Table 1.
Vector Doses Administered in Preclinical and Clinical Studies
| Study | Dose | Target | Sites/hemis. | Vi/hemis. (μl) | VG/hemis. | Total VG (×109) |
|---|---|---|---|---|---|---|
| Present study | pr.c. Cd/Put | 2 (UL) | 100 | 7.0×1011 | 700 | |
| NHP safety (Forsayeth et al., 2006) | Lowest | c./pt.c. Put | 2 (BL) | 100 | 6.0×109 | 12 |
| Highest | c./pt.c. Put | 2 (BL) | 100 | 5.0×1011 | 1000 | |
| Clinical phase 1 (Christine et al., 2009) | Low | pt.c. Put | 2 (BL) | 100 | 4.5×1010 | 90 |
| High | pt.c. Put | 2 (BL) | 100 | 1.5×1011 | 300 |
BL, bilateral; c., commissural; Cd, caudate nucleus; hemis., hemisphere; NHP, nonhuman primate; pr.c., precommissural; pt.c., postcommissural; Put, putamen; UL, unilateral; VG, vector genomes; Vi, infusion volume.