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老药新用：依布硒啉治疗遗传性运动神经元疾病

2018-05-28 MedSci MedSci原创

依布硒啉运动神经元疾病MND
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近日，一项最新的国际研究表明，抗炎镇痛药依布硒啉可以纠正导致遗传性运动神经元疾病（MND）的蛋白质的许多异常特征

近日，一项最新的国际研究表明，抗炎镇痛药依布硒啉可以纠正导致遗传性运动神经元疾病（MND）的蛋白质的许多异常特征。

MND是一种无法治愈的进行性疾病，它会攻击控制运动的神经，使肌肉不再起作用。MND在英国会影响约5000人，目前的治疗方案对于提高患者的生活质量作用不大。遗传性MND是一种罕见疾病（占全部病例的5-10％），大约20％的遗传性MND病例是由编码SOD1蛋白质的基因突变引起的。当SOD1基因发生突变时，蛋白质装配过程出现异常，某些步骤被遗漏，这使得SOD1蛋白在结构上不稳定，导致运动神经元中蛋白“团块”的形成。

英国利物浦大学、意大利佛罗伦萨大学和澳大利亚卧龙岗大学的科学家们使用最先进的晶体学技术、质谱和核内核磁共振技术来寻找可以“校正” SOD1组装线的药物。他们发现，一种在20世纪80年代被发现的药物依布硒啉，可有效恢复SOD1装配过程中的几个重要步骤，包括折叠、二聚和锌结合。目前依布硒啉已被作为各种神经系统疾病的潜在治疗药物进行研究。


原始出处：

https://www.europeanpharmaceuticalreview.com/news/75848/ebselen-treatment-mnd/

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