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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. 10.

Fig. 10

Small molecule targeting of r(CCUG)exp, the causative agent of myotonic dystrophy type 2 (DM2). (A) r(CCUG)exp sequesters MBNL1. (B) MBNL1 regulates splicing of insulin receptor (IR) exon 11. Aberrant splicing results in exon 11 being excluded from the mature IR transcript, contributing to DM2 pathology. (C) Structures of the kanamycin RNA-binding motif and dimeric compound that bind r(CCUG)exp.