In this context, the mdx mouse has been the most widely used murine model of DMD and has contributed over the last forty years to greatly advancing the understanding of the role of dystrophin and in developing some promising approaches [12,13,14,15,16] However, the mdx mouse suffers from the common limitations of animal-based translational research, such as high costs, long timelines, ethical concerns related to the large number of animals required, and low throughput [17]. The gene discussed is DMD; the disease is Duchenne muscular dystrophy.