Polyglutamine spinocerebellar ataxias-from genes to potential treatments

Henry L. Paulson, Vikram G. Shakkottai, H. Brent Clark, Harry T. Orr

Research output: Contribution to journalReview articlepeer-review

Abstract

The dominantly inherited spinocerebellar ataxias (SCAs) are a large and diverse group of neurodegenerative diseases. The most prevalent SCAs (SCA1, SCA2, SCA3, SCA6 and SCA7) are caused by expansion of a glutamine-encoding CAG repeat in the affected gene. These SCAs represent a substantial portion of the polyglutamine neurodegenerative disorders and provide insight into this class of diseases as a whole. Recent years have seen considerable progress in deciphering the clinical, pathological, physiological and molecular aspects of the polyglutamine SCAs, with these advances establishing a solid base from which to pursue potential therapeutic approaches.

Original languageEnglish (US)
Pages (from-to)613-626
Number of pages14
JournalNature Reviews Neuroscience
Volume18
Issue number10
DOIs
StatePublished - Sep 19 2017
Externally publishedYes

ASJC Scopus subject areas

  • Neuroscience(all)

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