Disease Modeling Core

疾病建模核心 项目摘要/摘要 疾病建模核心(DM CORE)的总体任务是为糖尿病研究人员提供 用于研究这两种类型细胞和分子特征的新型人类干细胞体外细胞模型 1型和2型糖尿病。干细胞、器官培养、基因编辑和定向分化研究进展 技术为开发最先进的临床前人体模型提供了机会。然而， 因为使用了复杂的实验方案和专门的试剂，以及相当大的 开发这些复杂的基于干细胞的模型所需的前期成本和时间，既不现实也不现实 在个别实验室开发这些模型具有成本效益。DM CORE的目标是提供专业知识， 基础设施以及向刚果民主共和国调查人员提供新的人体模型系统和技术。帮助 克服希望建立人类干细胞的基础科学家和临床研究人员面临的障碍 在他们的实验室中，我们将提供最先进的干细胞方面的专业知识、资源和培训 技术与严格的质量控制测试、验证标准化和认证都是 平台和试剂。这一目标将通过三种方式实现：1)提供现有和 用于体外人类疾病建模的新兴多能性和成体干细胞技术，包括使用 有机物；2)提供获取、培训和实施基因组编辑技术，定向细胞 差异/编程和基因表达系统；以及3)提供验证、认证和数据 共享所有与DM相关的技术。因此，这个核心的总体任务是促进访问、生成 以及使用新的人类干细胞来源的体外模型来促进基础和翻译创新 加州大学AMC的糖尿病研究。

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.