Table 2.

Gb3 levels in mouse tissues after tail vein administration of rAAV2/8-hAGA vector

Mice group	Weeks after injection	Gb3 levels (nmol/mg protein)
		Liver	Kidney	Spleen	Heart	Brain
Untreated Fabry mice		2.498 ± 0.261	7.466 ± 0.743	20.665 ± 5.999	7.179 ± 1.939	2.079 ± 1.099
Treated mice 2 × 1011a	6	0.001 ± 0.001	0.032 ± 0.018	0.014 ± 0.007	0.003 ± 0.001	0.139 ± 0.022
	12	0.010 ± 0.001	0.832 ± 0.108	1.098 ± 0.129	0.058 ± 0.047	0.520 ± 0.192
	24	0.015 ± 0.001	1.948 ± 1.487	1.392 ± 0.215	0.054 ± 0.040	1.832 ± 0.299
Treated mice 2 × 1012b	6	0.005 ± 0.002	0.019 ± 0.007	0.013 ± 0.006	0.002 ± 0.001	0.092 ± 0.043
	12	0.007 ± 0.004	0.640 ± 0.349	0.197 ± 0.054	0.019 ± 0.076	0.325 ± 0.146
	24	0.019 ± 0.020	0.359 ± 0.011	0.695 ± 0.252	0.040 ± 0.019	1.267 ± 0.210
	48	0.046 ± 0.001	0.008 ± 0.001	0.732 ± 0.026	0.069 ± 0.108	1.434 ± 0.097
	60	0.092 ± 0.001	0.023 ± 0.071	0.969 ± 0.049	0.502 ± 0.901	1.640 ± 0.127
Treated mice ERTc	6	0.002 ± 0.001	0.263 ± 0.062	0.015 ± 0.005	0.003 ± 0.001	0.098 ± 0.025
Wild-type mice		0.036 ± 0.013	0.150 ± 0.013	0.252 ± 0.058	0.036 ± 0.0087	0.025 ± 0.011

Data present as average ± SD, ^a mice treated with 2 × 10¹¹: rAAV2/8-hAGA (2 × 10¹¹ particles/mouse), ^b mice treated with 2 × 10¹²: rAAV2/8-hAGA (2 × 10¹² particles/mouse), n = 3. ^cERT treated mice: 1.0 mg/kg once a week for 6 consecutive weeks.