Figure 3
Figure 3. Expression of human FVIII activity in vivo in blood plasma of hemophiliac mice after intravenous injection of SIN LVs expressing bioengineered FVIII constructs. A total of 6 to 10 F8tm2Kaz hemophilic neonatal mice were injected intravenously via the superficial temporal vein with SIN LVs expressing bioengineered human FVIII constructs. Mice were bled at various time points over approximately 250 days, and a chromogenic assay was used to calculate the activity of human FVIII in blood plasma taken from each mouse. (A) SQ FVIII (◇) versus SQ FVIII (co) (▴). (B) SQ FVIII Fugu B (◇) versus SQ FVIII Fugu B (co) (▴). (C) SQ FVIII N6 (◇) versus SQ FVIII N6 (co) (▴). Points on graphs represent the mean; error bars represent SD. Statistical analyses were performed using general linear model based on 2-way analysis of variance with individual pairwise comparisons performed using Bonferroni simultaneous tests (Minitab software).

Expression of human FVIII activity in vivo in blood plasma of hemophiliac mice after intravenous injection of SIN LVs expressing bioengineered FVIII constructs. A total of 6 to 10 F8tm2Kaz hemophilic neonatal mice were injected intravenously via the superficial temporal vein with SIN LVs expressing bioengineered human FVIII constructs. Mice were bled at various time points over approximately 250 days, and a chromogenic assay was used to calculate the activity of human FVIII in blood plasma taken from each mouse. (A) SQ FVIII (◇) versus SQ FVIII (co) (▴). (B) SQ FVIII Fugu B (◇) versus SQ FVIII Fugu B (co) (▴). (C) SQ FVIII N6 (◇) versus SQ FVIII N6 (co) (▴). Points on graphs represent the mean; error bars represent SD. Statistical analyses were performed using general linear model based on 2-way analysis of variance with individual pairwise comparisons performed using Bonferroni simultaneous tests (Minitab software).

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