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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Main Authors: Tabebordbar, M. (Author), Zhu, K. (Author), Cheng, J. K. W. (Author), Chew, W. L. (Author), Widrick, J. J. (Author), Maesner, C. (Author), Wu, E. Y. (Author), Xiao, R. (Author), Ran, F. A. (Author), Vandenberghe, L. H. (Author), Church, G. M. (Author), Wagers, A. J. (Author), Yan, Winston Xia (Contributor), Cong, Le (Contributor), Zhang, Feng (Contributor)
Other Authors: Institute for Medical Engineering and Science (Contributor), Massachusetts Institute of Technology. Department of Biological Engineering (Contributor), Massachusetts Institute of Technology. Department of Brain and Cognitive Sciences (Contributor), McGovern Institute for Brain Research at MIT (Contributor)
Format: Article
Language:English
Published: American Association for the Advancement of Science (AAAS), 2017-12-13T16:10:42Z.
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