SGER: Development of Antisense Gene Knock-Down Procedures for in Vivo CNS Use

SGER：开发用于体内 CNS 使用的反义基因敲低程序

• 批准号: 9906871
• 负责人: Barbara Bregman
• 金额: $ 6.25万
• 依托单位: Georgetown University
• 依托单位国家: 美国
• 项目类别: Standard Grant
• 财政年份: 1999
• 资助国家: 美国
• 起止时间: 1999-04-15 至 2001-03-31
• 项目状态: 已结题
• 来源: https://www.nsf.gov/awardsearch/showAward?AWD_ID=9906871&HistoricalAwards=false
• 关键词: SGER; Development; Antisense; Gene; Knock

During development, different proteins are expressed by cells as different genes within those cells are turned on and off. Understanding such gene regulation is crucial for understanding developmental mechanisms. The developing brain is particularly complex, forming highly specific connections, and having whole groups of cells migrate through other cells to form discrete layers. Such activity depends in part on proteins responsible for cellular recognition and guidance. This Small Grant for Exploratory Research (SGER) exploits new molecular technology for a novel approach to regulating gene 'knockdown.' By injecting compounds called oligonucleotides directly into relevant regions, particular genes in a local area and at a particular time may be prevented from normal protein expression. If successful, this new technique has great advantages of simplicity and specificity, not requiring creation or maintenance of stocks of transgenic or mutant animals. The potential impact of this novel approach is to provide a relatively cheap and rapid approach for analysis of molecular aspects of brain development. Its impact will also extend beyond developmental neuroscience to many areas of biology where issues of gene regulation are important.

在发育过程中，随着细胞内不同基因的开启和关闭，细胞表达不同的蛋白质。了解这种基因调控对于了解发育机制至关重要。发育中的大脑特别复杂，形成高度特定的连接，并让整组细胞通过其他细胞迁移，形成不同的层。这种活动在一定程度上取决于负责细胞识别和引导的蛋白质。这一小笔探索性研究拨款(SGER)利用新的分子技术，找到了一种调控基因“敲除”的新方法。通过将称为寡核苷酸的化合物直接注射到相关区域，局部区域和特定时间的特定基因可能会阻止正常的蛋白质表达。如果成功，这项新技术具有简单和特异性的巨大优势，不需要创建或维护转基因或突变动物的种群。这种新方法的潜在影响是为大脑发育的分子方面的分析提供了一种相对廉价和快速的方法。它的影响还将从发展神经科学延伸到生物学的许多领域，在这些领域，基因调控问题是重要的。