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.
2010
14
7
813
819
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]
Palmieri, Beniamino; Tremblay, Jp; Daniele, L.
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11380/716043
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