Article

Metformin decreases RAN proteins, rescues splicing abnormalities and improves behavioral phenotypes in SCA8 BAC mice

2025-08-25

Abstract excerpt

<h4>ABSTRACT</h4> Spinocerebellar ataxia type 8 is one member of a larger group of dominantly inherited, debilitating neurological diseases caused by CTG*CAG expansions for which there are no effective disease-targeting treatments. RAN translation, which was discovered in SCA8, has previously been shown to occur across CAG and CUG expansion transcripts, making treatments that work for SCA8 potentially relevant to...

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Literature Corpus work: d75f5443-8439-51a9-8533-63051f1222fc
DOI: 10.1101/2025.08.21.671563

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