Figure 1.

Hepatic AAV8 gene transfer of codon-optimized BDD-FVIII (coFVIII) to C57BL/6 mice. (a) Systemic FVIII antigen levels as a function of time after gene transfer of 1 × 10¹⁰ vg (n = 5), 1 × 10¹¹ vg (n = 4–8), or 1 × 10¹² vg (n = 4–8) to C57BL/6 mice or to hemophilia A mice (129/BL6-F8^−/Y, labeled with “HA” in caption). (b) Anti-FVIII IgG1 levels at 4 weeks post gene transfer in C57BL/6 mice treated with the indicated vectors. A previously identified anti-FVIII IgG1 positive plasma sample from a FVIII immunized 129/BL6-F8^−/Y mouse was included as a control. (c) Liver enzyme levels (ALT, alanine transaminase) in plasma 4 weeks after hepatic gene transfer to C57BL/6 mice. For comparison, data from AAV-F9 transduced and naive mice are shown. Data are mean ± SD. AAV, adeno-associated virus; BDD, B domain deleted; FVIII, functional factor VIII; SD, standard deviation.