Article
Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing.
Science advances - 1 Jan 2018
Long Chengzu, Li Hui, Tiburcy Malte, Rodriguez-Caycedo Cristina, Kyrychenko Viktoriia, Zhou Huanyu, Zhang Yu, Min Yi-Li, Shelton John M, Mammen Pradeep P A, Liaw Norman Y, Zimmermann Wolfram-Hubertus, Bassel-Duby Rhonda, Schneider Jay W, Olson Eric N
Abstract excerpt
Genome editing with CRISPR/Cas9 is a promising new approach for correcting or mitigating disease-causing mutations. Duchenne muscular dystrophy (DMD) is associated with lethal degeneration of cardiac and skeletal muscle caused by more than 3000 different mutations in the X-linked dystrophin gene (DMD). Most of these mutations are clustered in "hotspots." There is a fortuitous correspondence between the eukaryotic...
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