1. Effect of 14 hit compounds on SMN protein expression in type I SMA patient fibroblast cells (GM03813).
Effect of 14 hit compounds on SMN protein expression in type I SMA patient fibroblast cells (GM03813). GM03813 fibroblast cells were treated with potential molecules. (A, B) Western blot (A) and quantification data (B) showing that compound #3, #4, #8, and #10 could significantly increase the SMN protein level, among which compound #8 was most significant. n = 4. Data were presented as mean ± SEM, n = 4. *P < 0.05, as compared with the DMSO group by two-tailed t test.
Yiran Wang et al. LSA 2019;2:e
© 2019 Wang et al.