Bottom-up design of small molecules that stimulate exon 10 skipping in mutant MAPT pre-mRNA.

Abstract excerpt

One challenge in chemical biology is to develop small molecules that control cellular protein content. The amount and identity of proteins are influenced by the RNAs that encode them; thus, protein content in a cell could be affected by targeting mRNA. However, RNA has been traditionally difficult to target with small molecules. In this report, we describe controlling the protein products of the mutated...

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