Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing

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 (DM...

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Udgivet i:Sci Adv
Main Authors: 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.
Format: Artigo
Sprog:Inglês
Udgivet: American Association for the Advancement of Science 2018
Fag:
Research Articles
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5796795/
https://ncbi.nlm.nih.gov/pubmed/29404407
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/sciadv.aap9004
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