Fig. 2. Neonatal ICV injection of AAV-sgSOD1 prevents motor function decline and significantly extends survival in H11^Cas9 SOD1^G93A mice.

a Study design of two independent cohorts (Cohorts #1 and #2) of H11^Cas9 SOD1^G93A mice treated with AAV-sgSOD1. b, c Kaplan-Meier survival curves of Cohorts #1 and #2. Log-Rank test, P < 0.0001. d Body weight of mice over time (Cohort #1). Behavioral assessments of SOD1^G93A mice treated with AAV-sgSOD1: rotarod performance measured as latency to fall over age (e), total distance travelled in open field over time (f), performance in inverted grid test measured as latency to fall at week 14 and 19 (g), and clasping score over age (h). Data are presented as mean with standard deviation (SD) unless otherwise stated. Two-way ANOVA was performed with all groups compared to WT group in d–f. One-way ANOVA Dunnett’s multiple comparisons test was performed with all groups compared to WT group in g (n.s. is not labeled). *p < 0.05, **p < 0.01, and ***p < 0.001, and n.s. not significant (p > 0.05).