A Consolidated Aav System for Single Cut CRISPR Correction of a Common Duchenne Muscular Dystrophy Mutation

Scientists devised a single cut gene editing method using a compact Staphylococcus aureus Cas9 to restore the open reading frame of exon 51, the most commonly affected out-of-frame exon in Duchenne muscular dystrophy. Editing of exon 51 in cardiomyocytes revealed a strong preference for exon reframing via a two nucleotide deletion.