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Current Drug Targets - CNS & Neurological Disorders


ISSN (Print): 1568-007X
ISSN (Online): 1568-007X

Molecular Targets and Therapeutic Strategies in Huntingtons Disease

Author(s): A. C. Rego and L. P. de Almeida

Volume 4 , Issue 4 , 2005

Page: [361 - 381] Pages: 21

DOI: 10.2174/1568007054546081

Price: $65


This article provides an overview of the molecular mechanisms associated with striatal neuronal degeneration in Huntingtons disease (HD), the most studied of the diseases caused by polyglutamine expansion. We discuss the current status of research in cellular and animal models of HD, in which protein aggregation, excitotoxicity, mitochondrial dysfunction, transcription deregulation, trophic factor starvation and the disruption of axonal transport appear to be key features for selective striatal neurodegeneration. We further emphasize some of the most promising current strategies in HD treatment. We delineate the molecular and cellular rationale underlying the development of new pharmaceutical interventions that offer new hope of future treatment for HD patients worldwide.

Keywords: huntingtons disease, huntingtin, cag, neuronal death, protein aggregates, polyglutamine diseases, trinucleotide repeat diseases

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