Fig. 2

Motor neuron rescued and tail retention following AAV9-coSMN1 treatment one year. A, B The effects of AAV9-coSMN1 treatment on motor function and tail retention in SMA 3 model mice one year post-injection. SMA 3 mice display disease-related changes of tail morphology, highlighting improved tail length in treated mice. C, D The group comparisons of mice treated with various doses of AAV9-coSMN1 (2.7E+13, 5.3E+13, 8.0E+13, and 1.6E+14 vg/kg), demonstrating improved phenotype compared to SMA 3 controls. E The body weight change curve did not show any statistically significant differences. F, G Numbers of ChAT-positive motor neurons in treated mice at a dose of 5.3E+13 vg/kg compared to untreated controls and WT, confirming motor neuron rescued, n = 6. Statistical analysis: Mean ± SD. **P < 0.01, ***P < 0.001. Scale bars: 50 µm