Introduction: Duchenne muscular dystrophy is a neuromuscular disorder, which is caused by abnormalities in the DMD gene that encodes the membrane cytoskeletal protein dystrophin. Besides progressive skeletal muscle wasting, dystrophinopathy also affects non-skeletal muscle tissues, including cells in the cardio-respiratory system, the central nervous system, the liver and the kidney.Areas covered: This review summarizes the proteomic characterization of a key class of lipid chaperones, the large family of fatty acid binding proteins, and their potential role in muscular dystrophy. Recent proteomic surveys using animal models and patient specimens are reviewed. Pathobiochemical changes in specific proteoforms of fatty acid binding protein in the multi-system pathology of dystrophinopathy are discussed.Expert opinion: The mass spectrometric identification of distinct changes in fatty acid binding proteins in muscle, heart, liver, kidney and serum demonstrates that considerable alterations occur in key steps of metabolite transport and fat metabolism in muscular dystrophy. These new findings might be helpful to further develop a comprehensive biomarker signature of metabolic changes in X-linked muscular dystrophy, which should improve (i) our understanding of complex pathobiochemical changes due to dystrophin deficiency, (ii) the identification of novel therapeutic targets, and (iii) the design of differential diagnostic, prognostic and therapy-monitoring approaches.

中文翻译：

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肌营养不良症中脂肪酸结合蛋白的蛋白质组分析。

简介：杜氏肌营养不良症是一种神经肌肉疾病，是由编码膜细胞骨架蛋白肌营养不良蛋白的DMD基因异常引起的。除了进行性骨骼肌消瘦外，肌营养不良症还影响非骨骼肌组织，包括心脏呼吸系统，中枢神经系统，肝脏和肾脏中的细胞。涵盖范围：本综述总结了关键脂质类别的蛋白质组学表征伴侣，脂肪酸结合蛋白的大家族及其在肌肉营养不良中的潜在作用。回顾了最近使用动物模型和患者标本进行的蛋白质组学调查。讨论了肌营养不良症多系统病理学中脂肪酸结合蛋白特定蛋白形式的病理生化变化。专家意见：肌肉，心脏，肝脏，肾脏和血清中脂肪酸结合蛋白的明显变化的质谱鉴定表明，肌肉营养不良的代谢物转运和脂肪代谢的关键步骤发生了重大变化。这些新发现可能有助于进一步开发X连锁肌营养不良症的代谢变化的综合生物标志物特征，这将改善（i）我们对肌营养不良蛋白缺乏症引起的复杂病理生化变化的理解，（ii）确定新的治疗靶点， （iii）设计不同的诊断，预后和治疗监测方法。肾脏和血清表明肌肉营养不良的代谢物运输和脂肪代谢的关键步骤发生了重大变化。这些新发现可能有助于进一步开发X连锁肌营养不良症的代谢变化的综合生物标志物特征，这将改善（i）我们对肌营养不良蛋白缺乏症引起的复杂病理生化变化的理解，（ii）确定新的治疗靶点， （iii）设计不同的诊断，预后和治疗监测方法。肾脏和血清证明肌肉营养不良的代谢物转运和脂肪代谢的关键步骤发生了显着变化。这些新发现可能有助于进一步开发X连锁肌营养不良症的代谢变化的综合生物标志物特征，这将改善（i）我们对肌营养不良蛋白缺乏症引起的复杂病理生化变化的理解，（ii）确定新的治疗靶点， （iii）设计不同的诊断，预后和治疗监测方法。