Figure 2.

i.c.v. Injection of AAV9-SYN-SMN and i.v. Injection of AAV9-PGK-SMN Restores SMN Expression to Similar Levels in the Spinal Cord and Brain of SMNΔ7 Mice

(A) Western blot analyses of SMN expression (37 kDa) in the spinal cords (upper panels) and brains (lower panels) of 14-day-old SMNΔ7 mice after i.c.v. injection of AAV9-SYN-SMN (i.c.v.-SYN, n = 3) at a dose of 1.2 × 10¹¹ vg/mouse or i.v. (i.v.-PGK, n = 3) or i.c.v. injection (i.c.v.-PGK, n = 3) of AAV9-PGK-SMN at a dose of 4.5 × 10¹⁰ vg/mouse. Protein lysates from an age-matched non-injected SMNΔ7 mouse (n = 1) and WT mice (n = 2) were used as references for SMN expression. Tuba1a (50 kDa) was used as a loading control. (B and C) Densitometric analysis of western blot signals in the spinal cords (B) and brains (C) of injected SMNΔ7 mice. Statistical analysis was performed on n = 3 mice for each group of injected mice. Significant differences in SMN expression were observed between i.c.v. injection of AAV9-PGK-SMN and either i.v. injection of the same vector or i.c.v. injection of AAV9-SYN-SMN. Values are expressed as the SMN mean intensity relative to that of Tuba1a ± SEM, and the differences between groups were analyzed by one-way ANOVA followed by Tukey’s post hoc test (∗∗p < 0.01, ∗∗∗p < 0.001, ∗∗∗∗p < 0.0001).