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Layout of different treatment groups
| Group | Treatment | MOI | Transplanted cells | Irradiation (Gy) | N |
|---|---|---|---|---|---|
| 1 | LV-GAAco/LV-IGF2.GAAco | 7 | 106 | 9 | 10 |
| 2 | LV-GAAco/LV-IGF2.GAAco | 2 | 106 | 9 | 10 |
| 3 | LV-GAAco/LV-IGF2.GAAco | 7 | 106 | 6 | 10 |
| 4 | LV-GAAco/LV-IGF2.GAAco | 2 | 106 | 6 | 10 |
| 5 | LV-GAAco/LV-IGF2.GAAco | 7 | 3 × 105 | 6 | 10 |
| 6 | LV-GAAco/LV-IGF2.GAAco | 2 | 3 × 105 | 6 | 10 |
| 7 | Untreated GAA−/− | 7 | NA | NA | 6 |
| 8 | Untreated WT | 2 | NA | NA | 6 |
MOI, multiplicity of infection; Gy, gray; Gaa−/− knockout; WT, FVB/N wild type; NA, not applicable.
