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

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, resulting in al...

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Detalles Bibliográficos
Autores principales: Maury, Yves, Poydenot, Pauline, Brinon, Benjamin, Lesueur, Lea, Gide, Jacqueline, Roquevière, Sylvain, Côme, Julien, Polvèche, Hélène, Auboeuf, Didier, Alexandre Denis, Jérome, Pietu, Geneviève, Furling, Denis, Lechuga, Marc, Baghdoyan, Sandrine, Peschanski, Marc, Martinat, Cécile
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6327858/
https://www.ncbi.nlm.nih.gov/pubmed/30639849
http://dx.doi.org/10.1016/j.isci.2018.12.019

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