Literature DB >> 26246170

Immune-mediated pathology in Duchenne muscular dystrophy.

Amy S Rosenberg1, Montserrat Puig2, Kanneboyina Nagaraju3, Eric P Hoffman3, S Armando Villalta4, V Ashutosh Rao2, Lalage M Wakefield5, Janet Woodcock2.   

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.
Copyright © 2015, American Association for the Advancement of Science.

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Year:  2015        PMID： 26246170      PMCID： PMC5951380          DOI： 10.1126/scitranslmed.aaa7322

Source DB:  PubMed          Journal:  Sci Transl Med        ISSN： 1946-6234            Impact factor:   17.956


  109 in total

Review 1.  Pleiotropic functions of the CXC-type chemokine CXCL14 in mammals.

Authors:  Takahiko Hara; Kosuke Tanegashima
Journal:  J Biochem       Date:  2012-03-20       Impact factor: 3.387

2.  Cromolyn administration (to block mast cell degranulation) reduces necrosis of dystrophic muscle in mdx mice.

Authors:  Hannah G Radley; Miranda D Grounds
Journal:  Neurobiol Dis       Date:  2006-06-23       Impact factor: 5.996

3.  Activation of AKT signaling promotes cell growth and survival in α7β1 integrin-mediated alleviation of muscular dystrophy.

Authors:  Marni D Boppart; Dean J Burkin; Stephen J Kaufman
Journal:  Biochim Biophys Acta       Date:  2011-01-07

4.  Osteopontin expression in normal and fibrotic liver. altered liver healing in osteopontin-deficient mice.

Authors:  Dionne Lorena; Ian A Darby; Alain-Pierre Gadeau; Laetitia Lam Shang Leen; Susan Rittling; Luís C Porto; Jean Rosenbaum; Alexis Desmoulière
Journal:  J Hepatol       Date:  2005-08-15       Impact factor: 25.083

5.  Upregulation of chemokines and their receptors in Duchenne muscular dystrophy: potential for attenuation of myofiber necrosis.

Authors:  Boel De Paepe; Kim K Creus; Jean-Jacques Martin; Jan L De Bleecker
Journal:  Muscle Nerve       Date:  2012-12       Impact factor: 3.217

Review 6.  Targeting the TGFβ signalling pathway in disease.

Authors:  Rosemary J Akhurst; Akiko Hata
Journal:  Nat Rev Drug Discov       Date:  2012-09-24       Impact factor: 84.694

7.  Cyclosporin A corrects mitochondrial dysfunction and muscle apoptosis in patients with collagen VI myopathies.

Authors:  Luciano Merlini; Alessia Angelin; Tania Tiepolo; Paola Braghetta; Patrizia Sabatelli; Alessandra Zamparelli; Alessandra Ferlini; Nadir M Maraldi; Paolo Bonaldo; Paolo Bernardi
Journal:  Proc Natl Acad Sci U S A       Date:  2008-03-24       Impact factor: 11.205

8.  T and B lymphocyte depletion has a marked effect on the fibrosis of dystrophic skeletal muscles in the scid/mdx mouse.

Authors:  A Farini; M Meregalli; M Belicchi; M Battistelli; D Parolini; G D'Antona; M Gavina; L Ottoboni; G Constantin; R Bottinelli; Y Torrente
Journal:  J Pathol       Date:  2007-10       Impact factor: 7.996

9.  TGF-β signalling and reactive oxygen species drive fibrosis and matrix remodelling in myxomatous mitral valves.

Authors:  Michael A Hagler; Thomas M Hadley; Heyu Zhang; Kashish Mehra; Carolyn M Roos; Hartzell V Schaff; Rakesh M Suri; Jordan D Miller
Journal:  Cardiovasc Res       Date:  2013-04-03       Impact factor: 10.787

10.  IL-6 signaling blockade increases inflammation but does not affect muscle function in the mdx mouse.

Authors:  Matthew C Kostek; Kanneboyina Nagaraju; Emidio Pistilli; Arpana Sali; San-Huei Lai; Brad Gordon; Yi-Wen Chen
Journal:  BMC Musculoskelet Disord       Date:  2012-06-20       Impact factor: 2.362
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  86 in total

1.  Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug.

