Summary of hFVIII expression in murine models
| Vector . | Capsid serotype . | Dose (vg/kg) . | Mouse model . | hFVIII:Ag levels (%; mean ± SD) . |
|---|---|---|---|---|
| rAAV-LP1-BDD-hFVIII | 5 | 2 × 1013 | Wild-type C57Bl/6 | 1 ± 0.3 |
| rAAV-LP1-hFVIII-N6 | 2 ± 0.6 | |||
| rAAV-LP1-codop-hFVIII-N6 | 19 ± 6 | |||
| rAAV-HLP-codop-hFVIII-N6 | 22 ± 6 | |||
| rAAV-HLP-codop-hFVIII-N6 | 8 | 2 × 1013 | Wild-type C57Bl/6 | 540 ± 218* |
| rAAV-HLP-codop-BDD-hFVIII | 8 | 2 × 1012 | Wild-type C57Bl/6 | 56 ± 27 |
| rAAV-HLP-codop-hFVIII-N6 | 83 ± 24 | |||
| rAAV-HLP-codop-hFVIII-V1 | 9 ± 2 | |||
| rAAV-HLP-codop-hFVIII-V3 | 177 ± 34 | |||
| rAAV-HLP-codop-BDD-hFVIII | 8 | 2 × 1012 | FVIII−/− | 243 ± 112 |
| rAAV-HLP-codop-hFVIII-N6 | 542 ± 124 | |||
| rAAV-HLP-codop-hFVIII-V3 | 732 ± 162 | |||
| rAAV-HLP-codop-BDD-hFVIII | 8 | 2 × 1013 | FVIII−/− | 671 ± 209 |
| rAAV-HLP-codop-hFVIII-N6 | 1330 ± 427 | |||
| rAAV-HLP-codop-hFVIII-V3 | 2578 ± 527 |
| Vector . | Capsid serotype . | Dose (vg/kg) . | Mouse model . | hFVIII:Ag levels (%; mean ± SD) . |
|---|---|---|---|---|
| rAAV-LP1-BDD-hFVIII | 5 | 2 × 1013 | Wild-type C57Bl/6 | 1 ± 0.3 |
| rAAV-LP1-hFVIII-N6 | 2 ± 0.6 | |||
| rAAV-LP1-codop-hFVIII-N6 | 19 ± 6 | |||
| rAAV-HLP-codop-hFVIII-N6 | 22 ± 6 | |||
| rAAV-HLP-codop-hFVIII-N6 | 8 | 2 × 1013 | Wild-type C57Bl/6 | 540 ± 218* |
| rAAV-HLP-codop-BDD-hFVIII | 8 | 2 × 1012 | Wild-type C57Bl/6 | 56 ± 27 |
| rAAV-HLP-codop-hFVIII-N6 | 83 ± 24 | |||
| rAAV-HLP-codop-hFVIII-V1 | 9 ± 2 | |||
| rAAV-HLP-codop-hFVIII-V3 | 177 ± 34 | |||
| rAAV-HLP-codop-BDD-hFVIII | 8 | 2 × 1012 | FVIII−/− | 243 ± 112 |
| rAAV-HLP-codop-hFVIII-N6 | 542 ± 124 | |||
| rAAV-HLP-codop-hFVIII-V3 | 732 ± 162 | |||
| rAAV-HLP-codop-BDD-hFVIII | 8 | 2 × 1013 | FVIII−/− | 671 ± 209 |
| rAAV-HLP-codop-hFVIII-N6 | 1330 ± 427 | |||
| rAAV-HLP-codop-hFVIII-V3 | 2578 ± 527 |
These values are from the 12-wk time point. The rest of the data in the table are from 6 wk after gene transfer to allow comparisons between different experiments. The shaded rows are a summary of studies in FVIII−/− mice, which were performed by the laboratory of P.J.L. using a different set of assays and standards.