In vivo expression of rAAV/BDD-hFVIII in mice.

Purified rAAV/DLZ6 virus was administered to the C57BL/6 mice via the portal vein. ELISA was used to determine hFVIII level in the plasma and BIA was used to measure anti-hFVIII inhibitor titer. A) BDD hFVIII antigen level and anti-hFVIII inhibitor titer in the plasma of the mice (n = 4) receiving 2 × 1011 rAAV/DLZ6. B) BDD-hFVIII antigen measurement of NOD/scid mice (n = 4) receiving 1.5 × 1011 rAAV/DLZ6. Solid line: hFVIII antigen level; dashed line: anti-BDD-hFVIII inhibitor titer. Human FVIII ELISA standard curve was performed using pooled normal human plasma diluted in either barbital buffered saline or mouse plasma.