Oligonucleotide therapy mitigates disease in Spinocerebellar Ataxia Type 3 mice

OBJECTIVE: Spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph disease, is the most common dominantly inherited ataxia. Despite advances in understanding this CAG repeat/polyglutamine expansion disease, there are still no therapies to alter its progressive fatal course. Here we invest...

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Bibliografiske detaljer
Udgivet i:Ann Neurol
Main Authors: McLoughlin, Hayley S., Moore, Lauren R., Chopra, Ravi, Komlo, Robert, McKenzie, Megan, Blumenstein, Kate G., Zhao, Hien, Kordasiewicz, Holly B., Shakkottai, Vikram G., Paulson, Henry L.
Format: Artigo
Sprog:Inglês
Udgivet: 2018
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6119475/
https://ncbi.nlm.nih.gov/pubmed/29908063
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ana.25264
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