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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Auteurs principaux:	Irene González-Martínez, Estefanía Cerro-Herreros, Nerea Moreno, Andrea García-Rey, Jorge Espinosa-Espinosa, Marc Carrascosa-Sàez, Diego Piqueras-Losilla, Andrey Arzumanov, David Seoane-Miraz, Yahya Jad, Richard Raz, Matthew J. Wood, Miguel A. Varela, Beatriz Llamusí, Rubén Artero
Format:	Artigo
Langue:	Inglês
Publié:	Elsevier 2023-12-01
Collection:	Molecular Therapy: Nucleic Acids
Sujets:	MT: Oligonucleotides: Therapies and Applications cell-penetrating peptides MBNL proteins alternative splicing antisense oligonucleotides myotonic dystrophy
Accès en ligne:	http://www.sciencedirect.com/science/article/pii/S2162253123002378
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