A Humanized Knock-In Mouse Model of Duchenne Muscular Dystrophy and Its Correction by CRISPR-Cas9 Therapeutic Gene Editing

Systemic CRISPR-Cas9 gene editing using an single guide RNAs (sgRNAs) that targeted human exon 51 efficiently restored dystrophin expression and ameliorated pathologic hallmarks of Duchenne muscular dystrophy, including histopathology and grip strength in a mouse model.