MyeloVec gene therapy protects X-CGD mice from B cepacia infection. (A) Murine X-CGD Lin− HSPCs were transduced with MyeloVec or pCCLchim at an equal vector dose of 2e7 TU/mL and transplanted into X-CGD mice. WT C57BL/6 HSPCs and nontransduced X-CGD HSPCs were also transplanted as positive and negative controls, respectively. Nontransplanted WT C57BL/6 mice were also included in the study as a positive control. Stable VCN (B) and percentage of DHR+ neutrophils (C) in the PB of the transplanted mice were measured 8 weeks after transplant. (D) Bacteremia was quantified 24 hours after infection as CFU per μL in the PB. (E) Proportion of surviving mice 14 days post experimental infection with 1 × 105B cepacia is shown. Data are presented as mean ± SD. Statistical significance for panels B-D was analyzed using an unpaired t test. All statistical tests were 2-tailed and P < .05 was deemed significant; ∗P < .05, ∗∗P < .01, ∗∗∗P < .001, ∗∗∗∗P < .0001. i.p., intraperitoneally.
