Figure 2.

Dynamic aPTT Improvement after an AAV8/hFVIII Re-administration

The hemophilia dogs U10 and U11 were treated with the dose of 4 × 10¹² particles/kg of AAV8/hFVIII, with bortezomib, at a dose of 1.3 mg/m² via the peripheral vein. At the indicated time points after re-administration, aPTT analysis from citrated plasma was performed. (A) FVIII-specific aPTT. FACT, normal human plasma; NCP, normal canine plasma; X, U10 died at day 120. (B) Whole-blood clotting time (WBCT). Normal range of 6–10 min in hemostatically normal dogs is indicated in the box region as normal. Hemophilic dogs had baseline WBCT > 20 min. (C) FVIII antigen protein level in blood at different time points up to day 64 post-AAV8/hFVIII re-administration was measured by hFVIII-specific ELISA.