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.
| Main Authors: | , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Nature Portfolio
2021-01-01
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| Series: | Nature Communications |
| Online Access: | https://doi.org/10.1038/s41467-020-20487-4 |
| _version_ | 1818840205092716544 |
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| author | Tomonori Shibata Konami Nagano Morio Ueyama Kensuke Ninomiya Tetsuro Hirose Yoshitaka Nagai Kinya Ishikawa Gota Kawai Kazuhiko Nakatani |
| author_facet | Tomonori Shibata Konami Nagano Morio Ueyama Kensuke Ninomiya Tetsuro Hirose Yoshitaka Nagai Kinya Ishikawa Gota Kawai Kazuhiko Nakatani |
| author_sort | Tomonori Shibata |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-12-19T04:06:29Z |
| format | Article |
| id | doaj.art-fb74152ab4824a57b44c57a7b829b312 |
| institution | Directory Open Access Journal |
| issn | 2041-1723 |
| language | English |
| last_indexed | 2024-12-19T04:06:29Z |
| publishDate | 2021-01-01 |
| publisher | Nature Portfolio |
| record_format | Article |
| series | Nature Communications |
| spelling | doaj.art-fb74152ab4824a57b44c57a7b829b3122022-12-21T20:36:32ZengNature PortfolioNature Communications2041-17232021-01-0112111310.1038/s41467-020-20487-4Small molecule targeting r(UGGAA) n disrupts RNA foci and alleviates disease phenotype in Drosophila modelTomonori Shibata0Konami Nagano1Morio Ueyama2Kensuke Ninomiya3Tetsuro Hirose4Yoshitaka Nagai5Kinya Ishikawa6Gota Kawai7Kazuhiko Nakatani8Department of Regulatory Bioorganic Chemistry, The Institute of Scientific and Industrial Research (ISIR), Osaka UniversityDepartment of Life and Environmental Sciences, Faculty of Engineering, Chiba Institute of TechnologyDepartment of Neurotherapeutics, Osaka University Graduate School of MedicineGraduate School of Frontier Biosciences, Osaka UniversityGraduate School of Frontier Biosciences, Osaka UniversityDepartment of Neurotherapeutics, Osaka University Graduate School of MedicineCenter for Personalized Medicine for Healthy Aging, Tokyo Medical and Dental UniversityDepartment of Life and Environmental Sciences, Faculty of Engineering, Chiba Institute of TechnologyDepartment of Regulatory Bioorganic Chemistry, The Institute of Scientific and Industrial Research (ISIR), Osaka UniversitySynthetic 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.https://doi.org/10.1038/s41467-020-20487-4 |
| spellingShingle | Tomonori Shibata Konami Nagano Morio Ueyama Kensuke Ninomiya Tetsuro Hirose Yoshitaka Nagai Kinya Ishikawa Gota Kawai Kazuhiko Nakatani Small molecule targeting r(UGGAA) n disrupts RNA foci and alleviates disease phenotype in Drosophila model Nature Communications |
| title | Small molecule targeting r(UGGAA) n disrupts RNA foci and alleviates disease phenotype in Drosophila model |
| title_full | Small molecule targeting r(UGGAA) n disrupts RNA foci and alleviates disease phenotype in Drosophila model |
| title_fullStr | Small molecule targeting r(UGGAA) n disrupts RNA foci and alleviates disease phenotype in Drosophila model |
| title_full_unstemmed | Small molecule targeting r(UGGAA) n disrupts RNA foci and alleviates disease phenotype in Drosophila model |
| title_short | Small molecule targeting r(UGGAA) n disrupts RNA foci and alleviates disease phenotype in Drosophila model |
| title_sort | small molecule targeting r uggaa n disrupts rna foci and alleviates disease phenotype in drosophila model |
| url | https://doi.org/10.1038/s41467-020-20487-4 |
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