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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Publicado en:ChemMedChem
Main Authors: Luu, Long M., Nguyen, Lien, Peng, Shaohong, Lee, JuYeon, Lee, Hyang Yeon, Wong, Chun-Ho, Hergenrother, Paul J., Chan, H. Y. Edwin, Zimmerman, Steven C.
Formato: Artigo
Idioma:Inglês
Publicado: 2016
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Article
Acceso en liña: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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