Stable suppression of gene function in mammals by RNAi

描述(申请人提供)：培养的哺乳动物细胞最多 在学术和工业生物学中常用的模式生物。 然而，可用于操作这些单元格的工具是 与其他模式生物相比相对原始，如 像线虫和果蝇一样。它一直是Genetica的主要使命 开发技术，提高操纵哺乳动物细胞的能力 文化。这类研究的附带好处也将得到改善。 操纵哺乳动物模型生物的技术。Genetica已经开发出一种 一套工具，马克思系统，使前向遗传学能够被利用 以解决细胞培养中的众多生物学问题。然而，到目前为止，我们 我最常使用这个系统来挽救异位的表型 表情。这项提议的目标是发展使用 双链RNA诱导的基因沉默(RNAi)作为稳定基因表达的工具 哺乳动物细胞中基因功能的抑制。这一目标根深蒂固 在有希望的初步数据中预测了这一最终的成功 努力吧。这样的成功将彻底改变调查人员的能力 研究哺乳动物细胞以确定基因功能，因此 对潜在治疗方法的识别和验证的深远影响 目标。 建议的商业应用：不可用

DESCRIPTION (provided by applicant): Cultured mammalian cells are the most commonly used model organisms, both in academic and in industrial biology. However, the tools that are available for manipulating these cells have been relatively primative compared to those available in other model organisms, such as C. elegans and Drosophila. It has been the primary mission of Genetica to develop technologies that improve the ability to manipulate mammalian cells in culture. A collateral benefit of such studies will also be improved technologies to manipulate mammalian model organisms. Genetica has developed a suite of tools, the MaRX system, which enable forward genetics to be harnessed to address numerous biological problems in cell culture. However, to date, we have most often used this system for rescue of phenotypes by ectopic expression. The goal of this proposal is to develop the ability to use double-stranded RNA-induced gene silencing (RNAi) as a tool for stable suppression of gene function in mammalian cells. This goal is strongly rooted in promising preliminary data that predicts the ultimate success of this endeavor. Such success would revolutionize the ability of investigators studying mammalian cells to determine gene function and would thus have profound affects on the identification and validation of potential therapeutic targets. PROPOSED COMMERCIAL APPLICATION: NOT AVAILABLE