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Study design
| Group | Number of Animals (Sex) | Number of Animals (Sex) at Termination | |||||
|---|---|---|---|---|---|---|---|
| Day 28 (Week 4) | Day 84 (Week 12) | Vector | Concentration (vg/mL) | Volume (μL) | Total Dose (vg/eye) | ||
| 1 | 20 (11M/9F) | 10 (6M/4F) | 10 (5M/5F) | Vehicle (Alcon balanced salt solution with 0.014% Tween 20) | 0 | 1 | 0 |
| 2 | 20 (10M/10F) | 10 (5M/5F) | 10 (5M/5F) | rAAV2tYF-GRK1-RPGRco | 4.0 × 1011 | 1 | 4.0 × 108 |
| 3 | 20 (10M/10F) | 10 (5M/5F) | 10 (5M/5F) | rAAV2tYF-GRK1-RPGRco | 4.0 × 1012 | 1 | 4.0 × 109 |
F, female; M, male.
