Figure 5.
Treatment of hemophilia B mice with exo- and standard AAV8 vectors. Male C57BL/6 F9−/− mice were injected IV with 109 vg standard (n = 3) or exo- (n = 4) AAV8-hF.IX vectors (n = 4). (A) Plasma hF.IX expression levels measured by ELISA. (B) hF.IX activity measured by aPTT assay. (C) VGCN per diploid genome measured in liver at sacrifice. (D) Anti-AAV8 IgG antibodies determined by ELISA. (E) Anti-AAV8 NAb titer. (F) Anti-hF.IX IgG antibodies determined by ELISA. hF.IX/CFA, positive control mice injected subcutaneously with 100 μg of hF.IX protein in complete Freund adjuvant (n = 5). All data shown are at 30 days posttreatment. Shown are mean ± SEM. Statistical analysis was performed using the unpaired Student t test. *P < .05; ***P < .001.
