Antisense oligonucleotide therapy for spinocerebellar ataxia type 2

Adult human neurodegenerative diseases have no disease-modifying treatments. We used spinocerebellar ataxia type 2 (SCA2), an autosomal dominant polyglutamine disease1, as a model to test RNA-targeted therapies(2) in two SCA2 mouse models. Both models recreate progressive adult-onset dysfunction and...

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Publicado en:Nature
Autores principales: Scoles, Daniel R., Meera, Pratap, Schneider, Matthew, Paul, Sharan, Dansithong, Warunee, Figueroa, Karla P., Hung, Gene, Rigo, Frank, Bennett, C. Frank, Otis, Thomas S., Pulst, Stefan M.
Formato: Artigo
Lenguaje:Inglês
Publicado: 2017
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Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC6625650/
https://ncbi.nlm.nih.gov/pubmed/28405024
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/nature22044
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