RNA interference-based gene therapy for successful treatment of rheumatoid arthritis
RNA interference-based gene therapy for successful treatment of rheumatoid arthritis
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DOI：
10.1517/14712590902926089
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发表时间：
2009-05-01
影响因子：
4.6
通讯作者：
Apparailly, Florence
中科院分区：
文献类型：
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作者：
Courties, Gabriel;Presumey, Jessy;Apparailly, Florence
背景：RNA干扰(RNAi)是由短双链RNA启动的一种强大的内源性过程，导致序列特异性转录后基因沉默。阻断任何蛋白质表达的可能性为广泛疾病的潜在治疗应用带来了巨大的期望。然而，对于临床开发，基于RNAi的疗法的使用必须克服主要障碍，主要是靶向递送和安全问题。目的/方法：在这篇简短的综述中，我们概述了基于RNAi的基因治疗类风湿关节炎(RA)的规范，并讨论了有效沉默的最新进展，重点是短发夹状RNA的表达。结果/结论：将RNAi方法学的进展与基因治疗技术相结合，为快速应用于RA开辟了道路。
Background: RNA interference (RNAi) is a powerful endogenous process initiated by short double-stranded RNAs, which results in sequence-specific posttranscriptional gene silencing. The possibility of blocking the expression of any protein carries huge expectations for potential therapeutic applications in a wide range of diseases. For clinical development, however, the use of RNAi-based therapeutics has to overcome major obstacles, mainly targeted delivery and safety issues. Objective/methods: In this short review, we provide an overview of specifications for RNAi-based gene therapy in rheumatoid arthritis (RA) and discuss recent progresses in the development of efficient silencing, focusing on expression of short hairpin RNAs. Results/conclusions: Combining advances in RNAi methodology with gene therapy technology opens avenues for rapid applications to RA.