Figure 1.

Schematic illustration of clustered regularly interspaced short palindromic repeats (CRISPR) editing in the ΔE50 canine Duchenne muscular dystrophy model. An adeno-associated virus (AAV) CRISPR-associated protein 9 vector and an AAV guide RNA vector were co-delivered to ΔE50 dogs by intramuscular or intravenous injection. In ΔE50 dogs, exon 50 is skipped in the RNA transcript by a naturally existing point mutation. This leads to frame-shift and dystrophin deficiency. CRISPR therapy restores dystrophin expression by reframing or skipping exon 51. Please note: immunostaining images are for illustration purposes. They are not from the Amoasii et al. publication.