Suppression of Mutant Protein Expression in SCA3 and SCA1 Mice Using a CAG Repeat-Targeting Antisense Oligonucleotide

Spinocerebellar ataxia type 3 (SCA3) and type 1 (SCA1) are dominantly inherited neurodegenerative disorders that are currently incurable. Both diseases are caused by a CAG-repeat expansion in exon 10 of the Ataxin-3 and exon 8 of the Ataxin-1 gene, respectively, encoding an elongated polyglutamine t...

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Publicado no:Mol Ther Nucleic Acids
Main Authors: Kourkouta, Eleni, Weij, Rudie, González-Barriga, Anchel, Mulder, Melissa, Verheul, Ruurd, Bosgra, Sieto, Groenendaal, Bas, Puoliväli, Jukka, Toivanen, Jussi, van Deutekom, Judith C.T., Datson, Nicole A.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2019
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6695277/
https://ncbi.nlm.nih.gov/pubmed/31394429
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2019.07.004
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