Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model
Duchenne muscular dystrophy (DMD) is a progressive X-linked disease caused by mutations in the DMD gene that prevent the expression of a functional dystrophin protein. Exon duplications represent 6%–11% of mutations, and duplications of exon 2 (Dup2) are the most common (∼11%) of duplication mutatio...
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| Principais autores: | , , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Elsevier
2023-12-01
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| coleção: | Molecular Therapy: Methods & Clinical Development |
| Assuntos: | |
| Acesso em linha: | http://www.sciencedirect.com/science/article/pii/S2329050123001833 |
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