A platform for high-throughput production of targeting systems for cell-type-specific transgene expression in wild-type animals

项目摘要 我们将使用高通量技术来生产一组病毒载体，这些载体将允许转基因在大脑中的特定神经元群体中选择性表达。沿着许多其他应用，这将允许靶向神经元群体的光遗传学控制、记录和基因组修饰，而不需要产生转基因或敲除系。这将为神经科学提供一套多功能和强大的工具，使一系列新的实验设计成为可能，这些设计可能会对神经元回路的组织以及正常认知和行为的生物学基础以及神经和精神障碍的功能障碍产生重大见解。此外，由于新工具也被设计用于人类，因此它们可能会为精神和神经疾病带来重要的新疗法。

PROJECT SUMMARY We will use high throughput techniques to produce a set of viral vectors that will allow selective expression of transgenes in specific populations of neurons in the brain. Along with many other applications, this will allow optogenetic control, recording, and genomic modification of targeted neuronal populations without the need for production of transgenic or knock-out lines. This will provide neuroscience with a versatile and powerful set of tools that will make possible a broad set of new experimental designs that are likely to yield major insights into the organization of neuronal circuits and the biological bases of normal cognition and behavior and the dysfunctions underlying neurological and mental disorders. Additionally, because the new tools are also designed to work in humans, they are likely to result in important new therapies for mental and neurological diseases.