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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Main Authors: Irene González-Martínez (Author), Estefanía Cerro-Herreros (Author), Nerea Moreno (Author), Andrea García-Rey (Author), Jorge Espinosa-Espinosa (Author), Marc Carrascosa-Sàez (Author), Diego Piqueras-Losilla (Author), Andrey Arzumanov (Author), David Seoane-Miraz (Author), Yahya Jad (Author), Richard Raz (Author), Matthew J. Wood (Author), Miguel A. Varela (Author), Beatriz Llamusí (Author), Rubén Artero (Author)
Format: Book
Published: Elsevier, 2023-12-01T00:00:00Z.
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