ASO silencing reverses abnormal neurochemistry in spinocerebellar ataxia 3 mice.

SCA3 mouse cerebellar and brainstem neurochemical trends parallel those in patients with SCA3. Decreased total choline may reflect oligodendrocyte abnormalities, decreased total N-acetylaspartate highlights neuronal health disturbances, and high glutamine may indicate gliosis. ASO treatment fully or partially reversed select neurochemical abnormalities in SCA3 mice, indicating the potential for these measures to serve as non-invasive treatment biomarkers in future SCA3 gene silencing trials. This article is protected by copyright. All rights reserved.