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

15 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 languageAmerican English
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

Keywords

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

Fingerprint

Dive into the research topics of 'A Toxic RNA Catalyzes the Cellular Synthesis of Its Own Inhibitor, Shunting It to Endogenous Decay Pathways'. Together they form a unique fingerprint.

Cite this