Figure 7: Slc6a3 gene therapy prevents motor deficits and the neurodegenerative phenotype in DAT-KO mice.

Rescue of motor performance in the pole test measured as (A) time to turn from an upside down position in treated KO animals (T-turn). (B) Decrease in the time treated KO descend from the pole when differs from the performance of WT mice. Rescue of (C) gait width and stride length (D) in treated KO animals compared to KO controls after Slc6a3 AAV delivery. (E) The prevention of clasping behavior in the tail suspension test for the treated KO cohort. (F) Total rescue of clasping behavior at the tail suspension test, in which treated KO animals demonstrated scores similar to those of WT mice. (G) Mantel-Cox survival curve (p = 0.0353) depicting the absence of mortality in KO control animals (n = 16) compared to treated KO mice (n = 16–12). (H) Monitoring of body weight increase. KO treated animals display higher body weight gain 48 days after AAV delivery (Two-way ANOVA F(1,171) = 13.56, Bonferroni post-hoc test, p = 0.003). Data are expressed as the mean ± SEM. One-way ANOVA+ Holm-Sidak post-hoc and two-way ANOVA+ Bonferroni post-hoc tests were used for multiple comparisons. ***p < 0.001; **p < 0.01; *p < 0.05; n.s – not significant.