CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells

Mutations in the dystrophin gene cause Duchenne muscular dystrophy (DMD), which is characterized by lethal degeneration of cardiac and skeletal muscles. Mutations that delete exon 44 of the dystrophin gene represent one of the most common causes of DMD and can be corrected in ~12% of patients by edi...

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Udgivet i:Sci Adv
Main Authors: Min, Yi-Li, Li, Hui, Rodriguez-Caycedo, Cristina, Mireault, Alex A., Huang, Jian, Shelton, John M., McAnally, John R., Amoasii, Leonela, Mammen, Pradeep P. A., Bassel-Duby, Rhonda, Olson, Eric N.
Format: Artigo
Sprog:Inglês
Udgivet: American Association for the Advancement of Science 2019
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Research Articles
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6402849/
https://ncbi.nlm.nih.gov/pubmed/30854433
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/sciadv.aav4324
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