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...
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 |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2019-09-01
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Series: | Molecular Therapy: Nucleic Acids |
Online Access: | http://www.sciencedirect.com/science/article/pii/S2162253119301945 |
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