Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy

Myotonic dystrophy type 1 (DM1) is a rare neuromuscular disease caused by expansion of unstable CTG repeats in a non-coding region of the DMPK gene. CUG expansions in mutant DMPK transcripts sequester MBNL1 proteins in ribonuclear foci. Depletion of this protein is a primary contributor to disease s...

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Detalles Bibliográficos
Autores principales: Cerro-Herreros, Estefanía, González-Martínez, Irene, Moreno, Nerea, Espinosa-Espinosa, Jorge, Fernández-Costa, Juan M., Colom-Rodrigo, Anna, Overby, Sarah J., Seoane-Miraz, David, Poyatos-García, Javier, Vilchez, Juan J., López de Munain, Adolfo, Varela, Miguel A., Wood, Matthew J., Pérez-Alonso, Manuel, Llamusí, Beatriz, Artero, Rubén
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8413838/
https://www.ncbi.nlm.nih.gov/pubmed/34513303
http://dx.doi.org/10.1016/j.omtn.2021.07.017

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8413838/
https://www.ncbi.nlm.nih.gov/pubmed/34513303
http://dx.doi.org/10.1016/j.omtn.2021.07.017

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