Immune-mediated pathology in Duchenne muscular dystrophy

被引：209
|
作者：
Rosenberg, Amy S. [1 ]
Puig, Montserrat [1 ]
Nagaraju, Kanneboyina [2 ]
Hoffman, Eric P. [2 ]
Villalta, S. Armando [3 ]
Rao, V. Ashutosh [1 ]
Wakefield, Lalage M. [4 ]
Woodcock, Janet [1 ]
机构：
[1] US FDA, Ctr Drug Evaluat & Res, Silver Spring, MD 20993 USA
[2] Childrens Natl Med Ctr, Ctr Genet Med Res, Washington, DC 20010 USA
[3] Univ Calif Irvine, Dept Physiol & Biophys, Inst Immunol, Irvine, CA 92697 USA
[4] NCI, Lab Canc Biol & Genet, Bethesda, MD 20892 USA
来源：
SCIENCE TRANSLATIONAL MEDICINE | 2015年 / 7卷 / 299期
关键词：
REGULATORY T-CELLS; GROWTH-FACTOR-BETA; MDX MOUSE MODEL; TOLL-LIKE RECEPTORS; TGF-BETA; SKELETAL-MUSCLE; TRANSFORMING GROWTH-FACTOR-BETA-1; INFLAMMATORY RESPONSE; DEFICIENT SKELETAL; DOUBLE-BLIND;
D O I：
10.1126/scitranslmed.aaa7322
中图分类号：
Q2 [细胞生物学];
学科分类号：
071009 ; 090102 ;
摘要：
Immunological and inflammatory processes downstream of dystrophin deficiency as well asmetabolic 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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页数：12
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