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...
| Hlavní autoři: | , , , , , , , , , |
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| Médium: | Journal article |
| Jazyk: | English |
| Vydáno: |
Cell Press
2022
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