Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

CRISPR/Cas9-mediated genome editing holds clinical potential for treating genetic diseases, such as Duchenne muscular dystrophy (DMD), which is caused by mutations in the dystrophin gene. To correct DMD by skipping mutant dystrophin exons in postnatal muscle tissue in vivo, we used adeno-associated...

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Publicado no:Science
Main Authors: Long, Chengzu, Amoasii, Leonela, Mireault, Alex A., McAnally, John R., Li, Hui, Sanchez-Ortiz, Efrain, Bhattacharyya, Samadrita, Shelton, John M., Bassel-Duby, Rhonda, Olson, Eric N.
Formato: Artigo
Idioma:Inglês
Publicado em: 2015
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4760628/
https://ncbi.nlm.nih.gov/pubmed/26721683
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/science.aad5725
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