MyeloVec restores gp91phox expression in human X-CGD BM cells engrafted into NSG mice. Human X-CGD CD34⁺ HSPCs were engrafted into NSG mice. Mice were harvested 16 weeks after transplant and stable BM VCN (A) and engraftment of human cells (B) were analyzed. (C) Restoration of gp91phox was measured across the different hematopoietic lineages in the BM. Data from each mouse are overlaid in the histograms. (D) The MFI of gp91^phox-positive cells is shown. Data are presented as mean ± SD. Statistical significance was analyzed using a 2-way ANOVA followed by multiple paired comparisons for normally distributed data (Tukey test). All statistical tests were 2-tailed and P < .05 was deemed significant; ∗P < .05, ∗∗P < .01, ∗∗∗P < .001, ∗∗∗∗P < .0001.