RNAi Therapeutics in Autoimmune Disease.
RNAi Therapeutics in Autoimmune Disease.
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DOI：
10.3390/ph6030287
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发表时间：
2013-03-05
期刊：
影响因子：
--
通讯作者：
Cha S
中科院分区：
文献类型：
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作者：
Pauley KM;Cha S
自从发现 RNA 干扰 (RNAi) 以来，人们对其潜在的治疗用途越来越兴奋。靶向 RNAi 途径提供了一种强大的工具，可以在癌症或自身免疫性疾病等各种健康状况下改变转录后的生物过程。 shRNA、siRNA 和 miRNA 的优化设计可增强基于 RNAi 的方法的稳定性和特异性，同时必须减少或防止不良的免疫反应或脱靶效应。了解自身免疫性疾病发病机制的最新进展使得这些工具在体外和体内的应用取得了一定程度的成功。对 RNAi 途径效应器的设计和传递以及 RNAi 的分子基础的进一步研究将保证基于 RNAi 的疗法在人类应用中的实际应用。本综述将重点关注当前治疗方法及其在自身免疫性疾病（包括类风湿性关节炎和干燥综合征）中的应用。
Since the discovery of RNA interference (RNAi), excitement has grown over its potential therapeutic uses. Targeting RNAi pathways provides a powerful tool to change biological processes post-transcriptionally in various health conditions such as cancer or autoimmune diseases. Optimum design of shRNA, siRNA, and miRNA enhances stability and specificity of RNAi-based approaches whereas it has to reduce or prevent undesirable immune responses or off-target effects. Recent advances in understanding pathogenesis of autoimmune diseases have allowed application of these tools in vitro as well as in vivo with some degree of success. Further research on the design and delivery of effectors of RNAi pathway and underlying molecular basis of RNAi would warrant practical use of RNAi-based therapeutics in human applications. This review will focus on the approaches used for current therapeutics and their applications in autoimmune diseases, including rheumatoid arthritis and Sjögren’s syndrome.