Article
CRISPR/Cas correction of muscular dystrophies.
Experimental cell research - 1 Nov 2021
Zhang Yu, Nishiyama Takahiko, Olson Eric N, Bassel-Duby Rhonda
Abstract excerpt
Muscular dystrophies are a heterogeneous group of monogenic neuromuscular disorders which lead to progressive muscle loss and degeneration of the musculoskeletal system. The genetic causes of muscular dystrophies are well characterized, but no effective treatments have been developed so far. The discovery and application of the CRISPR/Cas system for genome editing offers a new path for disease treatment with the...
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