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首页> 外文期刊>Neurochemistry International: The International Journal for the Rapid Publication of Critical Reviews, Preliminary and Original Research Communications in Neurochemistry >Myostatin as a therapeutic target in Amyotrophic Lateral Sclerosis
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Myostatin as a therapeutic target in Amyotrophic Lateral Sclerosis

机译：肌生长抑制素作为肌萎缩性侧索硬化症的治疗靶标

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摘要

Amyotrophic Lateral Sclerosis is a devastating neurological disease that is inevitably fatal after 3-5 years duration. Treatment options are minimal and as such new therapeutic modalities are required. In this review, we discuss the role of the myostatin pathway as a modulator of skeletal muscle mass and therapeutic approaches using biological based therapies. Both monoclonal antibodies to myostatin and a soluble receptor decoy to its high affinity receptor have been used in clinical trials of neuromuscular diseases and while there have been efficacy signals with the latter approach there have also been safety issues. Our approach is to target the high affinity receptor-binding site on myostatin and to develop a next generation set of therapeutic reagents built on a novel protein scaffold. This is the natural single domain VNAR found in sharks which is extremely versatile and has the ability to develop products with superior properties compared to existing therapeutics.
机译：肌萎缩性侧索硬化症是一种毁灭性的神经系统疾病，持续3-5年后不可避免地致命。治疗选择极少，因此需要新的治疗方法。在这篇综述中，我们讨论了肌肉生长抑制素途径作为骨骼肌质量调节剂的作用以及使用基于生物学的疗法的治疗方法。肌肉生长抑制素的单克隆抗体和其高亲和力受体的可溶性受体引诱物都已用于神经肌肉疾病的临床试验中，尽管在后一种方法中已产生功效信号，但也存在安全性问题。我们的方法是靶向肌生长抑制素上的高亲和力受体结合位点，并开发建立在新型蛋白质支架上的下一代治疗试剂。这是在鲨鱼中发现的天然单域VNAR，具有极强的通用性，并且能够开发出与现有疗法相比性能更优越的产品。

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