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...

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Main Authors: Eleni Kourkouta (Author), Rudie Weij (Author), Anchel González-Barriga (Author), Melissa Mulder (Author), Ruurd Verheul (Author), Sieto Bosgra (Author), Bas Groenendaal (Author), Jukka Puoliväli (Author), Jussi Toivanen (Author), Judith C.T. van Deutekom (Author), Nicole A. Datson (Author)
Format: Book
Published: Elsevier, 2019-09-01T00:00:00Z.
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3rd Floor Main Library

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