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Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression

Myotonic dystrophy type 1 (DM1) is a rare neuromuscular disease caused by a CTG repeat expansion in the DMPK gene that generates toxic RNA with a myriad of downstream alterations in RNA metabolism. A key consequence is the sequestration of alternative splicing regulatory proteins MBNL1/2 by expanded...

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Detalles Bibliográficos
Autores principales:	González-Martínez, Irene, Cerro-Herreros, Estefanía, Moreno, Nerea, García-Rey, Andrea, Espinosa-Espinosa, Jorge, Carrascosa-Sàez, Marc, Piqueras-Losilla, Diego, Arzumanov, Andrey, Seoane-Miraz, David, Jad, Yahya, Raz, Richard, Wood, Matthew J., Varela, Miguel A., Llamusí, Beatriz, Artero, Rubén
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2023
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10514136/ https://www.ncbi.nlm.nih.gov/pubmed/37744174 http://dx.doi.org/10.1016/j.omtn.2023.09.001

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