FIGURE 2.
FVa was functional in hemophilia mice after hydrodynamic injection of hFVa plasmids. (A) Constructs of FVa “hFV BDD”: complete deletion of B domain; “hFVa”: complete deletion of the B domain and a furin cleavage site linker between the FV heavy chain (HC) and the light chain (LC); “hFVBD SQ”: complete deletion of the B domain with an SQ sequence inserted between the HC and the LC. (B) aPTT change after hydrodynamic injection. In both FIX– /– and FVIII– /– mice, the three plasmid constructs were tested, and FVIII/FIX-specific aPTT was measured 48 h after hydrodynamic injection. FIX– /–/FVIII– /– mice (“FIX– /–”/“FVIII– /–”and their “WT”) controls injected with normal saline were used as the controls.