Article

AAV-mediated allele-specific silencing alleviates neuropathology in a novel non-human primate model of Spinocerebellar ataxia type 3

2025-10-02

Abstract excerpt

Spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph disease (MJD), is an autosomal dominant neurodegenerative disorder caused by an abnormal expansion of the cytosine-adenine-guanine (CAG) repeats in the ATXN3 gene. This mutation results in the production of an Ataxin-3 protein with an extended polyglutamine sequence, contributing to the disease’s neuropathology. Currently, no treatment is availabl...

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Literature Corpus work: 66e82b13-08ae-510d-a41d-c23bacfeb978
DOI: 10.1101/2025.10.02.680027

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