Duchenne muscular dystrophy (DMD) is a geneticX-linked recessive orphan disease that affects approximately1 in 3 500 male births. Boys with DMD haveprogressive and predictable muscle destruction due tothe absence of dystrophin, a protein present under themuscle fi ber membrane. This absence induces contraction-related membrane damage and activation ofinfl ammatory necrosis and fi brosis, leading to cardiac/diaphragmatic failure and death. The authors supportthe therapeutic role of myoblast transplantation inDMD, and describe the history and rationale for suchan approach.
MYOBLAST TRANSPLANTATION: A POSSIBLE SURGICAL TREATMENT FOR A SEVERE PEDIATRIC DISEASE / Palmieri, Beniamino; Tremblay, Jp. - In: SURGERY TODAY. - ISSN 0941-1291. - ELETTRONICO. - 40:(2010), pp. 902-908. [10.1007/s00595-009-4242-z]
MYOBLAST TRANSPLANTATION: A POSSIBLE SURGICAL TREATMENT FOR A SEVERE PEDIATRIC DISEASE
PALMIERI, Beniamino;
2010
Abstract
Duchenne muscular dystrophy (DMD) is a geneticX-linked recessive orphan disease that affects approximately1 in 3 500 male births. Boys with DMD haveprogressive and predictable muscle destruction due tothe absence of dystrophin, a protein present under themuscle fi ber membrane. This absence induces contraction-related membrane damage and activation ofinfl ammatory necrosis and fi brosis, leading to cardiac/diaphragmatic failure and death. The authors supportthe therapeutic role of myoblast transplantation inDMD, and describe the history and rationale for suchan approach.
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