Embryonic stem cell/induced Pluripotent stem cell growth and gene editing core

摘要 胚胎干细胞生长和转基因模型核心（核心B）将为所有四个提供服务 项目的目标是有效地产生和提供细胞和动物实验模型， 先天性心脏病（CHD）的患者。AIM1将提供人类和小鼠胚胎干细胞 细胞（hESC和mESC）衍生的CHD细胞模型。这将包括（1a）提供支助 对于hESC和mESC的基因组编辑，（1b）维持现有和新产生的高质量的hESC和mESC储备， 每个项目使用的hESC和mESC系，以及（1c）提供hESC和mESC分化方面的帮助 心脏谱系目的2为CHD的研究提供小鼠遗传模型。其中包括（2A） 为产生新的遗传修饰小鼠模型提供支持，（2b）建立和 为项目使用的新的和现有的小鼠模型维持育种种群，以及（2c）提供 每个项目的实验动物。核心B人员在细胞和动物的各个方面都接受过良好的培训 提供的服务。核心的目标是为项目提供集中的、质量受控的试剂， 从而最大化效率并最小化与使用细胞和动物模型相关的成本。 考虑到对转基因模型（细胞和动物）的严重依赖和已证实的性能， 在核心人员中，核心B对本计划项目补助金申请成功的重要性不能 被夸大了。

ABSTRACT The Embryonic Stem Cell Growth and Genetically Modified Models Core (Core B) will provide service to all four Projects with the goal of efficiently generating and providing cellular and animal experimental models of congenital heart disease (CHD) to Program participants. Aim 1 will provide human and mouse embryonic stem cell (hESC and mESC)-derived cellular models of CHD to the projects. This will include (1a) providing support for genome editing of hESC and mESCs, (1b) maintaining high quality stocks of existing and newly generated hESC and mESC lines used by each project, and (1c) providing assistance with hESC and mESC differentiation towards cardiac lineages. Aim 2 will provide mouse genetic models of CHD to the projects. This will include (2a) providing support for the generation of new genetically modified mouse models, (2b) establishing and maintaining breeding stocks for new and existing mouse models to be used by the Projects, and (2c) providing experimental animals for each Project. Core B personnel are well-trained in all aspects of the cellular and animal services to be provided. The goal of the Core is to provide centralized, quality-controlled reagents to the Projects, thereby maximizing efficiency and minimizing the costs associated with the usage of cellular and animal models. Given the heavy reliance on genetically modified models (both cellular and animal) and the proven performance of the Core personnel, the importance of Core B to the success of this Program Project Grant application cannot be overstated.