Figure 5.
Significant Improvement in Behavior Performance and Survival after a Systemic scAAV9-mCMV-hIDS Gene Delivery
(A–E) MPS II mice were treated at age 1 month, 3 months, 6 months, or 9 months with an i.v. injection of scAAV9-mCMV-hIDS at (A–C) 5 × 1012 vg/kg, (D) 1 × 1013 vg/kg, or (E) 2 × 1013vg/kg. Behavior testing was performed in a hidden task in the Morris water maze when they were 8 months and/or 12 months old (-Re). (A) Dose comparison. (B) Comparison of treatment ages. (C–E) Vector treatment at age 6 months; (C) 5 × 1012 vg/kg, (D) 1 × 1013 vg/kg, and (E) 2 × 1013 vg/kg. Data were means ± SD of latency to find hidden platform (in seconds) and swimming speed (in centimeters per second). *p < 0.05 versus WT; ˆp > 0.05 versus WT; #p < 0.05 versus non-treated MPS II; +p > 0.05 versus non-treated MPS II.
(F) Survival.