Small molecule targeting r(UGGAA) n disrupts RNA foci and alleviates disease phenotype in Drosophila model

Small molecule targeting r(UGGAA) n disrupts RNA foci and alleviates disease phenotype in Drosophila model

Synthetic small molecules modulating RNA structure and function have therapeutic potential for RNA diseases. Here the authors show the mechanism by which a small molecule targets the disease-causing r(UGGAA) n repeat RNAs in spinocerebellar ataxia type 31.

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Detalles Bibliográficos
Autores principales: Tomonori Shibata, Konami Nagano, Morio Ueyama, Kensuke Ninomiya, Tetsuro Hirose, Yoshitaka Nagai, Kinya Ishikawa, Gota Kawai, Kazuhiko Nakatani
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2021
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Science
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Acceso en línea:https://doaj.org/article/fb74152ab4824a57b44c57a7b829b312
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Sumario:Synthetic small molecules modulating RNA structure and function have therapeutic potential for RNA diseases. Here the authors show the mechanism by which a small molecule targets the disease-causing r(UGGAA) n repeat RNAs in spinocerebellar ataxia type 31.

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