Advanced Platform of RNAi Technology

RNAi技术先进平台

• 批准号: 6702393
• 负责人: Jiwu Wang
• 金额: $ 9.95万
• 依托单位: ALLELE BIOTECHNOLOGY AND PHARMACEUTICALS
• 依托单位国家: 美国
• 财政年份: 2003
• 资助国家: 美国
• 起止时间: 2003-08-01 至 2004-01-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6702393
• 关键词: DNA binding protein; DNA directed RNA polymerase; RNA; biotechnology; cell line; chemical conjugate; crosslink; double stranded RNA; gene delivery system; gene induction /repression; genetic promoter element; genetic techniques; genetic transduction; oligonucleotides; peptide chemical synthesis; polymerase chain reaction; reagent /indicator; technology /technique development; transfection; transport proteins

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.

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将RNAi诱导试剂递送到哺乳动物细胞中的效率的限制。该提案的目的是建立有效地转染siRNA表达盒的技术。我们已经开发了一个系统，以诱导RNA的影响，在哺乳动物I细胞中使用新的基于DNA的表达盒，提供了比其他RNA或质粒为基础的方法的巨大优势。我们计划利用我们现有的核心技术的独特设计，通过使用转导肽作为dell非常车辆。这对于传统上难以转染的细胞如原代培养物中基于RNAi的基因沉默将是特别有用的。这些技术可以进一步整合到用于高通量发现的新平台中，并适用于活动物。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。