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.
[Molecular Therapy-Methods & Clinical Development]
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