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Single Exon Skipping Can Address a Multi-Exon Duplication in the Dystrophin Gene
Duchenne muscular dystrophy (DMD) is a severe muscle wasting disease typically caused by protein-truncating mutations that preclude synthesis of a functional dystrophin. Exonic deletions are the most common type of DMD lesion, however, whole exon duplications account for between 10–15% of all report...
| Autores principales: | , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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MDPI
2020
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7350004/ https://www.ncbi.nlm.nih.gov/pubmed/32630425 http://dx.doi.org/10.3390/ijms21124511 |