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

Pub. Date : 2019 Sep 6

PMID : 31394429






1 Functional Relationships(s)
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Compound Name
Protein Name
Organism
1 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 tract that confers toxic properties to the resulting proteins. polyglutamine ataxin 3 Mus musculus