CRISPR-Cas9 homology-independent targeted integration of exons 1-19 restores full-length dystrophin in mice.
Journal Information
Full Title: Mol Ther Methods Clin Dev
Abbreviation: Mol Ther Methods Clin Dev
Country: Unknown
Publisher: Unknown
Language: N/A
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Subject Category: Medicine, Research & Experimental
Available in Europe PMC: Yes
Available in PMC: Yes
PDF Available: No
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"Declaration of interests A.A.S. and K.M.F. are inventors on a pending patent application filed by Nationwide Children’s Hospital for the gene editing system presented in this work."
"We wish to thank Dr. Robert B. Weiss of the University of Utah for his assistance in identifying candidate gRNAs. This study was supported by a Center of Research Translation Grant from the 10.13039/100000069National Institute of Arthritis and Musculoskeletal and Skin Diseases (P50AR070604). The Ohio State University Comprehensive Cancer Center Genomics Shared Resource is supported by an 10.13039/100000002NIH Center Core Grant (P30CA016058). Figures 2A, 3A, and 4A were created with BioRender.com."
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Last Updated: Aug 05, 2025