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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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2018
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5796795/
https://www.ncbi.nlm.nih.gov/pubmed/29404407
http://dx.doi.org/10.1126/sciadv.aap9004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5796795/
https://www.ncbi.nlm.nih.gov/pubmed/29404407
http://dx.doi.org/10.1126/sciadv.aap9004

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