Proof of concept of peptide-linked blockmiR-induced MBNL functional rescue in myotonic dystrophy type 1 mouse model

Myotonic dystrophy type 1 is a debilitating neuromuscular disease causing muscle weakness, myotonia, and cardiac dysfunction. The phenotypes are caused by muscleblind-like (MBNL) protein sequestration by toxic RNA in the DM1 protein kinase (DMPK) gene. DM1 patients exhibit a pathogenic number of rep...

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Detalles Bibliográficos
Autores principales: Overby, Sarah J., Cerro-Herreros, Estefanía, González-Martínez, Irene, Varela, Miguel A., Seoane-Miraz, David, Jad, Yahya, Raz, Richard, Møller, Thorleif, Pérez-Alonso, Manuel, Wood, Matthew J., Llamusí, Beatriz, Artero, Rubén
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8888893/
https://www.ncbi.nlm.nih.gov/pubmed/35282418
http://dx.doi.org/10.1016/j.omtn.2022.02.003

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