Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system

Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disease caused by mutations in the dystrophin gene (DMD). Previously, we applied CRISPR-Cas9–mediated “single-cut” genome editing to correct diverse genetic mutations in animal models of DMD. However, high doses of adeno-associated virus (A...

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Veröffentlicht in:Sci Adv
Hauptverfasser: Zhang, Yu, Li, Hui, Min, Yi-Li, Sanchez-Ortiz, Efrain, Huang, Jian, Mireault, Alex A., Shelton, John M., Kim, Jiwoong, Mammen, Pradeep P. A., Bassel-Duby, Rhonda, Olson, Eric N.
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Association for the Advancement of Science 2020
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Research Articles
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7030925/
https://ncbi.nlm.nih.gov/pubmed/32128412
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/sciadv.aay6812
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