Article

ASOs are an effective treatment for disease-associated oligodendrocyte signatures in premanifest and symptomatic SCA3 mice

2022-07-19

Abstract excerpt

<h4>ABSTRACT</h4> Spinocerebellar ataxia type 3 (SCA3) is the most common dominantly inherited ataxia. Currently, no preventative or disease-modifying treatments exist for this progressive neurodegenerative disorder, although efforts using gene silencing approaches are under clinical trial investigation. The disease is caused by a CAG repeat expansion in the mutant gene, ATXN3 , producing an enlarged polyglutami...

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Literature Corpus work: 47de99ed-eb59-5196-9c1b-922f9817bdfb
DOI: 10.1101/2022.07.18.500473

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