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Cas9-induced single cut enables highly efficient and template-free repair of a muscular dystrophy causing founder mutation
With thousands of patients worldwide, CAPN3 c.550delA is the most frequent mutation causing severe, progressive, and untreatable limb girdle muscular dystrophy. We aimed to genetically correct this founder mutation in primary human muscle stem cells. We designed editing strategies providing CRISPR-C...
| Autores principales: | , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
American Society of Gene & Cell Therapy
2023
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9972404/ https://www.ncbi.nlm.nih.gov/pubmed/36865086 http://dx.doi.org/10.1016/j.omtn.2023.02.005 |