Administrative Core

项目摘要 该多项目中心旨在建立一个框架，以了解FMRP介导的人类 大脑发育和电路功能在分子，细胞和系统电路水平，使用我们建立的 人类2D和3D模型系统以及小鼠模型。该中心将检验总体假设， FMRP通过转录调控新mRNA调控人脑发育和回路功能 这些靶点可能成为脆性X综合征的新治疗靶点。我们建议的中心带来了 来自埃默里大学医学院、斯坦福大学 纽约大学医学院宾夕法尼亚大学佩雷尔曼学院神经科学中心 南加州大学生物医学工程系。我们的调查人员 多年来，我们一直在进行富有成效的合作， 在准备本申请时。行政核心将作为成功的基础， 通过集中协调拟议项目和利用现有的 资源像这样的大型合作研究项目，旨在生成大型数据集 需要在多个地点的研究项目中进行分析和整合，需要一个集中的， 努力确保成功。行政核心将承担领导 努力保持透明度、组织和沟通。行政核心将负责 促进调查人员之间共享资源和数据，确保及时向 里程碑，与科学界和公众分享成果，并进行财政和 监管监督。我们致力于确定创新的、最先进的工具来促进这一点 共同努力实现我们的总体目标。使用一系列协作组织产品，我们将 能够确保开放的通信渠道，安全的数据存放地点，以及透明的操作， 中心。总而言之，行政核心是成功协作的基础， 集中协调拟议项目，并利用伙伴机构内的现有资源。 埃默里大学，主办机构，提供了大量的资源和专业知识，我们可以获得 促进本中心的有效管理。我们相信，我们的协调努力将创造科学的 协同作用，并显著地推进了我们对人脑中FMRP介导基因调控的理解 开发和实现脆性X综合征的新治疗开发。

Project Summary This multi-project Center aims to establish a framework to understand the FMRP-mediated regulation of human brain development and circuit functions at molecular, cellular and system circuitry levels using our established human 2D and 3D model systems as well as mouse models. The Center will test the overall hypothesis that FMRP regulates human brain development and circuit functions via translational regulation of novel mRNA targets, which could serve as new therapeutic targets for fragile X syndrome. Our proposed Center brings together an outstanding team of investigators from Emory University School of Medicine, Stanford University School of Medicine, New York University Center for Neural Science, University of Pennsylvania Perelman School of Medicine, and University of Southern California Department of Biomedical Engineering. Our investigators have had ongoing and productive collaborations with each other for many years and have been highly cooperative during the preparation of this application. The Administrative Core will serve as the foundation for a successful collaborative effort through centralized coordination of the proposed Projects and the leveraging of existing resources. A large collaborative research program such as this, which is designed to generate large datasets that need to be analyzed and integrated over research projects at multiple sites, requires a focused and dedicated effort to ensure success. The Administrative Core will assume primary responsibility for leading the effort to maintain transparency, organization, and communication. The Administrative Core will be responsible for facilitating the sharing of resources and data among investigators, ensuring timely progression towards milestones, sharing results with the scientific community and the general public, and conducting financial and regulatory oversight. We are committed to identifying innovative, state-of-the-art tools to facilitate this collaborative effort to reach our overarching goal. Using an array of collaborative organization products, we will be able to ensure open communication channels, secure sites for data deposition, and transparent operation of the Center. In summary, the Administrative Core is the foundation for a successful collaborative effort through centralized coordination of the proposed Projects and leverages existing resources within the partner institutions. Emory University, the host institution, offers a vast array of resources and expertise that we can access to facilitate the effective administration of this Center. We believe that our coordinated effort will create scientific synergy and significantly advance our understanding of FMRP-mediated gene regulation in human brain development and enable novel therapeutic development for fragile X syndrome.