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Review Article

现代临床试验中基于RNA的治疗方法

卷 19, 期 3, 2019

页: [172 - 196] 页: 25

弟呕挨: 10.2174/1566523219666190719100526

价格: $65

摘要

对各种类型的RNA的长期研究导致人们对各种机制的进一步了解，最终导致基于RNA的疗法迅速发展成为临床疾病治疗中的强大工具。一些正在开发的RNA药物遵循反义机制，包括反义寡核苷酸，小干扰RNA，微小RNA，小活化RNA和核酶。这些类型的RNA可用于抑制/激活基因表达或改变剪接以提供功能蛋白。同时，一些基于不同机制的其他信使，例如修饰的信使RNA，可以代替功能异常的内源基因来控制某些遗传疾病，具有特殊三维结构的适体可以高亲和力与特定靶标结合。此外，由最关键的单向导RNA组成的最新最流行的CRISPR-Cas技术可以直接编辑DNA以产生治疗效果。最近的临床试验所期望的结果表明，基于RNA的药物在治疗各种疾病方面具有巨大潜力，但是，要使基于RNA的新型药物转化为临床药物，还需要进一步研究改善输送材料和修饰RNA的方法。这篇综述着力于当前基于RNA的疗法的进展和临床研究，分析了它们的挑战和前景。

关键词: 反义寡核苷酸，小型干扰RNA，微型小RNA，小型活化RNA，核酶，适体，信使RNA，单向导RNA。

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图形摘要

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