Authors:  Laurie S Conklin; Jesse M Damsker; Eric P Hoffman; William J Jusko; Panteleimon D Mavroudis; Benjamin D Schwartz; Laurel J Mengle-Gaw; Edward C Smith; Jean K Mah; Michela Guglieri; Yoram Nevo; Nancy Kuntz; Craig M McDonald; Mar Tulinius; Monique M Ryan; Richard Webster; Diana Castro; Richard S Finkel; Andrea L Smith; Lauren P Morgenroth; Adrienne Arrieta; Maya Shimony; Mark Jaros; Phil Shale; John M McCall; Yetrib Hathout; Kanneboyina Nagaraju; John van den Anker; Leanne M Ward; Alexandra Ahmet; Michaelyn R Cornish; Paula R Clemens
Journal:  Pharmacol Res       Date:  2018-09-13       Impact factor: 7.658

2.  Splenic Ly6Chi monocytes are critical players in dystrophic muscle injury and repair.

Authors:  Giuseppe Rizzo; Rosanna Di Maggio; Anna Benedetti; Jacopo Morroni; Marina Bouche; Biliana Lozanoska-Ochser
Journal:  JCI Insight       Date:  2020-01-30

3.  Morpholino-induced exon skipping stimulates cell-mediated and humoral responses to dystrophin in mdx mice.

Authors:  Maria C Vila; James S Novak; Margaret Benny Klimek; Ning Li; Melissa Morales; Alexander G Fritz; Katie Edwards; Jessica F Boehler; Marshall W Hogarth; Travis B Kinder; Aiping Zhang; Davi Mazala; Alyson A Fiorillo; Bonnie Douglas; Yi-Wen Chen; John van den Anker; Qi L Lu; Yetrib Hathout; Eric P Hoffman; Terence A Partridge; Kanneboyina Nagaraju
Journal:  J Pathol       Date:  2019-04-16       Impact factor: 7.996

4.  Proteomic profiling of the dystrophin complex and membrane fraction from dystrophic mdx muscle reveals decreases in the cytolinker desmoglein and increases in the extracellular matrix stabilizers biglycan and fibronectin.

Authors:  Sandra Murphy; Heinrich Brinkmeier; Mirjam Krautwald; Michael Henry; Paula Meleady; Kay Ohlendieck
Journal:  J Muscle Res Cell Motil       Date:  2017-08-12       Impact factor: 2.698

5.  Biological scaffold-mediated delivery of myostatin inhibitor promotes a regenerative immune response in an animal model of Duchenne muscular dystrophy.

Authors:  Kenneth M Estrellas; Liam Chung; Lindsay A Cheu; Kaitlyn Sadtler; Shoumyo Majumdar; Jyothi Mula; Matthew T Wolf; Jennifer H Elisseeff; Kathryn R Wagner
Journal:  J Biol Chem       Date:  2018-08-23       Impact factor: 5.157

Review 6.  Tissue Tregs.

Authors:  Marisella Panduro; Christophe Benoist; Diane Mathis
Journal:  Annu Rev Immunol       Date:  2016-05-20       Impact factor: 28.527

7.  Association Study of Exon Variants in the NF-κB and TGFβ Pathways Identifies CD40 as a Modifier of Duchenne Muscular Dystrophy.

Authors:  Luca Bello; Kevin M Flanigan; Robert B Weiss; Pietro Spitali; Annemieke Aartsma-Rus; Francesco Muntoni; Irina Zaharieva; Alessandra Ferlini; Eugenio Mercuri; Sylvie Tuffery-Giraud; Mireille Claustres; Volker Straub; Hanns Lochmüller; Andrea Barp; Sara Vianello; Elena Pegoraro; Jaya Punetha; Heather Gordish-Dressman; Mamta Giri; Craig M McDonald; Eric P Hoffman
Journal:  Am J Hum Genet       Date:  2016-10-13       Impact factor: 11.025

Review 8.  Genetic modifiers of Duchenne and facioscapulohumeral muscular dystrophies.

Authors:  Rylie M Hightower; Matthew S Alexander
Journal:  Muscle Nerve       Date:  2017-09-22       Impact factor: 3.217

9.  Agent-based model illustrates the role of the microenvironment in regeneration in healthy and mdx skeletal muscle.

Authors:  Kelley M Virgilio; Kyle S Martin; Shayn M Peirce; Silvia S Blemker
Journal:  J Appl Physiol (1985)       Date:  2018-08-02

10.  Disruption of a key ligand-H-bond network drives dissociative properties in vamorolone for Duchenne muscular dystrophy treatment.

Authors:  Xu Liu; Yashuo Wang; Jennifer S Gutierrez; Jesse M Damsker; Kanneboyina Nagaraju; Eric P Hoffman; Eric A Ortlund
Journal:  Proc Natl Acad Sci U S A       Date:  2020-09-11       Impact factor: 11.205
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