Densitometric analysis of Southern blot yields gene copy no. in injected muscle
. | . | Actual gene copy no. . | . | Relative gene copy no. . | . | Relative circulating F.IX, ng/mL . | . | |||
|---|---|---|---|---|---|---|---|---|---|---|
| Vector dose, vg/kg . | Fold increase in dose . | AAV-2 . | AAV-1 . | AAV-2 . | AAV-1 . | AAV-2 . | AAV-1 . | |||
| 2 × 1011 | 1 | 0.4 | 14.5 | 1 | 36 | 1.0 | 10 | |||
| 1 × 1012 | 5 | 3.0 | 20.0 | 8 | 50 | 1.0 | 20 | |||
| 4 × 1012 | 20 | 15.0 | 33.0 | 38 | 82 | 2.5 | 56 | |||
. | . | Actual gene copy no. . | . | Relative gene copy no. . | . | Relative circulating F.IX, ng/mL . | . | |||
|---|---|---|---|---|---|---|---|---|---|---|
| Vector dose, vg/kg . | Fold increase in dose . | AAV-2 . | AAV-1 . | AAV-2 . | AAV-1 . | AAV-2 . | AAV-1 . | |||
| 2 × 1011 | 1 | 0.4 | 14.5 | 1 | 36 | 1.0 | 10 | |||
| 1 × 1012 | 5 | 3.0 | 20.0 | 8 | 50 | 1.0 | 20 | |||
| 4 × 1012 | 20 | 15.0 | 33.0 | 38 | 82 | 2.5 | 56 | |||
Relative gene copy number values obtained by normalizing to AAV-2—injected value at lowest dose. Note that the difference in gene copy number between AAV-1—and AAV-2—injected tissue is most marked at low doses. Relative circulating F.IX levels were determined by normalizing to levels obtained with the lowest dose (2 × 1011 vg/kg) of AAV-2.