Article
miR760 regulates ATXN1 levels via interaction with its 5' untranslated region.
Genes & development - 1 Sept 2020
Nitschke Larissa, Tewari Ambika, Coffin Stephanie L, Xhako Eder, Pang Kaifang, Gennarino Vincenzo A, Johnson Jennifer L, Blanco Francisco A, Liu Zhandong, Zoghbi Huda Y
Abstract excerpt
Identifying modifiers of dosage-sensitive genes involved in neurodegenerative disorders is imperative to discover novel genetic risk factors and potential therapeutic entry points. In this study, we focus on Ataxin-1 (ATXN1), a dosage-sensitive gene involved in the neurodegenerative disease spinocerebellar ataxia type 1 (SCA1). While the precise maintenance of ATXN1 levels is essential to prevent disease, the...
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