Drug Screen Tugs at Common Thread for Repeat Disorders

Trends Pharmacol Sci. 2020 Feb;41(2):71-73. doi: 10.1016/j.tips.2019.12.004. Epub 2020 Jan 8.


Repeat-associated non-ATG (RAN) translation is emerging as a driver of pathogenesis in microsatellite expansion disorders. Green and colleagues recently identified several candidate RAN translation inhibitors from a high-throughput small-molecule screen for fragile X tremor ataxia syndrome. Their study establishes a path forward for identifying inhibitors of RAN translation for multiple disorders.

Keywords: RAN translation; RNA binding; multiple targets; therapeutic compound screen.

Publication types

  • Comment

MeSH terms

  • Ataxia / genetics
  • Fragile X Mental Retardation Protein* / genetics
  • Fragile X Mental Retardation Protein* / metabolism
  • Fragile X Syndrome* / drug therapy
  • Fragile X Syndrome* / genetics
  • Humans
  • Tremor / drug therapy
  • Tremor / genetics
  • Trinucleotide Repeat Expansion


  • Fragile X Mental Retardation Protein