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，这项技术的应用在很大程度上受到将RNAi诱导试剂输送到哺乳动物细胞的效率的限制。这项建议的目的是建立有效地转导siRNA表达盒的技术。我们已经开发了一种系统，使用基于DNA的新型表达盒在哺乳动物I细胞中诱导R-NaI效应，与其他基于RNA或质粒基的方法相比，这种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，将转换多肽作为戴尔非常重要的载体。这将对传统上难以转化的细胞，如原代培养细胞中基于RNAi的基因沉默特别有用。这些技术可以进一步集成到新的平台中，以实现高通量的发现，并适用于活体动物。这些方法的发展也可能为我们的RNAi技术的令人兴奋的应用奠定基础 作为治疗剂。

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.