. Author manuscript; available in PMC: 2014 Dec 23.

Published in final edited form as: Mol Ther Methods Clin Dev. 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)
Group	Vector conc. (vg/mL)	Vector dose (vg)	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)

Abbreviations: Conc.: concentration, vg: vector genome.