Past, present and future of myoblast transplantation in the treatment of Duchenne muscular dystrophy

Abstract

DMD is a genetic X-linked recessive disease that affects approximately one in 3500 male births. Boys with DMD have progressive and predictable muscle destruction because of the absence of Dys, a protein present under the muscle fiber membrane. Dys deficiency induces contraction-related membrane damages, activation of inflammatory-necrosis-fibrosis up to the cardiac-diaphragmatic failure and death. This review supports the therapeutic role of MT associated with immunosuppression in DMD patients, describing the history and the rationale of such approach. The authors underline the importance to evaluate a protocol of myoblast intradermal multi-injection to apply in young DMD patients