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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...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6862616/ https://www.ncbi.nlm.nih.gov/pubmed/31683630 http://dx.doi.org/10.3390/ijms20215434 |