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Huntington's chorea: emerging fields in therapeutics (Review)

Aisha Tahir1, Sania Jamal2, Usman Ali Shams3

  • 1Department of Biochemistry, University of Health Sciences, Khayaban-e- Jamia Punjab, 54600, Lahore, Punjab, Pakistan. dr.aisha@uhs.edu.pk.

Neurogenetics
|September 6, 2025
PubMed

Insights

Huntington's disease (HD) is a genetic neurodegenerative disorder. Emerging therapies aim to modify the disease by targeting mutant huntingtin protein, offering hope for a cure.

Area of Science:

  • Neuroscience
  • Genetics
  • Molecular Biology

Background:

  • Huntington's disease (HD) is a progressive neurodegenerative disorder.
  • It is characterized by motor, cognitive, and psychiatric symptoms.
  • HD stems from CAG repeat expansions in the HTT gene, leading to toxic mutant huntingtin protein aggregation.

Purpose of the Study:

  • To review the pathogenesis of Huntington's disease.
  • To discuss current and emerging therapeutic strategies for HD.
  • To highlight the shift towards disease-modifying interventions.

Main Methods:

  • Literature review of genetic, molecular, and environmental factors in HD pathogenesis.
  • Analysis of current management approaches.
  • Exploration of novel therapeutic strategies including RNA interference, antisense oligonucleotides (ASOs), peptide inhibitors, and CRISPR/Cas9 gene editing.

Main Results:

  • Identified key genetic, molecular, and environmental contributors to HD.
  • Detailed various management strategies.
  • Showcased promising disease-modifying therapies like ASOs and gene editing.

Conclusions:

  • Advancements in novel therapies signal a move towards modifying HD progression.
  • Despite progress, a definitive cure for Huntington's disease requires further research.
  • Continued clinical and translational research is crucial for developing effective treatments.

Keywords:
ASOCAG repeatCRISPRGene therapyHTT geneHuntington’s diseaseRNAi

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