In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a devastating disease affecting about 1 out of 5000 male births and caused by mutations in the dystrophin gene. Genome editing has the potential to restore expression of a modified dystrophin gene from the native locus to modulate disease progression. In this stu...

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Main Authors: Nelson, C. E. (Author), Hakim, C. H. (Author), Ousterout, D. G. (Author), Thakore, P. I. (Author), Moreb, E. A. (Author), Rivera, R. M. C. (Author), Madhavan, S. (Author), Pan, X. (Author), Ran, F. A. (Author), Yan, W. X. (Author), Asokan, A. (Author), Duan, D. (Author), Gersbach, C. A. (Author), Zhang, Feng (Contributor)
Other Authors: 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), 2017-12-13T16:02:35Z.
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