Table 1.

Study design.

Study number	Injection route	GNE-Lipoplex dose (total volume injected)	Treatment	Mice/cohort	Assessment
I	Intramuscular	10 μg (80 μl)	GNE-lipoplex	6 female, 6 male	Safety and toxicology,
		40 μg (80 μl)	GNE-lipoplex	6 female, 6 male	GNE expression
		0 μg (80 μl)	Empty liposomes	6 female, 6 male
		No injection	–	6 female, 6 male
II	Intramuscular	40 μg (80 μl)	GNE-lipoplex	15 female	GNE expression
		0 μg (80 μl)	Empty liposomes	6 female
III	Intravenous	10 μg (200 μl)	GNE-lipoplex	6 female, 6 male	Safety and toxicology,
		40 μg (200 μl)	GNE-lipoplex	6 female, 6 male	GNE expression
		100 μg (200 μl)	GNE-lipoplex	6 female, 6 male
		0 μg (200 μl)	Empty liposomes	6 female, 6 male