Efficiency of liver transduction with exo-AAV vectors in vivo. (A-B) Naive male and female C57BL/6 mice (n = 5) treated with exo- or standard AAV8-hF.IX vectors at 5 × 1010 vg per mouse. (A) Plasma hF.IX levels measured by enzyme-linked immunosorbent assay (ELISA). (B) Vector genome copy number (VGCN) per diploid genome at sacrifice (day 42) in liver and other tissues. (C-D) Naive male and female C57BL/6 mice treated with exo- or standard AAV5-hF.IX vectors at 1 × 109 vg per mouse. (A) Plasma hF.IX levels measured by ELISA. (B) VGCN per diploid genome at sacrifice (day 42) in liver and other tissues. Data are shown as mean ± SEM. For hF.IX plasma levels, a 2-way ANOVA with Bonferroni posttest was used to determine statistical significance; for VGCN, an unpaired Student t test was used. *P < .05; **P < .01; ***P < .001.
