In vivo gene editing in dystrophic mouse muscle and muscle stem cells
Frame-disrupting mutations in the DMD gene, encoding dystrophin, compromise myofiber integrity and drive muscle deterioration in Duchenne muscular dystrophy (DMD). Removing one or more exons from the mutated transcript can produce an in-frame mRNA and a truncated, but still functional, protein. In t...
Main Authors: | , , , , , , , , , , , , , , |
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Other Authors: | |
Format: | Article |
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American Association for the Advancement of Science (AAAS)
2017
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Online Access: | http://hdl.handle.net/1721.1/112733 https://orcid.org/0000-0002-3067-479X https://orcid.org/0000-0003-2782-2509 |