Figure 6.

HC_HL improved the phenotypic hemophilia correction in vivo. CD4KOHA mice were injected with 5 × 10¹¹ vg/mouse AAV8-HC_HL+AAV8-LC vector (n = 5, open square) or AAV8-HC⁷⁴⁵+AAV8-LC (n = 5, close diamond) at a ratio of 4:1. (a) The expression of FVIII heavy chain (y-axis) in mouse plasma was measured by FVIII heavy-chain-specific ELISA biweekly (x-axis). (b) Functional FVIII was then assayed and converted from aPTT (y-axis). Error bars indicate standard error. aPTT, activated partial thromboplastin time; FVIII, factor VIII; HC, heavy chain; LC, light chain.