Fig. 5
Prolonged FVIII functional and phenotype correction in F8null mice after tail vein injection LV-VEC-F8BDD. The F8null mice were treated with non-myeloablative radiation and given an iv injection of LV-F8BDD under the control of EF1α, VEC, Gp and ITGA promoters (1 × 107 TU per animal) or PBS (100 μL per animal) mock control. A The kinetics of FVIII activities in plasma examined by the chromogenic assay on days 7, 15, 30, 45, 60, 120 and 180 (n = 3). B The FVIII acticities in 1 × 109 platelets based on the chromogenic assay at day 60 (n = 3). C The tail bleeding time analysis recorded at day 120. The time required to stop bleeding into the collection tubes containing saline solution was recorded and plotted (n = 3). D The percentage survival curves of mice after LV injection for up to 180 days. The tail clipping experiment was carried out at day 120. The differences in characteristics between groups were analyzed using the one way Welch ANOVA tests with Turkey post hoc tests (A, C) or Games-Howell post hoc tests (A and B); *p < 0.05, **p < 0.01, ****p < 0.0001