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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...

Täydet tiedot

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Bibliografiset tiedot
Julkaisussa:Int J Mol Sci
Päätekijät: McIntosh, Craig S., Aung-Htut, May Thandar, Fletcher, Sue, Wilton, Steve D.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: MDPI 2019
Aiheet:
Linkit: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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