A group of 20 age-matched, normal C57BL/6 mice and groups of 10-12 exon 16–(Ex 16 KO) or exon 17–disrupted (Ex 17 KO) hemophiliac mice were treated with the indicated vectors (6 × 10¹⁰particles per mouse) or were untreated. FVIII plasma levels were measured before vector administration and 5 days after treatment. All mice treated with Av3H8101 showed expression of functional FVIII at levels well above therapeutic. On day 6, a 2-cm section of the mouse tail was clipped and the mouse survival rate recorded 24 hours later.

One mouse died before tail clipping.

Two mice died before tail clipping.