Figure 6. Correction of behavioral deficits and prolonged survival following intra-CSF AAV9-Sgsh delivery.

Naive-tested WT, untreated, and treated MPS IIIA male mice were evaluated by the open field test 4 months after i.c. delivery of 5 × 10⁹ (LD) or 5 × 10¹⁰ (HD) vg of AAV9-Sgsh or 5 × 10¹⁰ vg of AAV9-null vectors. Data correspond to the locomotor and exploratory activity recorded during the first 3 minutes and are represented as the mean ± SEM of 10 to 15 animals per group. (A) Total number of lines crossed, (B) total distance traveled, and (C) total number of rearings on the hind limbs. *P < 0.05, **P < 0.01, and ***P < 0.001 versus MPS IIIA–null. (D) Kaplan-Meier survival analysis in WT (n = 23), MPS IIIA– (n = 12), and HD-treated (n = 8) MPS IIIA male mice. P = 0.013 for untreated versus AAV9-treated MPS IIIA mice.