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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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Vydáno v:Int J Mol Sci
Hlavní autoři: McIntosh, Craig S., Aung-Htut, May Thandar, Fletcher, Sue, Wilton, Steve D.
Médium: Artigo
Jazyk:Inglês
Vydáno: MDPI 2019
Témata:
On-line přístup: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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