Figure 1.

In vitro production of wildtype and codon-optimized factor VIII. (A, B) DNA in lipofectamine (0.1 μg) (A) or mRNA formulated in TransIT^® (0.4 μg) (B) encoding B domain-deleted (BDD) factor VIII (FVIII^HSQ) and codon-optimized BDD-FVIII (CoFVIII^HSQ) were used to transfect BHK (A) or HEK293 (B) cells. mRNA encoding luciferase was used as a control (B). FVIII:Ag (left panels) and FVIII:C (middle panels) were measured in cell supernatant 24 h after transfection. The right panels show the specific activities as calculated by dividing the FVIII:C values by the FVIII:Ag values. Statistical differences were assessed using a two-tailed t test (ns: non-significant, *P<0.05, **P<0.01, ***P<0.001). Results are presented as the mean ± standard error of mean of three independent experiments.