Abstract
Huntingtons disease (HD) is a dominantly inherited neurodegenerative disorder caused by expansion of a polyglutamine (polyQ) tract in the huntingtin protein, resulting in intracellular aggregate formation and neurodegeneration. Biochemical pathways leading from polyQ expansion to disease pathogenesis are largely unknown. Recent approaches using genetic models systems have begun to uncover nuclear and cytoplasmic pathologies that represent potential targets for therapeutic intervention.
Keywords: Axonal transport, neurodegeneration, vesicle trafficking, protein aggregation, polyQ, huntington's disease
Mini-Reviews in Medicinal Chemistry
Title: Therapeutic Prospects for the Prevention of Neurodegeneration in Huntingtons Disease and the Polyglutamine Repeat Disorders
Volume: 7 Issue: 1
Author(s): Yoko Kimura, Wyan-Ching Mimi Lee and J. Troy Littleton
Affiliation:
Keywords: Axonal transport, neurodegeneration, vesicle trafficking, protein aggregation, polyQ, huntington's disease
Abstract: Huntingtons disease (HD) is a dominantly inherited neurodegenerative disorder caused by expansion of a polyglutamine (polyQ) tract in the huntingtin protein, resulting in intracellular aggregate formation and neurodegeneration. Biochemical pathways leading from polyQ expansion to disease pathogenesis are largely unknown. Recent approaches using genetic models systems have begun to uncover nuclear and cytoplasmic pathologies that represent potential targets for therapeutic intervention.
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Cite this article as:
Kimura Yoko, Mimi Lee Wyan-Ching and Troy Littleton J., Therapeutic Prospects for the Prevention of Neurodegeneration in Huntingtons Disease and the Polyglutamine Repeat Disorders, Mini-Reviews in Medicinal Chemistry 2007; 7 (1) . https://dx.doi.org/10.2174/138955707779317867
DOI https://dx.doi.org/10.2174/138955707779317867 |
Print ISSN 1389-5575 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5607 |
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