Table 1.
Age | Route of Administration | Dose Volume | Number of Animals |
---|---|---|---|
Adult (3–6 years) | ICM | 1 mL | 3 |
LP | 5 mL | 4 | |
Juvenile (12–14 months) | ICM | 1 mL | 3 |
LP | 5 mL | 2 |
Macaques were administered 3 × 1013 GC of an AAVhu68 vector expressing a human SMN transgene. The study duration was 28 days.