Advanced Platform of RNAi Technology

RNA干扰（RNAi）是基因功能研究和药物开发的有力工具。目前，该技术的应用在很大程度上受到rnai诱导试剂进入哺乳动物细胞的效率的限制。本提案的目的是建立有效转染sirna表达盒的技术。我们已经开发了一种系统，可以在哺乳动物I细胞中使用新的基于dna的表达盒来诱导R NAi效应，这种表达盒比其他基于RNA或质粒的方法具有更大的优势。我们计划利用我们现有核心技术的独特设计，利用转导肽作为我们的载体。这对于传统上难以转染的细胞（如原代培养）中基于rnai的基因沉默尤其有用。这些技术可以进一步整合到高通量发现的新平台中，并适用于活体动物。这些方法的发展也可能为RNAi技术令人兴奋的应用奠定基础

RNA interference (RNAi) is a powerful tool for gene function studies and drug development. Currently, application of this technology is largely limited by the efficiency of delivering RNAi-inducing reagents into mammalian cells. The aim of this proposal is to establish techniques to efficiently transfect siRNA-expressing cassettes. We have already developed a system to induce R NAi effects in mammalian I cells using novel DNA-based expression cassettes that offer gr eat advantages over other RNA or plasmid-based methods. We plan to take advantage of the unique design of our existing core technology by using transducing peptides as dell very vehicles. This will be particularly useful for RNAi-based gene silencing in traditionally hard to transfect cells such as primary cultures. T hese techniques can be further integrated into novel platforms for high throughput discovery and adapted for use in live animals. Development of these methods may also lay the foundations for exciting applications of our RNAi technology as therapeutic agents.