Molecular Targets and Therapeutic Strategies in Huntingtons Disease

Title: Molecular Targets and Therapeutic Strategies in Huntingtons Disease

Volume: 4 Issue: 4

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

Affiliation:

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

Abstract: 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.

Cite this article as:

Rego C. A. and de Almeida P. L., Molecular Targets and Therapeutic Strategies in Huntingtons Disease, Current Drug Targets - CNS & Neurological Disorders 2005; 4 (4) . https://dx.doi.org/10.2174/1568007054546081