F.IX levels in the conditioned medium from murine C2C12 cells transduced with AAV-CMV-F.IX vectors at a range of multiplicity of infection
Multiplicity of Infection (MOI) . | AAV-1, F.IX ng/mL per 48 h . | AAV-2, F.IX ng/mL per 48 h . | Fold difference . |
|---|---|---|---|
| Undifferentiated C2C12 cells | |||
| 1 000 | 3 ± 1 | 12 ± 2.6 | 4 |
| 5 000 | 19 ± 4 | 60 ± 13 | 3 |
| 20 000 | 29 ± 10 | 108 ± 37 | 3.7 |
| 40 000 | 36 ± 4.5 | 129 ± 40 | 3.5 |
| Differentiated C2C12 cells | |||
| 1 000 | 33 ± 10 | 107 ± 47 | 3.2 |
| 5 000 | 63 ± 29 | 126 ± 29.4 | 2 |
| 20 000 | 103 ± 12 | 208 ± 30 | 2 |
| 40 000 | 115 ± 8 | 247 ± 58 | 2 |
| Fold range difference | 3 to 10 | 2 to 15 |
Multiplicity of Infection (MOI) . | AAV-1, F.IX ng/mL per 48 h . | AAV-2, F.IX ng/mL per 48 h . | Fold difference . |
|---|---|---|---|
| Undifferentiated C2C12 cells | |||
| 1 000 | 3 ± 1 | 12 ± 2.6 | 4 |
| 5 000 | 19 ± 4 | 60 ± 13 | 3 |
| 20 000 | 29 ± 10 | 108 ± 37 | 3.7 |
| 40 000 | 36 ± 4.5 | 129 ± 40 | 3.5 |
| Differentiated C2C12 cells | |||
| 1 000 | 33 ± 10 | 107 ± 47 | 3.2 |
| 5 000 | 63 ± 29 | 126 ± 29.4 | 2 |
| 20 000 | 103 ± 12 | 208 ± 30 | 2 |
| 40 000 | 115 ± 8 | 247 ± 58 | 2 |
| Fold range difference | 3 to 10 | 2 to 15 |
Over prolonged periods and over a broad dose range, AAV-1—and AAV-6—based vectors result in higher levels of expression of a F.IX transgene compared with AAV-2 vectors in immunodeficient mice.