Antisense oligonucleotide-mediated exon skipping as a strategy to reduce proteolytic cleavage of ataxin-3

Spinocerebellar ataxia type-3 (SCA3) is a neurodegenerative disorder caused by a polyglutamine repeat expansion in the ataxin-3 protein. Cleavage of mutant ataxin-3 by proteolytic enzymes yields ataxin-3 fragments containing the polyglutamine stretch. These shorter ataxin-3 fragments are thought to...

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Detalles Bibliográficos
Autores principales: Toonen, Lodewijk J. A., Schmidt, Iris, Luijsterburg, Martijn S., van Attikum, Haico, van Roon-Mom, Willeke M. C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5059676/
https://www.ncbi.nlm.nih.gov/pubmed/27731380
http://dx.doi.org/10.1038/srep35200

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5059676/
https://www.ncbi.nlm.nih.gov/pubmed/27731380
http://dx.doi.org/10.1038/srep35200

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