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The potential of sarcospan in adhesion complexreplacement therapeutics for the treatment ofmuscular dystrophy

机译:肌节在粘附复合物替代疗法中治疗肌肉营养不良的潜力

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摘要

Three adhesion complexes span the sarcolemma and facilitate critical connectionsbetween the extracellular matrix and the actin cytoskeleton: thedystrophin– and utrophin–glycoprotein complexes and a7b1 integrin. Lossof individual protein components results in a loss of the entire proteincomplex and muscular dystrophy. Muscular dystrophy is a progressive,lethal wasting disease characterized by repetitive cycles of myofiber degenerationand regeneration. Protein-replacement therapy offers a promisingapproach for the treatment of muscular dystrophy. Recently, we demonstratedthat sarcospan facilitates protein–protein interactions amongst theadhesion complexes and is an important potential therapeutic target. Here,we review current protein-replacement strategies, discuss the potential benefitsof sarcospan expression, and identify important experiments that mustbe addressed for sarcospan to move to the clinic.
机译:三种粘附复合物跨越肌膜,并促进细胞外基质与肌动蛋白细胞骨架之间的关键连接:抗肌萎缩蛋白和尿素蛋白糖蛋白复合物以及a7b1整联蛋白。单个蛋白质成分的损失导致整个蛋白质复合物和肌肉营养不良的丧失。肌营养不良症是一种进行性致死性消耗性疾病,其特征是肌纤维变性和再生的反复循环。蛋白质替代疗法为治疗肌营养不良症提供了一种有前途的方法。最近,我们证明了肌节可促进粘附复合物之间的蛋白质相互作用,并且是重要的潜在治疗靶标。在这里,我们回顾了当前的蛋白质替代策略,讨论了肌节表达的潜在益处,并确定了肌节要移至临床必须解决的重要实验。

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