Myoblast transplantation: a possible surgical treatment for a severe pediatric disease.
Duchenne muscular dystrophy (DMD) is a genetic X-linked recessive orphan disease that affects approximately 1 in 3 500 male births. Boys with DMD have progressive and predictable muscle destruction due to the absence of dystrophin, a protein present under the muscle fiber membrane. This absence induces contraction-related membrane damage and activation of inflammatory necrosis and fibrosis, leading to cardiac/diaphragmatic failure and death. The authors support the therapeutic role of myoblast transplantation in DMD, and describe the history and rationale for such an approach.
Palmieri
Beniamino
B
Department of General Surgery and Surgical Specialties, University of Modena and Reggio Emilia Medical School, Surgical Clinic, 41100 Modena, Italy.
Tremblay
Jacques P
JP
D017690:Cell Transplantation / Q000379:methods*; D002648:Child; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000175:diagnosis / Q000601:surgery*; D032446:Myoblasts / Q000637:transplantation*; D012720:Severity of Illness Index; D016896:Treatment Outcome