Figure 2.

Significant Reduction of GAG Content in the CNS and Peripheral Tissues in MPS II Mice following a Systemic scAAV9-hIDS Gene Delivery

MPS II mice were treated at age 1 month (A and B), 3 months (C), 6 months (D–F), or 9 months (G) with an i.v. injection of scAAV9-mCMV-hIDS at 2.5 × 10¹² vg/kg (A), 5 × 10¹² vg/kg (B–D), 1 × 10¹³ vg/kg (E), or 2 × 10¹³ vg/kg (F and G). Tissues were assayed for GAG contents at 1 month pi, age 8 months, or humane endpoint (n ≥ 4 per group). GAG content is expressed as micrograms per milligram of wet tissue. WT, non-treated WT mice; MPS II, non-treated MPS II mice; m/m, injection age/testing age in months; LIV, liver; Kid, kidney; Int, intestine; Hrt, heart; Spl, spleen; Mus, skeletal muscle. *p ≤ 0.05 versus WT; ^#p > 0.05 versus WT; ˆp ⩽ 0.05 versus non-treated MPS II; ^@p > 0.05 versus non-treated MPS II (t test).