DMD and Duchenne muscular dystrophy: A number of groups have shown efficient restoration of the dystrophin ORF through exon reframing in human iPSC-derived cardiac myocytes, mouse models, and large animal models of DMD (Ousterout et al., 2015; Amoasii et al., 2017; 2018; Bengtsson et al., 2017; Kyrychenko et al., 2017; Zhang et al., 2017; Long et al., 2018; Yuan et al., 2018).