Defined d-hexapeptides bind CUG repeats and rescue phenotypes of myotonic dystrophy myotubes in a Drosophila model of the disease

Defined d-hexapeptides bind CUG repeats and rescue phenotypes of myotonic dystrophy myotubes in a Drosophila model of the disease

Abstract In Myotonic Dystrophy type 1 (DM1), a non-coding CTG repeats rare expansion disease; toxic double-stranded RNA hairpins sequester the RNA-binding proteins Muscleblind-like 1 and 2 (MBNL1 and 2) and trigger other DM1-related pathogenesis pathway defects. In this paper, we characterize four d...

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Autores principales: Anna Rapisarda, Ariadna Bargiela, Beatriz Llamusi, Isabel Pont, Roger Estrada-Tejedor, Enrique Garcia-España, Ruben Artero, Manuel Perez-Alonso
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2021
Materias:
Medicine
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Science
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Acceso en línea:https://doaj.org/article/0dbedd9c52a24137b9ee035f7dd3ec6c
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