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Huntington's Disease: Complex Pathogenesis and Therapeutic Strategies

Huichun Tong1, Tianqi Yang1, Shuying Xu1

  • 1Guangdong Key Laboratory of Non-Human Primate Research, Key Laboratory of CNS Regeneration (Ministry of Education), Guangdong-Hongkong-Macau Institute of CNS Regeneration, Jinan University, Guangzhou 510632, China.

International Journal of Molecular Sciences
|April 13, 2024
PubMed

Insights

Huntington's disease (HD) is caused by expanded CAG repeats in the huntingtin gene, leading to mutant huntingtin protein (mHTT). Current research focuses on therapies targeting mHTT toxicity and gene expression to combat this neurodegenerative disorder.

Area of Science:

  • Neuroscience
  • Genetics
  • Pharmacology

Background:

  • Huntington's disease (HD) is a neurodegenerative disorder caused by CAG repeat expansion in the huntingtin gene (HTT).
  • Mutant huntingtin protein (mHTT) forms aggregates, disrupting neuronal function and leading to disease pathology.
  • Current treatments for HD are limited, with no effective cure available.

Purpose of the Study:

  • To review current therapeutic strategies for Huntington's disease.
  • To discuss the mechanisms of mHTT toxicity and potential treatment targets.
  • To highlight the promise of gene therapy for reducing mHTT expression.

Main Methods:

  • Literature review of existing Huntington's disease treatments.
  • Analysis of therapeutic approaches targeting mHTT.
  • Discussion of gene therapy strategies for HD.

Main Results:

  • Significant progress has been made in developing diverse therapeutic strategies for HD.
  • Targeting mHTT neuronal toxicity and reducing mutant HTT gene expression show therapeutic promise.
  • Gene therapy approaches offer a promising avenue for effective HD treatment.

Conclusions:

  • While no cure exists, various therapeutic strategies are under development for Huntington's disease.
  • Reducing mutant huntingtin protein expression via gene therapy is a key focus for future treatments.
  • Continued research is essential for advancing Huntington's disease therapeutics.

Keywords:
Huntington’s diseaseaggregateshuntingtinpolyglutaminetherapy

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