Table 1.
Group Designations and Dose
| Group | Group Designation | Genotype | No. of Animals | Each ZFN Dose Level (vg/Mouse) | Donor Dose Level (vg/Mouse) | Total AAV Dose (vg/Mouse) |
|---|---|---|---|---|---|---|
| 1 | formulation buffer control | C57BL/6 wild-type littermates | 13 | 0 | 0 | 0 |
| 2 | formulation buffer control | MPS II littermates | 13 | 0 | 0 | 0 |
| 3 | ZFNs+donor low dose | MPS II | 8 | 2.50E+10 | 2.00E+11 | 2.50E+11 |
| 4 | ZFNs+donor mid dose | MPS II | 8 | 5.00E+10 | 4.00E+11 | 5.00E+11 |
| 5 | ZFNs+donor high dose | MPS II | 13 | 1.50E+11 | 1.20E+12 | 1.50E+12 |
| 6 | donor only | MPS II | 8 | 0 | 4.00E+11 | 4.00E+11 |