Article
Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system.
Science advances - 1 Feb 2020
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
Abstract excerpt
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 (AAV) are required for efficient in vivo genome editing, posing challenges for clinical application. In this study, we...
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