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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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Principais autores: Craig S. McIntosh, Dunhui Li, Steve D. Wilton, May T. Aung-Htut
Format: Artigo
Sprog:Inglês
Udgivet: MDPI AG 2021-10-01
Serier:Biomedicines
Fag:
polyglutamine
ataxia
antisense oligonucleotides
expansion diseases
Online adgang:https://www.mdpi.com/2227-9059/9/11/1499
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