Past, present and future of myoblast transplantation in the treatment of Duchenne muscular dystrophy
Article first published online: 23 SEP 2010
© 2010 John Wiley & Sons A/S
Volume 14, Issue 7, pages 813–819, November 2010
How to Cite
Palmieri, B., Tremblay, J. P. and Daniele, L. (2010), Past, present and future of myoblast transplantation in the treatment of Duchenne muscular dystrophy. Pediatric Transplantation, 14: 813–819. doi: 10.1111/j.1399-3046.2010.01377.x
- Issue published online: 15 OCT 2010
- Article first published online: 23 SEP 2010
- Accepted for publication 25 June 2010
- Duchenne muscular dystrophy;
- exon skipping;
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.