FVII-deficient neonatal mice treated with an AAV8 vector-encoding murine FVII. (A) A schematic of the rAAV-LP1-mFVII construct consisting of the murine FVII cDNA under the control of a liver-specific LP1 promoter/enhancer element, followed by the SV40 virus large T Ag poly-A signal. (B) FVII activity as assessed by a chromogenic assay in plasma of low FVII mice after treatment with 2 × 1013 vg/kg AAV8-LP1-mFVII (broken line, N = 9) compared with control untreated low FVII animals (solid line, N = 3). Vertical arrow indicates virus administration on day 35. (C) Kaplan-Meier survival curve showing that low FVII mice treated with a single tail vein injection of AAV8-LP1-mFVII survived for at least 65 days after administration (100 days postnatal; broken line, N = 19), a significant (P < .0001) improvement over untreated mice (solid line, N = 23).
