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A Toxic RNA Catalyzes the Cellular Synthesis of Its Own Inhibitor, Shunting It to Endogenous Decay Pathways

  • Raphael I. Benhamou
  • , Alicia J. Angelbello
  • , Eric T. Wang
  • , Matthew D. Disney*
  • *Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

17 Scopus citations

Abstract

RNA repeat expansions cause >30 diseases, and both oligonucleotides and small molecules have been developed to inhibit their dysfunction. Benhamou et al. show for the first time that small molecules targeting structured, disease-causing RNAs can shunt them toward native decay pathways by affecting their processing.

Original languageEnglish
Pages (from-to)223-231.e4
JournalCell Chemical Biology
Volume27
Issue number2
DOIs
StatePublished - 20 Feb 2020
Externally publishedYes

Bibliographical note

Publisher Copyright:
© 2020 Elsevier Ltd

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

  1. SDG 3 - Good Health and Well-being
    SDG 3 Good Health and Well-being

Keywords

  • RNA
  • chemical biology
  • click chemistry
  • drug design
  • intron retention
  • medicinal chemistry
  • microsatellite disease
  • myotonic dystrophy
  • nucleic acids
  • repeat expansion disorder

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