ReviewDuchenne Muscular Dystrophy

Immune-mediated pathology in Duchenne muscular dystrophy

Science Translational Medicine  05 Aug 2015:
Vol. 7, Issue 299, pp. 299rv4
DOI: 10.1126/scitranslmed.aaa7322

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Abstract

Immunological and inflammatory processes downstream of dystrophin deficiency as well as metabolic abnormalities, defective autophagy, and loss of regenerative capacity all contribute to muscle pathology in Duchenne muscular dystrophy (DMD). These downstream cascades offer potential avenues for pharmacological intervention. Modulating the inflammatory response and inducing immunological tolerance to de novo dystrophin expression will be critical to the success of dystrophin-replacement therapies. This Review focuses on the role of the inflammatory response in DMD pathogenesis and opportunities for clinical intervention.

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