Phenotypic correction of hemophilia in Hem-A mice
Group no. . | Dose . | Average FVIII activity, mU/mL . | No. of surviving mice . |
|---|---|---|---|
| 1 | 3 × 1010 | 390 ± 191 | 4/4 |
| 2 | 3 × 1011 | 15 485 ± 4839 | 4/4 |
| 3 | 9 × 1011 | 27 194 ± 16 172 | 4/4 |
| 4 | No vector | 0 | 0/2 |
Group no. . | Dose . | Average FVIII activity, mU/mL . | No. of surviving mice . |
|---|---|---|---|
| 1 | 3 × 1010 | 390 ± 191 | 4/4 |
| 2 | 3 × 1011 | 15 485 ± 4839 | 4/4 |
| 3 | 9 × 1011 | 27 194 ± 16 172 | 4/4 |
| 4 | No vector | 0 | 0/2 |
Hem-A mice were injected with AAV-cFVIII-HC and AAV-cFVIII-LC vectors. At 12 weeks after injection, the mice were lightly anesthetized with isoflurane and their tails were clipped at an internal diameter of 2.0 mm. Mice were monitored for survival over a 24-hour period. Activity levels at time of clipping are shown.