Figure 4.

Regulation of actin cytoskeleton pathway in fibroblasts from SMA patients and family‐matched controls. (A) ACTB, RHOA, and ROCK1 mRNA expression in two independent cell lines with four technical replicates from independent experiments for each line. (B) Transient SMN knockdown in healthy fibroblasts using a complex of SMN siRNA and lipofectamine, n = 12 technical replicates. (C) ACTB, RHOA, and ROCK1 mRNA expression in fibroblasts from SMA patients after SMN replacement using an AAV2 vector, n = 4 replicates from independent experiments. Unpaired (A) or paired (B and C) student’s t tests were used to compare groups. *P ≤ 0.05; **P ≤ 0.01; ****P ≤ 0.0001