Washington University Center for Translational Neuroscience

病毒载体核心(核心D)将支持由Blueprint资助的华盛顿大学(Wu)神经科学 研究人员通过开发用于细胞培养和体内实验的病毒载体。核心有三个 目标：1)协助华盛顿大学神经科学研究人员进行载体设计、亚克隆和病毒 制作需要慢病毒和/或腺相关病毒(AAV)的实验，2)维护数据库 校园内可用的病毒和媒介主干，并帮助调查人员遵守监管规定 使用病毒载体的要求，以及3)协助华盛顿大学神经科学研究人员 使用华盛顿大学和商业上提供的基于慢病毒的siRNA文库，包括 生成和筛选病毒并促进与华盛顿大学高中的互动 吞吐量筛选机器人核心。核心的大部分活动将是由调查员推动的。时间 在资源允许的情况下，核心还将开发具有潜在广泛兴趣的新病毒载体 神经科学研究人员(例如，针对特定细胞类型或特定细胞功能的记者)和 测试优化病毒介导的基因传递的新技术。病毒载体核心将密切互动 具有细胞成像核心(核心C)和分子分析核心(核心E)，以便于 协作和最大限度地利用资源。病毒载体核心将与 信息学和数据集成核心(核心G)，以开发数据共享和数据管理工具。 慢病毒和腺相关病毒载体为基因表达和基因沉默提供了强大的工具 在完整的大脑中。病毒载体核心将提供专业知识和方法开发，这将扩大 华盛顿大学神经科学研究人员的能力和资源。 与公共卫生相关：病毒载体核心将使调查人员能够进一步 了解包括脑缺血在内的神经系统疾病的机制和可能的治疗方法 (中风)和神经退行性疾病，如阿尔茨海默病和帕金森病。病毒式传播 核心病毒、慢病毒和腺相关病毒所使用的载体系统与目前的病毒系统相似 用于人类疾病的临床试验，因此病毒疗法在动物模型中被发现是有效的 这种疾病有可能被迅速转化为治疗剂，用于人体临床试验。

The Viral Vectors Core (Core D) will support Blueprint-funded Washington University (WU) Neuroscience investigators by developing viral vectors for use in cell culture and in vivo experiments. The Core has three aims: 1) Assist Washington University neuroscience researchers with vector design, subcloning and virus production for experiments requiring lentivirus and/or adeno-associated virus (AAV), 2) Maintain a database of viruses and vector backbones available on campus and help investigators comply with regulatory requirements in the use of viral vectors, and 3) Assist Washington University neuroscience investigators in the use of lentivirus-based siRNA libraries available at Washington University and commercially, including generating and screening viruses and facilitating interactions with the Washington University High Throughput Screening Robotics Core. The majority of the core's activities will be investigator-driven. Time and resources permitting, the core will also develop new viral vectors of potentially broad interest to neuroscience researchers (e.g., targeted at specific cell types, or reporters of specific cellular functions) and test new techniques for optimal viral-mediated gene delivery. The Viral Vectors Core will interact closely with the Cellular Imaging Core (Core C) and Molecular Analysis Core (Core E) in order to facilitiate collaborations and maximal use of resources. The Viral Vectors Core will work closely with the Informatics and Data Integration Core (Core G) to develop data-sharing and data-management tools. Lentiviral and adeno-associated viral vectors provide powerful tools for gene expression and gene silencing in the intact brain. The Viral Vectors Core will provide expertise and methods development that will expand the capabilities and resources of neuroscience investigators at Washington University. RELEVANCE TO PUBLIC HEALTH: The Viral Vectors Core will enable investigators to further our understanding of mechanisms and potential therapies for neurological disorders, including cerebral ischemia (stroke) and neurodegenerative disorders such as Alzheimer's disease and Parkinson's disease. The viral vector systems used by the core, lentivirus and adeno-associated virus, are similar to viral systems currently used in clinical trials for human disorders, so viral therapies found to be efficacious in animal models of disease could potentially be rapidly translated into therapeutic agents for clinical trials in humans.