Journal
JOURNAL OF THE AMERICAN CHEMICAL SOCIETY
Volume 130, Issue 48, Pages 16254-16261Publisher
AMER CHEMICAL SOC
DOI: 10.1021/ja804398y
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Funding
- NIAMS NIH HHS [T32 AR007472, K24 AR048143, T32 AR007472-19, T32AR007472] Funding Source: Medline
- NINDS NIH HHS [U54 NS048843, 08U54NS048843-05, NS58345, R21 NS058345] Funding Source: Medline
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Myotonic dystrophy type 1 (DM1), the most common form of muscular dystrophy in adults, is an RNA-mediated disease. Dramatically expanded (CUG) repeats accumulate in nuclei and sequester RNA-binding proteins such as the splicing regulator MBNL1. We have employed resin-bound dynamic combinatorial chemistry (RBDCC) to identify the first examples of compounds able to inhibit MBNL1 binding to (CUG) repeat RNA, Screening an RBDCL with a theoretical diversity of 11 325 members yielded several molecules with significant selectivity for binding to (CUG) repeat RNA over other sequences. These compounds were also able to inhibit the interaction of GGG-(CLlG)(109)-GGG RNA with MBNL1 in vitro, with K-i values in the low micromolar range.
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