Fig. 7
From: Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers

Working model of the proposed molecular interactions of the SMN-C type SMN2 splicing modifiers. SMN-C type compounds bind to two sites at the SMN2 exon 7, namely ESE2 and 5′ss when U1 snRNA is bound. Whereas interaction with the 5′ss drives promotion of exon 7 inclusion in the splicing process, binding to ESE2 results in hnRNP G dislocation and allows the U1 snRNP complex to bind to ESE2. Both binding processes in combination provide high selectivity to the SMN2 and STRN3 pre-mRNAs over other genes