Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules
This review, one in a series on myotonic dystrophy (DM), is focused on the development and potential use of small molecules as therapeutics for DM. The complex mechanisms and pathogenesis of DM are covered in the associated reviews. Here, we examine the various small molecule approaches taken to tar...
| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
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MDPI AG
2019-08-01
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| Series: | International Journal of Molecular Sciences |
| Subjects: | |
| Online Access: | https://www.mdpi.com/1422-0067/20/16/4017 |
| _version_ | 1811223159192420352 |
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| author | Kaalak Reddy Jana R. Jenquin John D. Cleary J. Andrew Berglund |
| author_facet | Kaalak Reddy Jana R. Jenquin John D. Cleary J. Andrew Berglund |
| author_sort | Kaalak Reddy |
| collection | DOAJ |
| description | This review, one in a series on myotonic dystrophy (DM), is focused on the development and potential use of small molecules as therapeutics for DM. The complex mechanisms and pathogenesis of DM are covered in the associated reviews. Here, we examine the various small molecule approaches taken to target the DNA, RNA, and proteins that contribute to disease onset and progression in myotonic dystrophy type 1 (DM1) and 2 (DM2). |
| first_indexed | 2024-04-12T08:28:16Z |
| format | Article |
| id | doaj.art-07bc9784ee99494da1cf11281293837a |
| institution | Directory Open Access Journal |
| issn | 1422-0067 |
| language | English |
| last_indexed | 2024-04-12T08:28:16Z |
| publishDate | 2019-08-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | International Journal of Molecular Sciences |
| spelling | doaj.art-07bc9784ee99494da1cf11281293837a2022-12-22T03:40:17ZengMDPI AGInternational Journal of Molecular Sciences1422-00672019-08-012016401710.3390/ijms20164017ijms20164017Mitigating RNA Toxicity in Myotonic Dystrophy using Small MoleculesKaalak Reddy0Jana R. Jenquin1John D. Cleary2J. Andrew Berglund3The RNA Institute, University at Albany–SUNY, Albany, NY 12222, USACenter for NeuroGenetics and Biochemistry & Molecular Biology, University of Florida, Gainesville, FL 32608, USAThe RNA Institute, University at Albany–SUNY, Albany, NY 12222, USAThe RNA Institute, University at Albany–SUNY, Albany, NY 12222, USAThis review, one in a series on myotonic dystrophy (DM), is focused on the development and potential use of small molecules as therapeutics for DM. The complex mechanisms and pathogenesis of DM are covered in the associated reviews. Here, we examine the various small molecule approaches taken to target the DNA, RNA, and proteins that contribute to disease onset and progression in myotonic dystrophy type 1 (DM1) and 2 (DM2).https://www.mdpi.com/1422-0067/20/16/4017myotonic dystrophysmall moleculestherapies |
| spellingShingle | Kaalak Reddy Jana R. Jenquin John D. Cleary J. Andrew Berglund Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules International Journal of Molecular Sciences myotonic dystrophy small molecules therapies |
| title | Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules |
| title_full | Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules |
| title_fullStr | Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules |
| title_full_unstemmed | Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules |
| title_short | Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules |
| title_sort | mitigating rna toxicity in myotonic dystrophy using small molecules |
| topic | myotonic dystrophy small molecules therapies |
| url | https://www.mdpi.com/1422-0067/20/16/4017 |
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