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. Author manuscript; available in PMC: 2021 Apr 17.
Published in final edited form as: ACS Chem Biol. 2020 Mar 18;15(4):849–855. doi: 10.1021/acschembio.0c00036

Figure 1.

Figure 1.

Small-molecule cleavage of r(CUG)exp. (A) DM1 is caused by r(CUG)exp, located in the 3′ UTR of the DMPK gene, which forms a structure with repeating 1 × 1 U/U internal loops. The loops bind and sequester MBNL1, resulting in nuclear foci and pre-mRNA splicing defects. (B) MBNL1 protein regulates the splicing of its own pre-mRNA. When MBNL1 is sequestered by r(CUG)exp, its exon 5 is included too frequently. (C) Scheme of small-molecule cleavage of r(CUG)exp, resulting in improvement of DM1-associated defects.