Secondary transplant of HSPCs from HemA mice IO-treated with G-F8-LVs maintains phenotypical correction. HemA mice were treated with IO infusion of G-F8-LV (1.5 × 109 ifu/mL). Four weeks after treatment, total BM cells were isolated from LV-treated mice and transplanted into recipient HemA mice (1 × 106 BM cells per mouse). One month after the secondary transplant, we performed the ROTEM assay. (A) Schematic of self-inactivating LV genome encoding FVIII under the platelet-specific promoter, GpIbα (top panel), and the schedule of the secondary transplant experiments (bottom panel). (B) Representative ROTEM assays were shown for an untreated naive HemA mouse (HemA), a wild-type C57BL/6 mouse control (WT), a G-F8-LV–treated donor mouse (IO-LV), and a secondary BM transplant recipient mouse (BMT). (C) Summary of ROTEM assay results. The data are presented as means with standard deviation from 2 separate experiments. ∗P < .05, ∗∗P < .01, ∗∗∗P < .001, and ∗∗∗∗P < .0001.
