Molecular BiologyMolecular Therapy — Nucleic AcidsPeer-reviewed
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…·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.
21 citations