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.
