TABLE 1.

Design of AAV‐GFP mouse study. WT and Pah^enu2 mice were assigned to one of six treatment groups with escalating doses of Anc80‐GFP, an adeno‐associated virus vector expressing GFP under the control of the liver‐specific A1AT promoter. Mice were kept on study for 3 weeks after a single administration of vector on day 1. Mice underwent a focused necropsy at the end of the study to evaluate transduction and expression of the vector‐mediated GFP transgene

Group	Genotype	Article	Dose (VG/kg)	Day 21 (M/F)
1	Pahenu2	Anc80‐GFP	1.5 × 1012	3/3
2	Pahenu2	Anc80‐GFP	5 × 1012	3/3
3	Pahenu2	Anc80‐GFP	1.5 × 1013	3/3
4	Pahenu2	Saline	Saline	2/2
5	WT	Anc80‐GFP	5 × 1012	3/3
6	WT	Saline	Saline	2/2

Abbreviations: A1AT, alpha‐1 antitrypsin; AAV, adeno‐associated virus; f, female; GFP, green fluorescent protein; m, male; Pah^enu2, PKU mouse; VG, vector genomes; WT, wild type.