Table 1.
Study Design: Systemic scAAV9-mCMV-hIDS Gene Delivery in MPS II Mice
| Mice | Vector Dose (vg/kg) | Injection Age (Months) | No. of Animals |
||||||
|---|---|---|---|---|---|---|---|---|---|
| Total | Behavior Testing | Necropsy |
Longevity | ||||||
| 1 Month pi | Age 8 Months | Age 12 Months | Endpoint | ||||||
| MPS II | 2.5 × 1012 | 1 | 20 | 15a | 5 | 5 | 0 | 6 | 10 |
| MPS II | 5 × 1012 | 1 | 20 | 15a | 5 | 5 | 0 | 5 | 10 |
| MPS II | 5 × 1012 | 3 | 19 | 13a | 5 | 5 | 0 | 5 | 9 |
| MPS II | 5 × 1012 | 6 | 19 | 14b | 4 | 4 | 0 | 6 | 11 |
| MPS II | 1 × 1013 | 6 | 22 | 16b | 4 | 5 | 0 | 4 | 13 |
| MPS II | 2 × 1013 | 6 | 18 | 14c | 4 | 5 | 0 | 4 | 9 |
| MPS II | 2 × 1013 | 9 | 19 | 0 | 4 | 0 | 4 | 4 | 11 |
| WT | 0 | N/A | 86d | 83d | 2 | 10 | 0 | – | 74d |
| MPS II | 0 | N/A | 24 | 20 | 1 | 5 | 0 | 3 | 18 |
a
Tested at age 8 months.
b
Tested at age 8 months and re-tested at age 12 months.
c
Tested at age 12 months.
d
Combined data from multiple studies.