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Updated: Sep 8, 2025
07:08
Fractionation for Resolution of Soluble and Insoluble Huntingtin Species
Published on: February 27, 2018
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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
Summary
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.
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