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

• DOI: 10.1126/sciadv.aay6812
• Publication Date: 2020-02-20T00:19:53Z
• Authors: Yu Zhang; Hui Li; Yi-Li Min; Efrain Sanchez-Ortiz; Jian Huang; Alex A. Mireault; John M. Shelton; Jiwoong Kim; Pradeep P. A. Mammen; Rhonda Bassel-Duby; Eric N. Olson
• Keywords: mdx mouse

Self-complementary AAV-packaged CRISPR-Cas9 genome editing components rescue Duchenne muscular dystrophy.