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Removal of the Polyglutamine Repeat of Ataxin-3 by Redirecting pre-mRNA Processing

Spinocerebellar ataxia type 3 (SCA3) is a devastating neurodegenerative disease for which there is currently no cure, nor effective treatment strategy. One of nine polyglutamine disorders known to date, SCA3 is clinically heterogeneous and the main feature is progressive ataxia, which in turn affect...

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Publicado en:Int J Mol Sci
Autores principales: McIntosh, Craig S., Aung-Htut, May Thandar, Fletcher, Sue, Wilton, Steve D.
Formato: Artigo
Lenguaje:Inglês
Publicado: MDPI 2019
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Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC6862616/
https://ncbi.nlm.nih.gov/pubmed/31683630
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms20215434
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