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...

Full description

Bibliographic Details
Main Authors: Nelson, C. E., Hakim, C. H., Ousterout, D. G., Thakore, P. I., Moreb, E. A., Rivera, R. M. C., Madhavan, S., Pan, X., Ran, F. A., Yan, W. X., Asokan, A., Duan, D., Gersbach, C. A., Zhang, Feng
Other Authors: Massachusetts Institute of Technology. Department of Biological Engineering
Format: Article
Published: American Association for the Advancement of Science (AAAS) 2017
Online Access:http://hdl.handle.net/1721.1/112732
https://orcid.org/0000-0003-2782-2509

Similar Items

Similar Items