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Targeting Expanded Repeats by Small Molecules in Repeat Expansion Disorders.

Masayuki Nakamori1, Hideki Mochizuki1

  • 1Department of Neurology, Osaka University Graduate School of Medicine, Osaka, Japan.

Movement Disorders : Official Journal of the Movement Disorder Society
|December 7, 2020
PubMed
Summary

New small molecules can target and shorten expanded DNA repeats, offering a potential treatment for neurodegenerative movement disorders like Huntington's disease. This approach addresses the root cause of repeat expansion disorders, aiming to prevent disease onset and alleviate symptoms.

Keywords:
Huntington's diseasenaphthyridine-azaquinolonenucleic acid-targeting small moleculerepeat instabilitytriplet repeat

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Area of Science:

  • Genetics
  • Neurodegenerative diseases
  • Molecular biology

Background:

  • Repeat expansion disorders are genetic conditions caused by abnormal genome sequence expansions.
  • Many of these disorders manifest as neurodegenerative movement disorders, with no current radical cures.
  • Existing treatments focus on abnormal mRNA and proteins, but targeting the causative DNA repeats is a novel strategy.

Purpose of the Study:

  • To explore therapeutic approaches targeting the root cause of repeat expansion disorders: the expanded DNA sequences.
  • To investigate the potential of small molecules in treating these genetic neurological conditions.

Main Methods:

  • Discovery and characterization of small molecules that interact with expanded DNA repeats.
  • Focus on small molecules binding to and shortening abnormally expanded CAG repeats, as seen in Huntington's disease.

Main Results:

  • Identification of a small molecule capable of binding to and shortening expanded CAG repeats.
  • Demonstration of a potential therapeutic mechanism targeting the DNA level of repeat expansion disorders.

Conclusions:

  • Small molecules targeting nucleic acids represent a promising therapeutic avenue for repeat expansion disorders.
  • These novel drugs could ameliorate symptoms and potentially prevent the onset of diseases like Huntington's disease.