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A Novel CUG(exp)·MBNL1 Inhibitor with Therapeutic Potential for Myotonic Dystrophy Type 1
Myotonic dystrophy type 1 (DM1) is caused by an expanded CUG repeat (CUG(exp)) that sequesters muscleblind-like 1 protein (MBNL1), a protein that regulates alternative splicing. CUG(exp) RNA is a validated drug target for this currently untreatable disease. Herein, we develop a bioactive small molec...
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| Main Authors: | , , , , , |
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| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
2013
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3683132/ https://ncbi.nlm.nih.gov/pubmed/23480597 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/cb400046u |
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