Exon skipping induces uniform dystrophin rescue with dose-dependent restoration of serum miRNA biomarkers and muscle biophysical properties
<p>Therapies that restore dystrophin expression are presumed to correct Duchenne muscular dystrophy (DMD), with antisense-mediated exon skipping being the leading approach. Here we aimed to determine whether exon skipping using a peptide-phosphorodiamidate morpholino oligonucleotide (PPMO) con...
| Main Authors: | , , , , , , , , , |
|---|---|
| 格式: | Journal article |
| 语言: | English |
| 出版: |
Cell Press
2022
|