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

在发育过程中，随着细胞内不同基因的开启和关闭，细胞会表达不同的蛋白质。 了解此类基因调控对于理解发育机制至关重要。 发育中的大脑特别复杂，形成高度特异性的连接，并且整组细胞在其他细胞之间迁移，形成离散的层。 这种活性部分取决于负责细胞识别和引导的蛋白质。 这项探索性研究小额资助 (SGER) 利用新的分子技术开发了一种调节基因“敲低”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，可以阻止局部区域和特定时间的特定基因的正常蛋白质表达。 如果成功，这种新技术具有简单性和特异性的巨大优势，不需要创建或维持转基因或突变动物的种群。 这种新方法的潜在影响是提供一种相对便宜且快速的方法来分析大脑发育的分子方面。 它的影响也将超越发育神经科学，扩展到基因调控问题很重要的许多生物学领域。

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.