Centenary of the UCT Faculty of Health Sciences
Polyglutamine disease: from pathogenesis to therapy
Abstract
An understanding of polyglutamine disease mechanisms is critical for development of novel therapeutics. Here we summarise theories of molecular pathogenesis, and examine ways in which this knowledge is being harnessed for therapy, with reference to work under way at the University of Cape Town. Despite a plethora of preclinical data, clinical trials of therapies for polyglutamine diseases have had only limited success. However, recently initiated trials, including those using gene silencing approaches, should provide valuable insights into the safety and efficacy of therapies directly targeting polyglutamine pathogenesis. This is particularly relevant in the South African context, where the frequencies of two polyglutamine diseases, spinocerebellar ataxia types 1 and 7, are among the highest globally.
Authors' affiliations
Lauren M Watson, University of Cape Town, South Africa and University of Oxford, UK
Janine Scholefield, University of Oxford, UK, and CSIR, South Africa
L Jacquie Greenberg, National Health Laboratory Services and Faculty of Health Sciences, University of Cape Town
Matthew JA Wood, University of Oxford, UK, and University of Cape Town, South Africa
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Article History
Date published: 2012-03-02
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