基于 RNA 干扰的基因治疗成功治疗类风湿关节炎。
RNA interference-based gene therapy for successful treatment of rheumatoid arthritis.
机构信息
Inserm, U844, Université Montpellier 1, CHU Lapeyronie, Montpellier, France.
出版信息
Expert Opin Biol Ther. 2009 May;9(5):535-8. doi: 10.1517/14712590902926089.
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.
背景
RNA 干扰（RNAi）是一种由短双链 RNA 引发的强大内源性过程，可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的疾病治疗应用带来了巨大的期望。然而，对于临床开发，基于 RNAi 的治疗方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 的基因治疗在类风湿关节炎（RA）中的规格，并讨论了开发高效沉默的最新进展，重点是短发夹 RNA 的表达。
结果/结论：将 RNAi 方法的进展与基因治疗技术相结合，为 RA 的快速应用开辟了道路。
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