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Small molecule modulation of splicing in SMA. (A) Schematic of SMN2 splicing. Inclusion of Exon 7 results in a functional SMN2 protein. (B) Structures of two documented SMN2 splicing modulators. (C) SMN-C5 (yellow) stabilizes an adenine residue (red) between Exon 7 and a spliceosomal U1 small nuclear ribonucleoprotein (snRNP), facilitating Exon 7 inclusion. SS: splice site. Adapted from ref. 37 with permission from Springer Nature, Copyright 2019.
