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Summary of Primary Toxicology and Biodistribution Dose Groups
| Group number | Number of SD rats (sex) | Substance | Total dose (vg) | Total dose volume (μL) | Dosing regimen | Route | Observation period |
|---|---|---|---|---|---|---|---|
| 1 | 8 (4F/4M) | BSS | — | 2 | 1 eye, 1 time | SR | 90 days |
| 2 | 19 (10F/9M) | AAV2-VMD2-hMERTK | 4×108 | 2 | 1 eye, 1 time | SR | 90 days |
| 3 | 19 (10F/9M) | AAV2-VMD2-hMERTK | 4×109 | 2 | 1 eye, 1 time | SR | 90 days |
BSS, balanced salt solution; SD, Sprague–Dawley; SR, subretinal; vg, vector genomes.
