Figure 5.

i.c.v. Injection of SMNΔ7 Mice with AAV9-PGK-SMN Provides Better and Sustained Protection of Locomotor Functions than Does Systemic Delivery

(A) The rotarod performance of SMNΔ7 mice injected i.v. (i.v.-PGK, blue dots, n = 17) or i.c.v. (i.c.v.-PGK, pink dots, n = 12) with AAV9-PGK-SMN at a dose of 4.5 × 10¹⁰ vg/mouse and He mice (black dots, n = 34) was assessed daily from 10 weeks of age. Mice were trained for 10 days and the rotarod performance was recorded during the next 10 days. Data are expressed as the mean ± SEM. Differences between groups were analyzed by the Kruskal-Wallis test followed by Dunn’s post hoc test (∗∗p < 0.01, ∗∗∗p < 0.001). (B–D) Spontaneous motor activity monitored during 1 h using an actimeter for SMNΔ7 mice injected i.v. (i.v.-PGK, blue dots, n = 11) or i.c.v. (i.c.v.-PGK, pink dots, n = 13) with AAV9-PGK-SMN and He controls (black dots, n = 39) at 12 weeks of age. Overall time spent moving (B), the covered distance (C), and the number of rearings (D) were recorded. Data are expressed as the mean ± SEM. Differences between groups were analyzed by one-way ANOVA followed by Tukey’s post hoc test (∗p < 0.05, ∗∗p < 0.01, ∗∗∗p < 0.001, ∗∗∗∗p < 0.0001).