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

• Glavni autori: Irene González-Martínez (Autor), Estefanía Cerro-Herreros (Autor), Nerea Moreno (Autor), Andrea García-Rey (Autor), Jorge Espinosa-Espinosa (Autor), Marc Carrascosa-Sàez (Autor), Diego Piqueras-Losilla (Autor), Andrey Arzumanov (Autor), David Seoane-Miraz (Autor), Yahya Jad (Autor), Richard Raz (Autor), Matthew J. Wood (Autor), Miguel A. Varela (Autor), Beatriz Llamusí (Autor), Rubén Artero (Autor)
• Format: Knjiga
• Izdano: Elsevier, 2023-12-01T00:00:00Z.
• Teme: article
• Online pristup: Connect to this object online.

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...