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BlockmiR AONs as Site-Specific Therapeutic MBNL Modulation in Myotonic Dystrophy 2D and 3D Muscle Cells and HSA(LR) Mice

The symptoms of Myotonic Dystrophy Type 1 (DM1) are multi-systemic and life-threatening. The neuromuscular disorder is rooted in a non-coding CTG microsatellite expansion in the DM1 protein kinase (DMPK) gene that, upon transcription, physically sequesters the Muscleblind-like (MBNL) family of splic...

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Detalles Bibliográficos
Autores principales:	Overby, Sarah J., Cerro-Herreros, Estefanía, Espinosa-Espinosa, Jorge, González-Martínez, Irene, Moreno, Nerea, Fernández-Costa, Juan M., Balaguer-Trias, Jordina, Ramón-Azcón, Javier, Pérez-Alonso, Manuel, Møller, Thorleif, Llamusí, Beatriz, Artero, Rubén
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2023
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10141141/ https://www.ncbi.nlm.nih.gov/pubmed/37111604 http://dx.doi.org/10.3390/pharmaceutics15041118

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