title: Immune-mediated pathology in Duchenne muscular dystrophy.
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.
authors.affiliation: Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Building 71/2238, Silver Spring, MD 20993, USA. amy.rosenberg@fda.hhs.gov.
authors.forename: Amy S
authors.initials: AS
authors.lastname: Rosenberg
authors.affiliation: Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Building 71/2238, Silver Spring, MD 20993, USA.
authors.forename: Montserrat
authors.initials: M
authors.lastname: Puig
authors.affiliation: Center for Genetic Medicine Research, Children's National Medical Center, Washington, DC 20010, USA.
authors.forename: Kanneboyina
authors.initials: K
authors.lastname: Nagaraju
authors.affiliation: Center for Genetic Medicine Research, Children's National Medical Center, Washington, DC 20010, USA.
authors.forename: Eric P
authors.initials: EP
authors.lastname: Hoffman
authors.affiliation: Department of Physiology and Biophysics, Institute for Immunology, University of California, Irvine, Irvine, CA 92697, USA.
authors.forename: S Armando
authors.initials: SA
authors.lastname: Villalta
authors.affiliation: Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Building 71/2238, Silver Spring, MD 20993, USA.
authors.forename: V Ashutosh
authors.initials: VA
authors.lastname: Rao
authors.affiliation: Laboratory of Cancer Biology and Genetics, National Cancer Institute, Building 37, Room 4032A, Bethesda, MD 20892, USA.
authors.forename: Lalage M
authors.initials: LM
authors.lastname: Wakefield
authors.affiliation: Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Building 71/2238, Silver Spring, MD 20993, USA.
authors.forename: Janet
authors.initials: J
authors.lastname: Woodcock
mesh_terms: D016207:Cytokines / Q000378:metabolism; D016189:Dystrophin / Q000378:metabolism; D005355:Fibrosis; D006801:Humans; D007113:Immunity, Innate; D007249:Inflammation; D020388:Muscular Dystrophy, Duchenne / Q000209:etiology / Q000276:immunology / Q000378:metabolism / Q000473:pathology; D015398:Signal Transduction; D055411:TGF-beta Superfamily Proteins / Q000378:metabolism
chemical_list: D016207:Cytokines; D016189:Dystrophin; D055411:TGF-beta Superfamily Proteins