MyeloVec corrects the X-CGD mouse model in vivo. (A) Proviral map of MyeloVec. (B) Murine CD45.2 X-CGD Lin⁻ HSPCs were transduced at an equal vector dose of 2e7 TU/mL and transplanted into congenic CD45.1 (Pepboy) mice. WT C57BL/6 HSPCs and nontransduced X-CGD HSPCs were also transplanted as positive and negative controls, respectively. VCN of the cell product are shown in the bottom panel. Mice were harvested 16 weeks after transplant and engraftment (C) and stable VCN (D) in the BM were measured. Restoration of gp91^phox (E-F) and oxidase activity (G-H) was evaluated across the different lineages in the PB of the mice at time of harvest. Each mouse is represented as a different colored line in the histograms in panels E and G. MFI of gp91^phox and rhodamine 123 of all cells within each lineage are shown. Data are presented as mean ± SD. Statistical significance for panel D was analyzed using an unpaired t test, whereas panels F and H used 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. bp, base pair; NT, nontransduced.