Specifically, Duchene et al. demonstrated that SaCas9 administration with two sgRNAs to a humanized mouse model of DMD (del52hDMD/mdx) can lead to DNA breaks in exons 47 and 58, restoration of the ORF, and synthesis of a functional dystrophin with normally phased spectrin-like repeats [161] (Fig. 6C). Here, DMD is linked to Duchenne muscular dystrophy.