Efficacy of AAV-mediated gene transfer of rat FVIIa in HA rats. (A) Average transgene expression (ng/mL, baseline subtracted) after AAV infusion and (B) PT values before and after AAV infusion in HA animals at 4 weeks of age. HA-AAV ≥708 (▪, transgene expression in nanograms per milliliter; n = 15), HA-AAV <708 (●, transgene expression in nanograms per milliliter; n = 18) and untransduced control animals (WT [▼] controls [n = 12]; HA [▲] controls [n = 11]). Arrow indicates time of AAV administration. Thromboelastometry clot time (C) and α angle of recalcified whole blood (NATEM) (D). Each animal included in study had a minimum of 2 samples analyzed throughout the study. Sample sizes for panels C and D: WT controls (n = 9), HA controls (n = 8), HA animals with transgene expression ≥708 ng/mL (n = 12), and HA animals with transgene expression <708 ng/mL (n = 13). ****P < .0001; ***P < .001; **P < .01; *P < .05. Data are presented as mean ± SD. Experimental group comparison with HA control animals: repeated-measures 1-way ANOVA with Tukey’s multiple comparison test (A-B); 1-way ANOVA with Tukey’s multiple comparison test (C-D). NS, not significant.