PPMO-mediated exon skipping induces uniform sarcolemmal dystrophin rescue with dose-dependent restoration of circulating microRNA biomarkers and muscle biophysical properties
Katarzyna Chwalenia, Jacopo Oieni, Joanna Zemła, Małgorzata Lekka, Nina Ahlskog, Anna M. L. Coenen-Stass, Graham McClorey, Matthew J. A. Wood, Yulia N. Lomonosova, Thomas C. Roberts
This study shows that PPMO-mediated exon skipping in mdx mice restores uniform dystrophin expression and dose-dependently improves serum microRNA biomarkers and muscle biophysical properties.
Molecular Therapy — Nucleic Acids · Aug 25, 2022Peer-reviewedMolecular Biology