Figure 1.

Acute impact of systemic rAAV9-hNAGLU gene delivery on body weights, blood liver enzyme levels, and lymphocytes in 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). (a) Body weights (BD) were measured weekly from day 1 through 24 weeks pi. Data are means ± SD. ^#P ≤ 0.05 1 × 10¹⁴ vg/kg cohort vs. saline controls; *P ≤ 0.05 2 × 10¹⁴ vg/kg vs. saline controls; **no significant differences in BD between rAAV9-treated and saline control cohorts after 6 weeks pi. Blood was assayed for clinical chemistry and hematology at 6 weeks, 12 weeks, and 24 weeks pi. (b, c) Blood AST and ALT levels and (d) blood lymphocyte counts. *P < 0.05 vs. saline controls (n = 3–5/group); gray shades: historical normal range.