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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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Detalles Bibliográficos
Autores principales: McIntosh, Craig S., Li, Dunhui, Wilton, Steve D., Aung-Htut, May T.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8615177/
https://www.ncbi.nlm.nih.gov/pubmed/34829728
http://dx.doi.org/10.3390/biomedicines9111499