Table 1.
Summary of gene transfer and expression results for mice receiving AAV8-FVIII-BDD or AAV8-FVIII-RH
Dose | Gene copy number (copies per cell) | RNA transcripts (FVIII/18S RNA) | Circulating FVIII (ng/mL) | |||
---|---|---|---|---|---|---|
AAV8-BDD | AAV8-RH | AAV8-BDD | AAV8-RH | AAV8-BDD | AAV8-RH | |
8 × 1012 vg/kg | 16.8 ± 4.2 | 18.7 ± 4.0 | 0.09 ± 0.03 | 0.11 ± 0.04 | 49.9 ± 9.4 | 78.9 ± 6.6 |
2 × 1013 vg/kg | 64.6 ± 15.6 | 35.0 ± 9.1 | 0.04 ± 0.01 | 0.09 ± 0.03 | 71.4 ± 7.5 | 123.5 ± 19.3 |
4 × 1013 vg/kg | 84.7 ± 24.0 | 71.4 ± 12.9 | 0.10 ± 0.06 | 0.23 ± 0.12 | 75.1 ± 9.8 | 158.1 ± 28.3 |