Fig. 3
From: Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers
SMN-C3 and NVS-SM1 share a pharmacophore, but show different selectivity. a Pharmacophore model and alignment of SMN-C3 (blue) and NVS-SM1 (brown) with MOE2014.09. b, c RNA-seq analysis in type 1 SMA patient fibroblasts treated with SMN-C3 (500 nM) or NVS-SM1 (24 nM) for 24 h. b Transcriptome-wide splicing events for all treatment conditions with a ∆PSI of >0.4 (total of 42). c Transcriptome-wide gene expression changes significant at the p = 0.001 level in any of the treatment conditions (total of 313). Data in b and c represent mean values of 5 independent replicates. **p < 0.01, ***p < 0.001 as estimated by Student’s t test
