Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression
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 E. Jad, Richard Raz, Matthew J. A. Wood, Miguel A. Varela, Beatriz Llamusí, Rubén Artero
Abstract
mice, intravenous injections of CPP-PMOs improve molecular, histopathological, and functional phenotypes, without signs of toxicity. Our findings place CPP-PMOs as promising antimiR candidates to overcome the treatment delivery challenge in DM1 therapy.
Topics & Concepts
MorpholinoRNA splicingMyotonic dystrophymicroRNARNACancer researchCell biologyAlternative splicingBiologyChemistryGeneMessenger RNAGene knockdownBiochemistryGeneticsGenetic Neurodegenerative DiseasesRNA Research and SplicingMuscle Physiology and Disorders