Figure 2.

rAAV9-mediated long-term expression of rNAGLU in the CNS and somatic tissues in C57BL/6 mice. WT C57BL/6 mice (8 weeks old) were treated with an IV injection of 1 × 10¹⁴ vg/kg or 2 × 10¹⁴ vg/kg rAAV9-CMV-hNAGLU vector (N = 30/group, M:F = 1:1). Tissues were assayed for NAGLU activity at 6 weeks, 12 weeks, and 24 weeks pi (n = 8–10/group). (a, b) Dose response comparison of tissue NAGLU activity levels at 12 weeks (a) and 24 weeks pi. *P ≤ 0.05 vs. group 1; ^{^}P > 0.05 vs. saline controls. (c) Temporal comparison of tissue NAGLU activity levels in mice treated with 2 × 10¹⁴ vg/kg rAAV9. *P ≤ 0.05 vs. 6 weeks pi; ⁺P > 0.05 vs. 6 weeks pi; ^$P > 0.05 vs. 12 weeks pi. (d) Temporal comparison of tissue NAGLU activity in mice treated with 1 × 10¹⁴ vg/kg rAAV9. NAGLU activity is expressed as units/mg protein; 1 unit = nmol 4-methylumbelliferone (4MU) released per hour. Data are means ± SD.