Fig. 4.
Biochemical correction in NSG-IDUAX/X mice by human IDUA-HSPCs. IDUA activity and GAG accumulation in heterozygous sham-treated (W/X sham- clear), heterozygous transplanted (W/X Tx- black), homozygous sham-treated (X/X sham- blue), and homozygous transplanted (X/X Tx- red) mice. a Percent human and YFP + cells in BM in experiments using bulk and sorted cells. b Urinary GAGs at 4,8, and 18 weeks in experiments using bulk cells (n = 5 mice per cohort, two measurements per mouse). cPlasma and tissue IDUA activity in experiments using bulk cells (n = 5 per cohort). d Fold GAG storage in liver, spleen, and brain (normalized by W/X sham, n = 5 per cohort). e Plasma and tissue IDUA activity in experiments using sorted cells (n = 5 for W/X Tx and sham mice, and n = 13 for X/X Tx and sham mice). f Fold GAG urinary excretion and tissue storage in experiments using sorted cells (normalized by W/X sham). Median values shown in all scatter plots. d, f shows box plots with whiskers at the 5–95th percentiles. ****p < 0.0001 in one-way ANOVA test. Post hoc comparisons were made with the Tukey’s multiple comparisons test. Source data are provided as a Source Data file