Allele-Specific CRISPR/Cas9 Correction of a Heterozygous DNM2 Mutation Rescues Centronuclear Myopathy Cell Phenotypes
Genome editing with the CRISPR/Cas9 technology has emerged recently as a potential strategy for therapy in genetic diseases. For dominant mutations linked to gain-of-function effects, allele-specific correction may be the most suitable approach. In this study, we tested allele-specific inactivation...
| Главные авторы: | , , , , , , |
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| Формат: | Статья |
| Язык: | English |
| Опубликовано: |
Elsevier
2019-06-01
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| Серии: | Molecular Therapy: Nucleic Acids |
| Online-ссылка: | http://www.sciencedirect.com/science/article/pii/S2162253119300502 |