Fig. 6

AAV-mediated gene therapy improves behavioural testing and is inversely correlated with cortical neuronal loss in treated animals. a Open field test and b accelerating rotarod performed in 2-month-old mice. c, d Apoptotic cells at time of culling in WT (aged 9–13 months), untreated (aged 21 days − 13 months), adult-injected (aged 13 months) and neonatally injected Asl^Neo/Neo mice (aged 9 months). c Cell counting in cortex. d Representative cortex image of an untreated 25-day-old Asl^Neo/Neo mouse symptomatic of hyperammonaemia and culled for humane endpoint. Horizontal lines display the mean ± SEM. a, b One-way ANOVA with Dunnett’s post-test compared to WT: ns—not significant, *p < 0.05, **p < 0.01, ***p < 0.001. c Graph displays not transformed data. Statistical analysis used log-transformed data. WT n = 20–22; untreated Asl^Neo/Neo n = 8–9; adult-injected Asl^Neo/Neo n = 5; neonatally injected Asl^Neo/Neo n = 5. Scale bars: 500 μm and 125 μm in main and inset pictures, respectively