Influence of sex on rAAV-mediated hFIX expression. (A) Schematic of rAAV-2 CAGG-FIX consisting of CMV-IE enhancer, β-actin promoter, a chicken β-actin/rabbit β-globin composite intron (CAGG), 1.6-kb human FIX cDNA (hFIX), and a rabbit β-globin polyadenylation signal (hatched box) flanked by the AAV internal terminal repeats (ITR shown as hairpin loop). (B) Human FIX concentration (hFIX) in murine plasma after liver-directed administration of 1 × 10¹¹ rAAV CAGG-FIX in cohorts (n = 6) of male (▪) and female (▵) B6.CB17-Prkdc-SzJ mice. All transgene expression values are depicted as average together with the standard error of the mean.