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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Auteurs principaux: Jahromi, Amin Haghighat, Nguyen, Lien, Fu, Yuan, Miller, Kali A., Baranger, Anne M., Zimmerman, Steven C.
Format: Artigo
Langue:Inglês
Publié: 2013
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Article
Accès en ligne: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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