A leading example is Duchenne muscular dystrophy, resulting from loss of dystrophin, which has been studied extensively with important translational insights (Long et al., 2018; Kamdar et al., 2020; Moretti et al., 2020; Pioner et al., 2020; Mekies et al., 2021). The gene discussed is DMD; the disease is Duchenne muscular dystrophy.