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Therapeutic Strategies for Huntington's Disease
Ladislav Mrzljak1, Ignacio Munoz-Sanjuan
1CHDI Management/CHDI Foundation Inc., Los Angeles, CA, USA.
Current Topics in Behavioral Neurosciences
|November 27, 2013
Summary
Huntington's disease (HD) is a genetic neurodegenerative disorder. This review examines current preclinical and clinical therapeutic strategies, including small molecules and gene therapies, for treating HD patients.
Area of Science:
- Neuroscience
- Genetics
- Pharmacology
Background:
- Huntington's disease (HD) is an autosomal dominant neurodegenerative disorder.
- It results from a CAG repeat expansion in the huntingtin (HTT) gene.
- Pathologically, HD involves neuronal loss in cortical, striatal, and hypothalamic areas.
Purpose of the Study:
- To review the current landscape of therapeutic development for Huntington's disease.
- To cover both preclinical and clinical advancements.
- To discuss small molecule and molecular therapies.
Main Methods:
- Literature review of preclinical and clinical studies.
- Analysis of therapeutic strategies for Huntington's disease.
- Focus on small molecules and molecular therapies.
Main Results:
- Multiple therapeutic avenues are under investigation for HD.
- Small molecules and gene-targeting therapies show promise.
- Clinical trials are evaluating the safety and efficacy of various treatments.
Conclusions:
- Therapeutic development for HD is progressing across multiple fronts.
- A combination of approaches may be necessary for effective treatment.
- Continued research is crucial for addressing this devastating disease.
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