Figure 1.
cFVIII expression and anti-cFVIII antibody responses in Chapel Hill hemophilia A dog K01 after liver delivery of AAV-cFVIII. One Chapel Hill dog (K01) with preexisting inhibitors to cFVIII was administered 2.5 × 1013 vg/kg of AAV8-TBG-cFVIII-HC and AAV8-TBG-cFVIII-LC by peripheral venous injection. (A) cFVIII antigen levels were assayed by a cFVIII-LC specific ELISA, and activity was monitored by FVIII assay. (B) Anti-cFVIII antibody responses were measured by anti-cFVIII IgG2 ELISA and Bethesda assays. Black arrows indicate 4 weekly challenges with 500 U of rBDD-cFVIII.