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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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Detalles Bibliográficos
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:	Online Artículo Texto
Lenguaje:	English
Publicado:	2017
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6625650/ https://www.ncbi.nlm.nih.gov/pubmed/28405024 http://dx.doi.org/10.1038/nature22044

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