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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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Detalles Bibliográficos
Publicado en: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.
Formato: Artigo
Idioma:Inglês
Publicado: American Association for the Advancement of Science 2019
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Acceso en liña: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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