Figure 2.

scAAV9-STMN1 treatment increases lifespan and birth-to-peak weight gain in SMA mice. Phenotypic analyses in SMA mice after ICV injection of 1.0 × 10¹¹ scAAV9-STMN1 viral injection at P2. (A) Survival was significantly increased (P < 0.001) in treated SMA mice compared to untreated SMA, healthy controls survived past 50 days. (B) Average weight gain was not different in treated SMA mice compared to untreated SMA up to day 25. (C) Birth-to-peak weight gain was significantly increased (P = 0.0486) in treated SMA mice compared to untreated SMA. (D) Representative image showing health pup at P25 and improvement in overt appearance of treated SMN mice compared to untreated SMA mice. N = 8, healthy control; n = 13, untreated SMA; n = 17, treated SMA. Data expressed as mean ± SEM.