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. Author manuscript; available in PMC: 2021 Oct 5.
Published in final edited form as: Chem Soc Rev. 2020 Oct 5;49(19):7167–7199. doi: 10.1039/d0cs00560f

Fig. 5.

Fig. 5

Mode of action of small molecule splicing modulators targeting SMN2 pre-mRNA. (A) Structures of small molecule splicing modulators targeting SMN2 pre-mRNA and the derivatives used to study their mechanisms of action. (B) Schematic mechanism of small molecules facilitating SMN2 exon 7 inclusion by stabilizing the complex between SMN2 exon 7, the 5′ splice site (SS), and the U1 snRNP. (C) Schematic representation of 5′ splice site bulge repair mediated by risdiplam. (D) Competing modes of action proposed for risdiplam.