Premium
Past, present and future of myoblast transplantation in the treatment of Duchenne muscular dystrophy
Author(s) -
Palmieri Beniamino,
Tremblay Jacques P.,
Daniele Lodi
Publication year - 2010
Publication title -
pediatric transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.457
H-Index - 69
eISSN - 1399-3046
pISSN - 1397-3142
DOI - 10.1111/j.1399-3046.2010.01377.x
Subject(s) - medicine , duchenne muscular dystrophy , transplantation , dystrophin , immunosuppression , myocyte , muscular dystrophy , bridge to transplantation , heart transplantation
Palmieri B, Tremblay JP, Lodi D. Past, present and future of myoblast transplantation in the treatment of Duchenne muscular dystrophy.
Pediatr Transplantation 2010: 14:813–819. © 2010 John Wiley & Sons A/S. 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.