Summary of gene transfer efficiency after peripheral vein delivery of scAAV2/8-LP1-hFIXco
| Monkey . | Wt, kg . | Steady-state hFIX antigen level, % of normal . | hFIX biologic activity, % of normal . | Transgene copy no. per diploid genome* . | Duration of expression, d . |
|---|---|---|---|---|---|
| M5-sc | 4.9 | 28 ± 4 | 21 ± 4 | 47 | > 401 |
| M6-sc | 5.7 | 16 ± 4 | 12 ± 4 | 45 | > 311 |
| M7-sc | 4.3 | 14 ± 1 | 12 ± 3 | 40 | > 292 |
| Monkey . | Wt, kg . | Steady-state hFIX antigen level, % of normal . | hFIX biologic activity, % of normal . | Transgene copy no. per diploid genome* . | Duration of expression, d . |
|---|---|---|---|---|---|
| M5-sc | 4.9 | 28 ± 4 | 21 ± 4 | 47 | > 401 |
| M6-sc | 5.7 | 16 ± 4 | 12 ± 4 | 45 | > 311 |
| M7-sc | 4.3 | 14 ± 1 | 12 ± 3 | 40 | > 292 |
Transgene copy number estimated by Southern blot analysis.