Phenotypic correction of C57BI/6 hemophilia-B mice treated with different amounts of the high-capacity AdFTC/hFIX or fgAdh/FIX
Group . | Treatment: virus (transducing particles) . | Serum levels hFIX, ng/mL . | Tail clip bleeding time, min . |
---|---|---|---|
1 | AdFTC/hFIX (1 × 109) | 34 227 ± 14 457 | 3.1 ± 0.5 |
2 | AdFTC/hFIX (5 × 108) | 12 143 ± 5 447 | 3.7 ± 0.3 |
3 | AdFTC/hFIX (1 × 108) | 3 341 ± 1 227 | 3.8 ± 0.4 |
4 | FgAdhFIX (1 × 109) | 910 ± 137 | 5.2 ± 0.2 |
4 | Vehicle (PBS) | 0 | No clotting (> 30 min) |
Group . | Treatment: virus (transducing particles) . | Serum levels hFIX, ng/mL . | Tail clip bleeding time, min . |
---|---|---|---|
1 | AdFTC/hFIX (1 × 109) | 34 227 ± 14 457 | 3.1 ± 0.5 |
2 | AdFTC/hFIX (5 × 108) | 12 143 ± 5 447 | 3.7 ± 0.3 |
3 | AdFTC/hFIX (1 × 108) | 3 341 ± 1 227 | 3.8 ± 0.4 |
4 | FgAdhFIX (1 × 109) | 910 ± 137 | 5.2 ± 0.2 |
4 | Vehicle (PBS) | 0 | No clotting (> 30 min) |
On day 3 after injection, an enzyme-linked immunosorbent assay for human factor IX expression levels was performed, and on day 6, a tail clip assay was performed. All groups are of hemophilic mice.
PBS indicates phosphate-buffered saline. For other abbreviations, see Table 2.