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Aurintricarboxylic Acid Decreases RNA Toxicity in a C. elegans Model of Repeat Expansions

Maya Braun1, Shachar Shoshani1, Anna Mellul-Shtern1

  • 1Department of Developmental Biology and Cancer Research, Institute for Medical Research Israel-Canada, Hebrew University of Jerusalem, Jerusalem 91120, Israel.

Toxins
|December 23, 2021
PubMed

Insights

Aurintricarboxylic acid can reduce toxic RNA in a model of myotonic dystrophy type 1 (DM1). This drug repositioning approach targets RNA toxicity, offering potential new therapies for DM1 and similar repeat expansion disorders.

Area of Science:

  • Genetics
  • Molecular Biology
  • Pharmacology

Background:

  • Pathologic expansions of DNA nucleotide tandem repeats can lead to toxic RNA.
  • RNA toxicity is a key mechanism in repeat expansion disorders like myotonic dystrophy type 1 (DM1).
  • Currently, no disease-modifying therapies exist for DM1.

Purpose of the Study:

  • To explore drug repositioning for ameliorating DM1 phenotypes.
  • To investigate the role of the RNA interference (RNAi) pathway in DM1 pathogenesis.
  • To evaluate the therapeutic potential of aurintricarboxylic acid (ATA) in a DM1 nematode model.

Main Methods:

  • Utilized a nematode model of myotonic dystrophy type 1.
  • Investigated the effect of aurintricarboxylic acid on the RNA interference pathway.
  • Assessed the impact of ATA on RNA toxicity and disease phenotype.

Main Results:

  • Aurintricarboxylic acid effectively perturbed the RNA interference machinery.
  • ATA treatment annihilated RNA toxicity in the DM1 model.
  • The drug ameliorated the disease phenotype in affected nematodes.

Conclusions:

  • Drug repositioning of aurintricarboxylic acid shows promise for treating DM1.
  • Targeting the RNA interference pathway offers a potential therapeutic strategy for RNA toxicity.
  • This approach may be applicable to other expansion repeat disorders.
Keywords:
C. elegansCUG repeatsRNA interferencedrug repositioningsmall RNA pathway

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