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长链非编码RNA疗法：最新进展和挑战

卷 23, 期 16, 2022

发表于: 10 October, 2022

页: [1457 - 1464] 页: 8

弟呕挨: 10.2174/1389450123666220919122520

摘要

除了信使RNA(如核酶)之外，还发现了调控RNA(如microRNA和长非编码RNA)的作用，这是令人着迷的。RNA现在被认为是几乎参与每个生物过程的重要调节因子。多年来，非编码RNA领域的研究，特别是microRNA（miRNA）和长非编码RNA（LncRNA）得到了极大的发展。最近的研究确定了不同的RNA，包括非编码RNA，如LncRNA，以及它们在细胞中的各种作用模式。这些RNA有望成为治疗各种疾病的关键靶标，因为它们控制着广泛的生物途径。LncRNA靶向药物平台为制药行业提供了无数的机会，并有可能在基因水平上调节疾病，同时克服不一致蛋白质的局限性。本文重点介绍该领域的最新进展和主要挑战，并描述了各种基于RNA的疗法，这些疗法改变了许多疾病的医疗保健质量，并使个性化药物成为现实。本文还总结了正在临床试验中进行测试或已获得FDA批准的基于RNA的疗法。

关键词: 微小RNA，长非编码RNA，环状RNA，急性髓性白血病, 慢性髓性白血病，核酶

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