Proof of concept of peptide-linked blockmiR-induced MBNL functional rescue in myotonic dystrophy type 1 mouse model
Sarah J. Overby, Estefanía Cerro-Herreros, Irene González-Martínez, Miguel A. Varela, David Seoane-Miraz, Yahya E. Jad, Richard Raz, Thorleif Møller, Manuel Pérez‐Alonso, Matthew J. A. Wood, Beatriz Llamusí, Rubén Artero
Abstract
mice. They also showed an increase in muscle strength and significant rescue of downstream splicing and histological phenotypes in mice without disturbing the endogenous levels of other miR-23b target transcripts.
Topics & Concepts
Myotonic dystrophyMyotoniaBiologyPhenotypeUntranslated regionRNA splicingCell biologymicroRNATrinucleotide repeat expansionThree prime untranslated regionTransgeneMessenger RNAGeneCancer researchInternal medicineRNAMedicineGeneticsAlleleGenetic Neurodegenerative DiseasesRNA Research and SplicingMuscle Physiology and Disorders