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...

Descripción completa

Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6695277/
https://www.ncbi.nlm.nih.gov/pubmed/31394429
http://dx.doi.org/10.1016/j.omtn.2019.07.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6695277/
https://www.ncbi.nlm.nih.gov/pubmed/31394429
http://dx.doi.org/10.1016/j.omtn.2019.07.004

Ejemplares similares