Investigation of Neural Pathogenic Mechanisms Associated with Congenital Disorders of Glycosylation

Icahn School Of Medicine At Mount Sinai, New York NY

Investigators

Abstract

Administrative Core Summary This Program Project grant involves seven investigators across five institutions who will use different model systems and approaches to define pathogenic mechanisms associated with neural disease in the Congenital Disorders of Glycosylation (CDG). The goals of the Administrative Core of this Program Project grant are 1) to guide an integrated and highly collaborative effort investigating Congenital Disorders of Glycosylation (CDG) neuronal pathogenesis across multiple sites and different model systems, 2) to provide responsible and transparent fiscal and operational management for the Program, 3) supervise and track sharing of biological samples and ensure data safety, 4) foster a dynamic and interdisciplinary environment for training and professional development, and 5) efficiently disseminate results within the Program, and across the glycoscience and patient advocacy (e.g. CDG-CARE) communities. The Administrative Core of the Program includes the two PD/PIs, supported by the Program Coordinator and interfacing with the patient advocacy groups. As multiple Research Project sites and a Research Core are involved, the Administrative Core will play a critical role in ensuring timely communication between the investigators, and efficient sharing of information and insight from the different projects and experimental systems. This Core will establish electronic databases for data sharing and manage a web-based platform for the distribution of information. This Core will guide collaboration between the Research Projects and Cores of the P01, organize meetings between the Research Project and Core PIs and the PD/PIs to review the broad scientific direction of the P01, and establish and coordinate quarterly web-based calls or in-person meetings with the full P01 team to present research progress, and highlight areas of new collaboration across the Projects and Core. The assignment of an External Advisory Committee with expertise in key areas of glycoscience, neural development/function, cell adhesion, and signaling will assist Research Project leaders and provide guidance to the Core leadership. The Core will execute a formal evaluation plan for the overall scientific direction of the P01, and the progress of each Research Project and provide fiscal management of the Program Project grant and responsible allocation of funding across sites. A process for conflict resolution, and mechanisms for monitoring and quality control of all activities, will also be enacted and governed by the Administrative Core. Lastly, this Core will organize an annual retreat to foster exchange of ideas among trainees, facilitate knowledge sharing across sites, and enhance communication about new research developments to patient advocacy groups, clinicians, investigators, the scientific community at large, and the public.