Pluripotent Stem Cell-Based Drug Screening Reveals Cardiac Glycosides as Modulators of Myotonic Dystrophy Type 1

Pluripotent Stem Cell-Based Drug Screening Reveals Cardiac Glycosides as Modulators of Myotonic Dystrophy Type 1

Summary: There is currently no treatment for myotonic dystrophy type 1 (DM1), the most frequent myopathy of genetic origin. This progressive neuromuscular disease is caused by nuclear-retained RNAs containing expanded CUG repeats. These toxic RNAs alter the activities of RNA splicing factors, result...

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Bibliographic Details
Main Authors: Yves Maury, Pauline Poydenot, Benjamin Brinon, Lea Lesueur, Jacqueline Gide, Sylvain Roquevière, Julien Côme, Hélène Polvèche, Didier Auboeuf, Jérome Alexandre Denis, Geneviève Pietu, Denis Furling, Marc Lechuga, Sandrine Baghdoyan, Marc Peschanski, Cécile Martinat
Format: Article
Language:English
Published: Elsevier 2019-01-01
Series:iScience
Online Access:http://www.sciencedirect.com/science/article/pii/S2589004218302499

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http://www.sciencedirect.com/science/article/pii/S2589004218302499

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