DMD is a genetic X-linked recessive disease that affectsapproximately one in 3500 male births. Boys with DMD have progressiveand predictable muscle destruction because of the absence ofDys, a protein present under the muscle fiber membrane. Dys deficiencyinduces contraction-related membrane damages, activation of inflammatory-necrosis-fibrosis up to the cardiac-diaphragmatic failure anddeath. This review supports the therapeutic role of MT associated withimmunosuppression in DMD patients, describing the history and therationale of such approach. The authors underline the importance toevaluate a protocol of myoblast intradermal multi-injection to apply inyoung DMD patients.
Past, present and future of myoblasttransplantation in the treatment ofDuchenne muscular dystrophy / Palmieri, Beniamino; Tremblay, Jp; Daniele, L.. - In: PEDIATRIC TRANSPLANTATION. - ISSN 1397-3142. - ELETTRONICO. - 14:7(2010), pp. 813-819. [10.1111/j.1399-3046.2010.01377.x]
Past, present and future of myoblasttransplantation in the treatment ofDuchenne muscular dystrophy
PALMIERI, Beniamino;
2010
Abstract
DMD is a genetic X-linked recessive disease that affectsapproximately one in 3500 male births. Boys with DMD have progressiveand predictable muscle destruction because of the absence ofDys, a protein present under the muscle fiber membrane. Dys deficiencyinduces contraction-related membrane damages, activation of inflammatory-necrosis-fibrosis up to the cardiac-diaphragmatic failure anddeath. This review supports the therapeutic role of MT associated withimmunosuppression in DMD patients, describing the history and therationale of such approach. The authors underline the importance toevaluate a protocol of myoblast intradermal multi-injection to apply inyoung DMD patients.File | Dimensione | Formato | |
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