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. 2014 Oct 15;3:14049. doi: 10.1038/mtm.2014.49

Table 1. Dosing groups and necropsy schedule.

Pilot study
Group Vector conc. (vg/ml) Vector dose (vg) Survival time
AAV2-hAADC high dose 8.3 × 1012 5.0 × 1011-6.0 × 1011 1 month (n = 2, female)
Pivotal toxicology study
Group Vector conc. (vg/ml) Vector dose (vg) Survival time groups (male + female)
1 month (n = 5) 3 months (n = 5) 9 months (n = 6)
Control 0 0 1 male 1 male 2(1 + 1)
AAV2-hAADC low dose 8.3 × 1011 5.0 × 1010 2(1 + 1) 2(1 + 1) 2(1 + 1)
AAV2-hAADC high dose 8.3 × 1012 5.0 × 1011 2(1 + 1) 2(1 + 1) 2(1 + 1)

Conc., concentration; vg, vector genome.