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  • RESEARCH HIGHLIGHT

Base editing tackles trinucleotide repeat diseases

By
  1. Sarah Crunkhorn

Many neurological diseases are caused by pathogenic unstable intragenic trinucleotide repeat (TNR) expansions, such as the GAA trinucleotide repeat within the FXN gene in Friedreich ataxia (FRDA) and the CAG repeat in the HTT gene in Huntington disease. There are no approved treatments that halt or reverse any TNR disease.

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Nature Reviews Drug Discovery 24, 588 (2025)

doi: https://doi.org/10.1038/d41573-025-00110-1

References

  1. Matuszek, Z. et al. Base editing of trinucleotide repeats that cause Huntington’s disease and Friedreich’s ataxia reduces somatic repeat expansions in patient cells and in mice. Nat. Genet. https://doi.org/10.1038/s41588-025-02172-8 (2025)

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