Summary of AAV8-hFIX-WT and hFIX-Padua expression in C57BL/6 mice
| Dose (vg/kg) . | hFIX-WT . | hFIX-Padua . | ||||
|---|---|---|---|---|---|---|
| Activity, % mean ± SEM . | Antigen, % mean ± SEM . | Specific activity . | Activity, % mean ± SEM . | Antigen, % mean ± SEM . | Specific activity . | |
| 1 × 1010 | 39.9 ± 0.9 | 2.8 ± 0.3 | NA* | 53.8 ± 6.4 | 3.0 ± 0.2 | NA* |
| 4 × 1010 | 63.6 ± 10.4 | 33.5 ± 2.4 | 1.9 | 173.5 ± 27.8 | 16.1 ± 3.2 | 10.8 |
| 1 × 1011 | 115.4 ± 21.9 | 62.0 ± 7.1 | 1.9 | 424.1 ± 59.8 | 45.9 ± 11.3 | 9.2 |
| 4 × 1011 | 456.3 ± 15.2 | 439.1 ± 17.4 | 1.0 | 1978.5 ± 344.1 | 271.2 ± 51.9 | 7.3 |
| 2 × 1012 | 698.6 ± 110.8 | 1020.4 ± 129.8 | 0.7 | ND | ND | ND |
| Average | 1.4 | 9.1 | ||||
| Dose (vg/kg) . | hFIX-WT . | hFIX-Padua . | ||||
|---|---|---|---|---|---|---|
| Activity, % mean ± SEM . | Antigen, % mean ± SEM . | Specific activity . | Activity, % mean ± SEM . | Antigen, % mean ± SEM . | Specific activity . | |
| 1 × 1010 | 39.9 ± 0.9 | 2.8 ± 0.3 | NA* | 53.8 ± 6.4 | 3.0 ± 0.2 | NA* |
| 4 × 1010 | 63.6 ± 10.4 | 33.5 ± 2.4 | 1.9 | 173.5 ± 27.8 | 16.1 ± 3.2 | 10.8 |
| 1 × 1011 | 115.4 ± 21.9 | 62.0 ± 7.1 | 1.9 | 424.1 ± 59.8 | 45.9 ± 11.3 | 9.2 |
| 4 × 1011 | 456.3 ± 15.2 | 439.1 ± 17.4 | 1.0 | 1978.5 ± 344.1 | 271.2 ± 51.9 | 7.3 |
| 2 × 1012 | 698.6 ± 110.8 | 1020.4 ± 129.8 | 0.7 | ND | ND | ND |
| Average | 1.4 | 9.1 | ||||
ND, not determined; SEM, standard error of the mean.
Low antigen level detection limits impact specific activity calculation.