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...
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| Main Authors: | , , , , , , |
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| Format: | Book |
| Published: |
Elsevier,
2019-06-01T00:00:00Z.
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| Online Access: | Connect to this object online. |
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