Identifying Molecular Properties of Ataxin-2 Inhibitors for Spinocerebellar Ataxia Type 2 Utilizing High-Throughput Screening and Machine Learning

Spinocerebellar ataxia type 2 (SCA2) is an autosomal dominant neurodegenerative disorder marked by cerebellar dysfunction, ataxic gait, and progressive motor impairments. SCA2 is caused by the pathologic expansion of CAG repeats in the ataxin-2 (<i>ATXN2</i>) gene, leading to a toxic gai...

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Main Authors: Smita Sahay, Jingran Wen, Daniel R. Scoles, Anton Simeonov, Thomas S. Dexheimer, Ajit Jadhav, Stephen C. Kales, Hongmao Sun, Stefan M. Pulst, Julio C. Facelli, David E. Jones
Format: Article
Language:English
Published: MDPI AG 2025-05-01
Series:Biology
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Online Access:https://www.mdpi.com/2079-7737/14/5/522
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