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A Potent Inhibitor of Protein Sequestration by Expanded Triplet (CUG) Repeats that Shows Phenotypic Improvements in a Drosophila Model of Myotonic Dystrophy
Myotonic dystrophy is the most common form of the adult-onset muscular dystrophy, originating in a CTG repeat expansion in the DMPK gene. The expanded CUG transcript sequesters MBNL1 a key regulator of alternative splicing, leading to the misregulation of numerous pre-mRNAs. We report an RNA-targete...
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| Τόπος έκδοσης: | ChemMedChem |
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| Κύριοι συγγραφείς: | , , , , , , , , |
| Μορφή: | Artigo |
| Γλώσσα: | Inglês |
| Έκδοση: |
2016
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| Θέματα: | |
| Διαθέσιμο Online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5074844/ https://ncbi.nlm.nih.gov/pubmed/27245480 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/cmdc.201600081 |
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