Disease Modeling Core

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DISEASE MODELING CORE PROJECT SUMMARY/ABSTRACT The overall mission of the Disease Modeling Core (DM core) is to provide diabetes researchers with access to novel human stem cell-derived in vitro cell models for investigating cellular and molecular features of both Type 1 and Type 2 diabetes. Recent progress in stem cell, organoid culture, gene editing and directed differentiation technologies has afforded opportunities to develop state-of-the-art pre-clinical human models. However, because of the complex experimental protocols and specialized reagents used, together with the considerable up-front costs and time required to develop these sophisticated stem cell-based models, it is not practical nor cost-effective to develop these models in individual laboratories. The goal of DM core is to provide the expertise, infrastructure and access to novel human model systems and technologies to DRC investigators. To help overcome the obstacles faced by basic scientists and clinical researchers who wish to establish human stem cell approaches in their laboratories, we will provide expertise, resources and training in start-of-the-art stem cell technologies together with rigorous quality control testing, validation standardization and authentication all model platforms and reagents. This goal will be achieved in three ways: 1) providing access and training in current and emerging pluripotent and adult stem cell technologies for in vitro human disease modeling, including the use of organoids; 2) providing access, training and implementation of genome editing technology, directed cell differentiation/programming, and gene expression systems; and 3) providing validation, authentication and data sharing of all DM-related technologies. Thus, the overall mission of this core is to facilitate access, generation and usage of novel human stem cell-derived in vitro models to promote basic and translational innovative diabetes research at UC AMC.