Figure 5
Figure 5. Hemostatic efficacy of mFVIIa-VEAY after high-dose gene transfer in hemophilia A mice. (A) aPTT in HA mice after delivery of AAV-mFVIIa-VEAY (1.2 × 1012 vg/mouse). The initial cohort consisted of 10 mice. The gray box indicates the range of aPTT values using human FVIII-deficient plasma in hemostatically normal mice (37.9 ± 1.6 seconds, n = 10). (B) Blood loss after a tail clip assay, compared with AAV-mFVIIa–treated HA mice, or untreated HA and wild-type (WT) mice, whose data are depicted in Figure 3C and are repeated here for comparison. *P < .02 vs HA; **P < .002 vs WT. (C) TAT levels in AAV-mFVIIa-VEAY–treated mice as a function of time. *P < .03 vs baseline (Pre). (D) Survival of HA mice treated with high-dose AAV-mFVIIa-VEAY (AAV-mFVIIa-VEAY [H], ●), relative to untreated HA controls (□). All cohorts initially consisted of 10 mice. *P < .004 between HA-mFVIIa-VEAY (H) survival compared with untreated HA. All data (except in panel D) shown as average ± 1 SD.

Hemostatic efficacy of mFVIIa-VEAY after high-dose gene transfer in hemophilia A mice. (A) aPTT in HA mice after delivery of AAV-mFVIIa-VEAY (1.2 × 1012 vg/mouse). The initial cohort consisted of 10 mice. The gray box indicates the range of aPTT values using human FVIII-deficient plasma in hemostatically normal mice (37.9 ± 1.6 seconds, n = 10). (B) Blood loss after a tail clip assay, compared with AAV-mFVIIa–treated HA mice, or untreated HA and wild-type (WT) mice, whose data are depicted in Figure 3C and are repeated here for comparison. *P < .02 vs HA; **P < .002 vs WT. (C) TAT levels in AAV-mFVIIa-VEAY–treated mice as a function of time. *P < .03 vs baseline (Pre). (D) Survival of HA mice treated with high-dose AAV-mFVIIa-VEAY (AAV-mFVIIa-VEAY [H], ●), relative to untreated HA controls (□). All cohorts initially consisted of 10 mice. *P < .004 between HA-mFVIIa-VEAY (H) survival compared with untreated HA. All data (except in panel D) shown as average ± 1 SD.

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