Myotonic dystrophy type 1 (DM1) is a triplet repeat disorder wherein aberrant expansion of CTG repeats occurs within the DMPK gene.  The disease is believed to occur by an RNA gain of function mechanism wherein expanded CUG RNA recruits MBNL, leading to misregulation of alternative splicing of certain RNAs.  This talk will focus on the development of small molecules that selectively complex CTG or CUG repeats with high affinity and, in the case of CUG, inhibit MBNL binding.  Strategies for amplifying affinity and selectivity will be discussed as will attempts to determine the structure of the RNA/DNA small molecule complexes.

Molecular recognition of the CUG triplet repeats in RNA, the causitive agent of myotonic dystrophy

243rd National Meeting (2012)

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