Unstable microsatellite DNA repeats and their expanded RNA transcripts have emerged as primary targets for small molecule intervention in several incurable diseases. In myotonic dystrophy type 1 and 2 (DM1 and DM2), the sequences are the tri- and tetra-nucleotide repeats (CTG)n and (CCTG)n in DNA and (CUG)n and (CCUG)n in RNA. This talk will focus on several approaches to the design and synthesis of agents that selectively recognize the nucleotide repeats that cause DM1 and DM2. In some cases, a combination of structure-based and computer-aided drug design, and target-based selection have successfully led to hits with good selectivity for inhibiting one or multiple pathways in the disease pathobiology.

DNA and RNA in multi-target drug design for the microsatellite disease myotonic dystrophy

251st National Meeting (2016)

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