Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 Mice

Spinocerebellar ataxia type 3 (SCA3) is a currently incurable neurodegenerative disorder caused by a CAG triplet expansion in exon 10 of the ATXN3 gene. The resultant expanded polyglutamine stretch in the mutant ataxin-3 protein causes a gain of toxic function, which eventually leads to neurodegener...

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Vydáno v:Mol Ther Nucleic Acids
Hlavní autoři: Toonen, Lodewijk J.A., Rigo, Frank, van Attikum, Haico, van Roon-Mom, Willeke M.C.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2017
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5504086/
https://ncbi.nlm.nih.gov/pubmed/28918024
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2017.06.019
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