Table 1.
Experimental groups and study design
| ID | Experimental group | Vector | Target sites (injected volume in μl) | Total TU/site | Total TU/brain | Time of analysis (days post‐injection) | ||
|---|---|---|---|---|---|---|---|---|
| P1 | Pilot | LV.GFP | Anterior EC (40) | Thal/CR (80) | 2 × 107 | 4 × 107 | 6 × 107 | 35–36 |
| P2 | Anterior EC (150) | Thal/CR (150) | 4 × 107 | 4 × 107 | 8 × 107 | |||
| S1.1 | Study group 1 | LV.hARSA | Anterior EC (80) | Thal (80) | 5 × 107 | 1 × 108 | 92–94 | |
| S1.2 | ||||||||
| S1.3 | ||||||||
| S2.1 | Study group 2 | Anterior EC (80) | Posterior EC (80) | 5 × 107 | 1 × 108 | 98–100 | ||
| S2.2 | ||||||||
| S2.3 | ||||||||
The indicated doses and volumes of LV.GFP suspension were injected via CED in the anterior external capsule (EC) and in the thalamus (Thal)/corona radiata (CR) of the two NHP in the pilot group. The remaining NHP were then randomly assigned to the study groups 1 and 2. A total dose of 1 × 108 TU of LV.hARSA was injected in two injection sites in NHP of study group 1 (anterior EC and Thal) and study group 2 (anterior and posterior EC). The time of analysis for each group is shown in the last column.