In vivo gene editing in dystrophic mouse muscle and muscle stem cells

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...

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Bibliographic Details
Main Authors: Tabebordbar, M., Zhu, K., Cheng, J. K. W., Chew, W. L., Widrick, J. J., Maesner, C., Wu, E. Y., Xiao, R., Ran, F. A., Vandenberghe, L. H., Church, G. M., Wagers, A. J., Yan, Winston Xia, Cong, Le, Zhang, Feng
Other Authors: Institute for Medical Engineering and Science
Format: Article
Published: American Association for the Advancement of Science (AAAS) 2017
Online Access:http://hdl.handle.net/1721.1/112733
https://orcid.org/0000-0002-3067-479X
https://orcid.org/0000-0003-2782-2509

Internet

http://hdl.handle.net/1721.1/112733
https://orcid.org/0000-0002-3067-479X
https://orcid.org/0000-0003-2782-2509

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