Likewise, different stem cell-based strategies aim to replenish the muscle stem cell pool with dystrophin-competent cells as a potential therapy for DMD: while a clinical trial using HLA-matched donor mesoangioblasts failed to show any functional improvements12, recent preclinical studies are focused on autologous transplantation of corrected stem cells13,14. The gene discussed is DMD; the disease is Duchenne muscular dystrophy.