Expanded DNA and RNA Trinucleotide Repeats in Myotonic Dystrophy Type 1 Select Their Own Multitarget, Sequence-Selective Inhibitors

Biochemistry. 2020 Sep 22;59(37):3463-3472. doi: 10.1021/acs.biochem.0c00472. Epub 2020 Sep 10.

Abstract

There are few methods available for the rapid discovery of multitarget drugs. Herein, we describe the template-assisted, target-guided discovery of small molecules that recognize d(CTG) in the expanded d(CTG·CAG) sequence and its r(CUG) transcript that cause myotonic dystrophy type 1. A positive cross-selection was performed using a small library of 30 monomeric alkyne- and azide-containing ligands capable of producing >5000 possible di- and trimeric click products. The monomers were incubated with d(CTG)16 or r(CUG)16 under physiological conditions, and both sequences showed selectivity in the proximity-accelerated azide-alkyne [3+2] cycloaddition click reaction. The limited number of click products formed in both selections and the even smaller number of common products suggests that this method is a useful tool for the discovery of single-target and multitarget lead therapeutic agents.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Cells, Cultured
  • DNA / antagonists & inhibitors*
  • DNA / genetics
  • DNA / metabolism
  • Humans
  • Myotonic Dystrophy / drug therapy*
  • Myotonic Dystrophy / genetics*
  • Myotonic Dystrophy / pathology
  • RNA / antagonists & inhibitors*
  • RNA / genetics
  • RNA / metabolism
  • Small Molecule Libraries / pharmacology*
  • Trinucleotide Repeat Expansion / drug effects*
  • Trinucleotide Repeat Expansion / genetics

Substances

  • Small Molecule Libraries
  • RNA
  • DNA