Polyglutamine Ataxias: Our Current Molecular Understanding and What the Future Holds for Antisense Therapies

Polyglutamine (polyQ) ataxias are a heterogenous group of neurological disorders all caused by an expanded CAG trinucleotide repeat located in the coding region of each unique causative gene. To date, polyQ ataxias encompass six disorders: spinocerebellar ataxia types 1, 2, 3, 6, 7, and 17 and accou...

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Auteurs principaux: Craig S. McIntosh, Dunhui Li, Steve D. Wilton, May T. Aung-Htut
Format: article
Langue:EN
Publié: MDPI AG 2021
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Accès en ligne:https://doaj.org/article/a2ca7d2f150f42ffa71be3091986a353
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