Full-Length Dystrophin Restoration via Targeted Genomic Integration by AAV-CRISPR in a Humanized Mouse Model of Duchenne Muscular Dystrophy

Researchers co-delivered CRISPR-Cas9 and a donor DNA sequence to insert the missing human exon 52 into its corresponding position within the Duchenne muscular dystrophy gene and achieved full-length dystrophin correction in skeletal and cardiac muscle.