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Pharmaceutical, cellular and genetic therapies for Huntington's disease

Handley, Olivia J., Naji, Jenny J., Dunnett, Stephen Bruce ORCID: and Rosser, Anne Elizabeth ORCID: 2006. Pharmaceutical, cellular and genetic therapies for Huntington's disease. Clinical Science 110 (1) , pp. 73-88. 10.1042/CS20050148

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HD (Huntington's disease) is a devastating neurodegenerative disorder caused by a polyglutamine expansion in the gene encoding the huntingtin protein. Presently, there is no known cure for HD and existing symptomatic treatments are limited. However, recent advances have identified multiple pathological mechanisms involved in HD, some of which have now become the focus of therapeutic intervention. In this review, we consider progress made towards developing safe and effective pharmaceutical-, cell- and genetic-based therapies, and discuss the extent to which some of these therapies have been successfully translated into clinical trials. These new prospects offer hope for delaying and possibly halting this debilitating disease.

Item Type: Article
Date Type: Publication
Status: Published
Schools: Biosciences
MRC Centre for Neuropsychiatric Genetics and Genomics (CNGG)
Neuroscience and Mental Health Research Institute (NMHRI)
Publisher: Biochemical Society
ISSN: 0143-5221
Last Modified: 27 Oct 2022 08:28

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