Duchenne Muscular Dystrophy Cell Culture Models Created by CRISPR/Cas9 Gene Editing and Their Application in Drug Screening

The authors optimized a CRISPR/Cas9 gene edition protocol and used it in wild type immortalised myoblasts to delete exon 52 of the dystrophin gene, modeling a common Duchenne muscular dystrophy mutation; and in patient’s immortalised cultures they deleted an inhibitory microRNA target region of the utrophin UTR, leading to utrophin upregulation.