| Autor: |
Katarzyna Chwalenia, Jacopo Oieni, Joanna Zemła, Małgorzata Lekka, Nina Ahlskog, Anna M.L. Coenen-Stass, Graham McClorey, Matthew J.A. Wood, Yulia Lomonosova, Thomas C. Roberts |
| Rok vydání: |
2022 |
| Předmět: |
|
| Zdroj: |
Molecular Therapy - Nucleic Acids. 29:955-968 |
| ISSN: |
2162-2531 |
| DOI: |
10.1016/j.omtn.2022.08.033 |
| Popis: |
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) conjugate results in dose-dependent restoration of uniform dystrophin localization, together with correction of putative DMD serum and muscle biomarkers. Dystrophin-deficient |
| Databáze: |
OpenAIRE |
| Externí odkaz: |
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