Genome Engineering Core

基因组工程核心-摘要 华盛顿大学风湿病研究中心基因组工程核心（GEC）的目标 基于资源的中心（WU-RDRRC）是提供获得创新基因组技术的工具， 这将有助于临床前建模和功能测试，以研究炎症和 自身免疫性疾病。对风湿性疾病基础的基因组研究已变得普遍。 然而，遗传发现的翻译可能具有挑战性，需要开发模型来询问 细胞和疾病的表型和功能的基本问题，由于确定的遗传变化。这需要 通过创新的基因组工程技术开发经验证的模型。这些技术， 例如开发基因工程小鼠和CRISPR/Cas9编辑诱导多能干细胞 （iPSC）和小鼠胚胎需要大量的经验和高度专业化的设备，而这些设备在 大多数实验室GEC为WU-RDRRC研究人员提供了最新的基因组工程 技术，利用转基因和敲除小鼠设施（TKMF）和基因组 工程与干细胞中心在麦克唐纳基因组研究所（GESC），与教育和领导， 促进项目。我们建议继续提供这些服务，并通过以下三个具体措施对GEC进行创新 目的： 1.维持，发展，并介绍新的国家的最先进的遗传学服务WU-RDRRC成员。 2.协助研究人员通过与 转基因和敲除小鼠设施（TKMF）。 3.协助研究人员生产用于创建基因工程细胞系iPSC的试剂， 利用McDonnell Genome的基因组工程和干细胞中心， 研究所（GESC）。

GENOME ENGINEERING CORE - ABSTRACT The goal of the Genome Engineering Core (GEC) of the Washington University Rheumatic Diseases Research Resource-based Center (WU-RDRRC) is to provide access to innovative genomic technologies to build tools that will facilitate pre-clinical modeling and functional testing for the investigation of inflammatory and autoimmune disease. Genomic investigation of the basis of rheumatic disease has become widespread. However, the translation of genetic findings can be challenging, requiring development of models to interrogate basic questions of cellular and disease phenotype and function due to identified genetic changes. This requires development of validated models through innovative genome engineering technologies. These technologies, such as development of genetically-engineered mice and CRISPR/Cas9 editing of induced pluripotent stem cells (iPSCs) and mouse embryos require significant experience and highly-specialized equipment not available within most laboratories. The GEC provides WU-RDRRC investigators with access to the latest in genomic engineering techniques by leveraging services from the Transgenic and Knockout Mouse Facility (TKMF) and the Genome Engineering & Stem Cell Center at McDonnell Genome Institute (GESC), with education and leadership to facilitate projects. We propose to continue these services and innovate the GEC through following three Specific Aims: 1. Sustain, develop, and introduce new state-of-the-art genetics services to WU-RDRRC members. 2. Assist investigators in the generation of genetically engineered mouse models by partnering with the Transgenic and Knockout Mouse Facility (TKMF). 3. Assist investigators in the generation of reagents for creation of genetically-engineered cell lines, iPSCs, and mouse models by leveraging the Genome Engineering & Stem Cell Center at McDonnell Genome Institute (GESC).