Genome Engineering Core

基因组工程核心-摘要 华盛顿大学风湿病研究基因组工程核心(GEC)的目标 基于资源的中心(WU-RDRRC)是提供获取创新基因组技术的工具 这将促进临床前建模和功能测试，以调查炎症和 自身免疫性疾病。对风湿病基础的基因组研究已经变得广泛。 然而，基因发现的翻译可能是具有挑战性的，需要开发模型来审问 由于已识别的基因变化而引起的细胞和疾病表型和功能的基本问题。这需要 通过创新的基因组工程技术开发经过验证的模型。这些技术， 例如基因工程小鼠的发展和诱导多能干细胞的CRISPR/Cas9编辑 (Ipscs)和小鼠胚胎需要丰富的经验和高度专业化的设备，这是 大多数实验室。GEC为WU-RDRRC研究人员提供了获得最新基因组工程的途径 利用转基因和基因敲除小鼠设施(TKMF)和基因组提供的服务 McDonnell Genome Institute(GESC)工程与干细胞中心，教育和领导 为项目提供便利。我们建议继续提供这些服务，并通过以下三个具体措施创新GEC 目标： 1.支持、发展并向WU-RDRRC成员提供最先进的遗传学服务。 2.协助研究人员培育基因工程小鼠模型 转基因和基因敲除小鼠设施(TKMF)。 3.协助研究人员研制用于创造基因工程细胞系、IPSCs、 利用McDonnell Genome的基因组工程和干细胞中心建立小鼠模型 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).