Figure 1.

ICV injection of scAAV9-SMN significantly increases the survival of SMNΔ7 mice when administered at early time points. (A) Kaplan–Meier survival curve of untreated SMNΔ7 mice and those injected with 1 × 10¹¹ v.g. of scAAV9-SMN on a single day P2–P8. Mice injected at the earliest time point P2 did not exhibit early deaths and they lived the longest with an average life span of 187 days and a median survival of 204 days. P3-injected mice lived up to 182 days (avg 102, median 75), the P4 group lived up to 211 days (avg 141, median 167), the P5 group lived up to 211 days (avg 76, median 37), the P6 group lived up to 211 days (avg 73, median 34), the P7 group lived up to 70 days (avg 30, median 28), the P8 group lived up to 22 days (avg 18, median 18) and the non-injected group lived up to 15 days (avg 13, median 14). (B) P-table demonstrating statistically significant differences in survival of mice in the treatment and non-treatment groups. All treated mice lived significantly longer than non-injected controls. P-values were calculated using the log-rank Mantel–Cox test and the table illustrates the significance in survival between groups. NI, non-injected. (n.s. no significance, *P ≤ 0.05, **P ≤ 0.01, ***P ≤ 0.001, ****P ≤ 0.0001).