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Group Assignment for the Pharmacology and Toxicology Studies in Mice
| Group | Dose (AAV-IDUA and AAV-Cas9 in vg/kg) | Group Size |
|---|---|---|
| MPS I, high dose | 3 × 1014 and 5 × 1013 | 11 |
| MPS I, middle dose | 3 × 1013 and 5 × 1012 | 9 |
| MPS I, low dose | 3 × 1012 and 5 × 1011 | 13 |
| MPS I, untreated | N/A | 9 |
| Normal heterozygotes, untreated | N/A | 11 |
N/A, not available.
