Figure 7.

AAV-FVIII gene therapy in the presence of low-titer FVIII inhibitors. (a) Anti-ET3 IgG titers are represented as the percent of the initial titer determined by ELISA in mice with low initial anti-ET3 Bethesda titers (0–25 BU). Naive hemophilia A mice were preimmunized with ET3 and treated with 4 × 10¹² vp/kg of AAV8-HLP-ET3 vector (n = 10). (b) Individual anti-ET3 IgG titers measured by ELISA in mice with low initial anti-ET3 Bethesda titers. Mice were preimmunized with ET3 and treated with 4 × 10¹² vp/kg of AAV8-HLP-ET3 vector. AAV, adeno-associated virus; HLP, hybrid liver-specific promoter.