Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy

Duchenne muscular dystrophy (DMD) is a severe, progressive muscle disease caused by mutations in the dystrophin gene. The majority of DMD mutations are deletions that prematurely terminate the dystrophin protein. Deletions of exon 50 of the dystrophin gene are among the most common single exon delet...

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Publicado en:Sci Transl Med
Main Authors: Amoasii, Leonela, Long, Chengzu, Li, Hui, Mireault, Alex A., Shelton, John M., Sanchez-Ortiz, Efrain, McAnally, John R., Bhattacharyya, Samadrita, Schmidt, Florian, Grimm, Dirk, Hauschka, Stephen D., Bassel-Duby, Rhonda, Olson, Eric N.
Formato: Artigo
Idioma:Inglês
Publicado: 2017
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC5749406/
https://ncbi.nlm.nih.gov/pubmed/29187645
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.aan8081
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