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A quantitative high-throughput screen identifies compounds that lower expression of the SCA2-and ALS-associated gene ATXN2
CAG repeat expansions in the ATXN2 (ataxin-2) gene can cause the autosomal dominant disorder spinocerebellar ataxia type 2 (SCA2) as well as increase the risk of ALS. Abnormal molecular, motor, and neurophysiological phenotypes in SCA2 mouse models are normalized by lowering ATXN2 transcription, and...
Autores principales: | Scoles, Daniel R., Gandelman, Mandi, Paul, Sharan, Dexheimer, Thomas, Dansithong, Warunee, Figueroa, Karla P., Pflieger, Lance T., Redlin, Scott, Kales, Stephen C., Sun, Hongmao, Maloney, David, Damoiseaux, Robert, Henderson, Mark J., Simeonov, Anton, Jadhav, Ajit, Pulst, Stefan M. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society for Biochemistry and Molecular Biology
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9356275/ https://www.ncbi.nlm.nih.gov/pubmed/35787375 http://dx.doi.org/10.1016/j.jbc.2022.102228 |
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