Article

rAAV-Delivered Bicistronic Artificial microRNAs for Allele-Specific Silencing Improve Motor and Molecular Outcomes in Spinocerebellar Ataxia Type 3

2025-10-02

Abstract excerpt

<h4>ABSTRACT</h4> Spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph disease, is an autosomal dominant neurodegenerative disorder caused by the expansion of CAG trinucleotide repeats in the ATXN3 gene. This mutation induces a toxic gain-of-function of the ATXN3 protein, leading to neurodegeneration, particularly in the cerebellum and brainstem. Despite extensive research, no disease-modifying tre...

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Literature Corpus work: 69e49ba5-449a-5c57-b8b1-b81cb0fea8e8
DOI: 10.1101/2025.10.02.680011

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