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Immune-mediated pathology in Duchenne muscular dystrophy

Year: 2015
Type: article
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.
DOI: 10.1126/scitranslmed.aaa7322
Authors: Amy S. Rosenberg, Montserrat Puig, Kanneboyina Nagaraju, Eric P. Hoffman, S. Armando Villalta, V. Ashutosh Rao, Lalage M. Wakefield, Janet Woodcock
Institutions: United States Food and Drug Administration, Center for Drug Evaluation and Research, Children's National, University of California, Irvine, National Institutes of Health, National Cancer Institute
Language: English
Open Access status: green
OpenAlex ID: W2116279332
FWCI: 5.401
Cites: 114
Cited by: 306
Related to: 10