow (BM) and peripheral blood (PM) of mice 16-weeks post-transplant with CB (blue dots) and PB (red dots)-derived HSPCs targeted with PGK-IDUA-YFP cassette. Each point represents an individual mouse; mock (n = 11), YFP- (n = 21), and YFP + (n = 36). c Percent human, YFP + cells in BM of mice in BM 16-weeks post-transplant. d Percent human cell chimerism in BM in mice transplanted with bulk cells without selection with two different human cell donors; donor 1 n = 9, donor 2 n = 5 e Percent modified alleles in engrafted cells by ddPCR. 28% was the starting allele modification frequency for both human donors. f Percent human cell chimerism in BM of mice in secondary transplants 32 weeks after genome editing; YFP- (n = 10), and YFP+ (n = 10). e Percent human, YFP+ cells in BM of mice in secondary transplants