There are a number of trinucleotide repeat (TNR) diseases that produce an expanded RNA transcript known to mediate the pathogenicity of the disease. This lecture will focus on the recent progress that our laboratory has made in targeting these unique RNAs using the principles of rational design. The talk will outline strategies to develop cell and nucleus permeable small molecules that selectively target the repeat RNA and DNA sequences associated with myotonic dystrophy (DM1 and DM2) and related ataxias. Efforts to achieve the goal of sequestering or degrading the RNA transcript have been at least partially successful. Whether these small molecules may serve as starting points for the development of therapeutic agents will be discussed.

Rational design of small molecules to target the DNA/RNA of trinucleotide repeat (TNR) diseases

249th National Meeting (2015)

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