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

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...

Full description

Bibliographic Details
Main Authors: Eleni Kourkouta, Rudie Weij, Anchel González-Barriga, Melissa Mulder, Ruurd Verheul, Sieto Bosgra, Bas Groenendaal, Jukka Puoliväli, Jussi Toivanen, Judith C.T. van Deutekom, Nicole A. Datson
Format: Article
Language:English
Published: Elsevier 2019-09-01
Series:Molecular Therapy: Nucleic Acids
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253119301945

Internet

http://www.sciencedirect.com/science/article/pii/S2162253119301945

Similar Items