To show restoration of the dystrophin protein in the diaphragm and cardiac muscle and functional improvement of the pathology in DMD, especially for the cardiorespiratory system in a preclinical study, as previously reported in mdx mice [44], the establishment of a new mouse model, such as a mouse with deletion of exon 44 of the dystrophin gene or a humanized dystrophin gene [45], is required. The gene discussed is DMD; the disease is Duchenne muscular dystrophy.