Combined treatment with peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) and AAV-U7 rescues the severe DMD phenotype in mice
Duchenne muscular dystrophy (DMD) is a devastating neuromuscular disease caused by an absence of the dystrophin protein, which is essential for muscle fiber integrity. Among the developed therapeutic strategies for DMD, exon skipping approach corrects the frame shift and partially restores dystrophi...
| Hlavní autoři: | , , , , , , , , , |
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| Médium: | Journal article |
| Jazyk: | English |
| Vydáno: |
Elsevier
2020
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