Table 1.
Summary of AAV8-cFIX-Padua treatment of 3 adult dogs with severe HB
| Dog | cFIX-Padua plateau levels | Bleeds per month | ||||||
|---|---|---|---|---|---|---|---|---|
| Body weight, kg | Vector dose, vg/kg | Vector dose, total dose | Activity, % mean ± standard deviation | Antigen, % mean ± standard deviation | Specific activity | Pretreatment | Posttreatment | |
| Wiley | 10.2 | 3 × 1012 | 30.6 × 1012 | 241.2 ± 31.9 | 28.8 ± 9.3 | 8.6 | 7/32 | 0/36 |
| Trex | 5.8 | 3 × 1012 | 17.4 × 1012 | 26.8 ± 5.5 | 3.81 ± 1.29 | 7.9 | 4/32 | 0/36 |
| Wick | 8.6 | 1 × 1012 | 8.6 × 1012 | 39.9 ± 5.8 | 3.33 ± 0.38 | 12 | 0/11 | 0/19 |
| Total | 11/75 | 0/91 | ||||||