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Updated: Feb 5, 2026
Whole-brain Segmentation and Change-point Analysis of Anatomical Brain MRI—Application in Premanifest Huntington's Disease
Published on: June 9, 2018
Therapeutic approaches to Huntington disease: from the bench to the clinic
Nicholas S Caron1, E Ray Dorsey2, Michael R Hayden1,3,4
1Centre for Molecular Medicine and Therapeutics (CMMT), University of British Columbia, Vancouver, British Columbia, Canada.
Abstract:
The 25 years since the identification of the gene responsible for Huntington disease (HD) have stood witness to profound discoveries about the nature of the disease and its pathogenesis. Despite this progress, however, the development of disease-modifying therapies has thus far been slow. Preclinical validation of the therapeutic potential of disrupted pathways in HD has led to the advancement of pharmacological agents, both novel and repurposed, for clinical evaluation. The most promising therapeutic approaches include huntingtin (HTT) lowering and modification as well as modulation of neuroinflammation and synaptic transmission. With clinical trials for many of these approaches imminent or currently ongoing, the coming years are promising not only for HD but also for more prevalent neurodegenerative disorders, such as Alzheimer and Parkinson disease, in which many of these pathways have been similarly implicated.
Insights
Huntington disease (HD) research has advanced understanding of its pathogenesis. Promising therapies targeting huntingtin (HTT) lowering and neuroinflammation are in clinical trials, offering hope for HD and similar neurodegenerative diseases.
Area of Science:
- Neuroscience
- Genetics
- Pharmacology
Background:
- Huntington disease (HD) is a neurodegenerative disorder with a known genetic cause.
- Despite 25 years of research since gene identification, disease-modifying therapies remain limited.
Purpose of the Study:
- To review progress in understanding HD pathogenesis.
- To highlight promising therapeutic strategies and their clinical advancement.
Main Methods:
- Review of preclinical findings and ongoing clinical trials.
- Focus on therapeutic targets such as huntingtin (HTT) lowering and modulation of neuroinflammation and synaptic transmission.
Main Results:
- Significant discoveries made regarding HD nature and pathogenesis.
- Several novel and repurposed pharmacological agents are in clinical evaluation.
Conclusions:
- Therapeutic approaches like HTT lowering and neuroinflammation modulation show promise.
- Advancements in HD therapies may benefit other neurodegenerative diseases like Alzheimer's and Parkinson's.
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