Induction and Reversal of Myotonic Dystrophy Type 1 Pre-mRNA Splicing Defects by Small Molecules

The ability to control pre-mRNA splicing with small molecules could facilitate the development of therapeutics or cell-based circuits that control gene function. Myotonic dystrophy type 1 (DM1) is caused by the dysregulation of alternative pre-mRNA splicing due to sequestration of muscleblind-like 1...

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Main Authors: Childs-Disney, Jessica L., Stepniak-Konieczna, Ewa, Tran, Tuan, Yildirim, Ilyas, Park, HaJeung, Chen, Catherine Z., Hoskins, Jason, Southall, Noel, Marugan, Juan J., Patnaik, Samarjit, Zheng, Wei, Austin, Chris P., Schatz, George C., Sobczak, Krzysztof, Thornton, Charles A., Disney, Matthew D.
Format: Artigo
Sprog:Inglês
Udgivet: 2013
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3710115/
https://ncbi.nlm.nih.gov/pubmed/23806903
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/ncomms3044
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