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

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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Autori principali:	Cerro-Herreros, E, González-Martínez, I, Moreno, N, Espinosa-Espinosa, J, Fernández-Costa, JM, Colom-Rodrigo, A, Overby, SJ, Seoane-Miraz, D, Poyatos-García, J, Vilchez, JJ, López de Munain, A, Varela, MA, Wood, MJ, Pérez-Alonso, M, Llamusí, B, Artero, R
Natura:	Journal article
Lingua:	English
Pubblicazione:	Cell Press 2021

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