Aurintricarboxylic acid decreases rna toxicity in a c. Elegans model of repeat expansions

Maya Braun, Shachar Shoshani, Anna Mellul-Shtern, Yuval Tabach*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

1 Scopus citations


Pathologic expansions of DNA nucleotide tandem repeats may generate toxic RNA that triggers disease phenotypes. RNA toxicity is the hallmark of multiple expansion repeat disorders, including myotonic dystrophy type 1 (DM1). To date, there are no available disease-modifying therapies for DM1. Our aim was to use drug repositioning to ameliorate the phenotype of affected individuals in a nematode model of DM1. As the RNA interference pathway plays a key role in mediating RNA toxicity, we investigated the effect of aurintricarboxylic acid. We demonstrated that by perturbing the RNA interference machinery using aurintricarboxylic acid, we could annihilate the RNA toxicity and ameliorate the phenotype. As our approach targets a universal disease mechanism, it is potentially relevant for more expansion repeat disorders.

Original languageAmerican English
Article number910
Issue number12
StatePublished - 20 Dec 2021

Bibliographical note

Publisher Copyright:
© 2021 by the authors. Licensee MDPI, Basel, Switzerland.


  • C. elegans
  • CUG repeats
  • Drug repositioning
  • RNA interference
  • Small RNA pathway


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