Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression

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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Bibliographic Details
Main Authors: 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: Article
Language:English
Published: Elsevier 2023-12-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Oligonucleotides: Therapies and Applications
cell-penetrating peptides
MBNL proteins
alternative splicing
antisense oligonucleotides
myotonic dystrophy
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253123002378

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http://www.sciencedirect.com/science/article/pii/S2162253123002378

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