Stable suppression of gene function in mammals by RNAi

描述（由申请人提供）：培养的哺乳动物细胞是最 通常使用的模式生物，无论是在学术和工业生物学。 然而，可用于操纵这些细胞的工具已经被 与其他模式生物相比， 作为C.线虫和果蝇。基因公司的主要使命是 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 操纵哺乳动物模式生物的技术。Genetica开发了一种 一套工具，马克思系统，使正向遗传学能够被利用， 以解决细胞培养中的许多生物学问题。然而，到目前为止，我们 我最常使用该系统来拯救表型， 表情本提案的目标是培养使用 双链RNA诱导的基因沉默（RNAi）作为稳定 抑制哺乳动物细胞中的基因功能。这一目标根深蒂固 在有希望的初步数据，预测最终的成功， 奋进。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， 对潜在治疗药物的识别和验证产生了深远的影响， 目标的 拟定商业应用：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