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Exon-Skipping Oligonucleotides Restore Functional Collagen VI by Correcting a Common COL6A1 Mutation in Ullrich CMD
Collagen VI-related congenital muscular dystrophies (COL6-CMDs) are the second most common form of congenital muscular dystrophy. Currently, there is no effective treatment available. COL6-CMDs are caused by recessive or dominant mutations in one of the three genes encoding for the α chains of colla...
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| Publicat a: | Mol Ther Nucleic Acids |
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| Autors principals: | , , , , , , |
| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
American Society of Gene & Cell Therapy
2020
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| Matèries: | |
| Accés en línia: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7321786/ https://ncbi.nlm.nih.gov/pubmed/32585628 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2020.05.029 |
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