Fig. 6

a RT-qPCR analysis of full-length human SMN mRNA in the spinal cord of SMA mice injected with AAV9-GFP, AAV9-SMN-WT or AAV9-SMN-2VA. Data represent mean and SEM (n = 5 animals). Statistical significance was determined by one-way ANOVA with Tukey’s post hoc test (adjusted P values: SMA + GFP vs. SMA + SMN-WT p < 0.0001; SMA + GFP vs. SMA + SMN-2VA p = 0.0001; SMA + SMN-WT vs. SMA + SMN-2VA p = 0.9742. b Western blot analysis of GFP and SMN WT or 2 VA protein levels in the spinal cord of AAV9-injected SMA mice and uninjected WT controls at P9. Tubulin is used as loading controls. c Quantification of four independent Western blot experiments. Data represent mean and SEM (n = 4). Statistical significance was determined by one-way ANOVA with Tukey’s post hoc test (adjusted P values: WT vs. SMA p < 0.0001; WT vs. SMA + GFP p < 0.0001; WT vs. SMA + SMN-WT p = 0.0032; WT vs. SMA + SMN-2VA p = 0.0063; SMA vs. SMA + GFP p = 0.9723; SMA vs. SMA + SMN-WT p < 0.0001; SMA vs. SMA + SMN-2VA p < 0.0001; SMA + GFP vs. SMA + SMN-WT p < 0.0001; SMA + GFP vs. SMA + SMN-2VA p < 0.0001; SMA + SMN-WT vs. SMA + SMN-2VA p = 0.9962). d–f Kaplan–Meyer analysis of survival (d; WT n = 15; SMA + GFP n = 16; SMA + SMN-WT n = 15, SMA + SMN-2VA n = 15), righting time (e; WT n = 15; SMA + GFP n = 13; SMA + SMN-WT n = 17, SMA + SMN-2VA n = 18), and weight gain (f; WT n = 15; SMA + GFP n = 18; SMA + SMN-WT n = 17, SMA + SMN-2VA n = 17). Data represent mean and SEM.