Washington University Rheumatic DiseasesResearch Resource-based Center

总体摘要 华盛顿大学风湿病研究资源的总体目标 中心（WU-RDRRC）旨在通过提供基础设施、资源来推进风湿病研究 以及进行多层次探究并将这些发现转化为治疗方法的机会 患者。 WU-RDRRC 致力于通过支持来促进风湿病患者的健康 支持技术并促进其成员的基础和转化研究兴趣。这些 兴趣围绕三大主题组织：（1）炎症和自身免疫的基本机制； (2) 转化基因组学和生物标志物发现； (3) 肌肉骨骼和再生医学。 我们的中心假设是风湿病研究的转化工作需要一个团队 在充满活力的环境中执行的方法，由跨学科专家团队和最新状态支持 最先进的服务。我们的重点是推广互动方法，以加快创新步伐 发现并将发现转化为风湿病患者治疗的改进。 为了实现我们的目标，我们提出以下建议：1）转化研究核心（TRC），包括 致力于支持转化研究工作的独立但相关的服务：i) 综合服务 可通过电子方式检索的风湿病登记和生物样本库（Biobank） 生物医学数据管理系统，允许注释、跟踪和共享组织和数据库； ii) 一个 由最新的下一代测序技术提供支持的基因组学和生物信息学设施 (GBF) 增强人类基因组数据的安全使用和分析的技术和生物信息学工具，以及 iii) 免疫监测设施 (IMF)，提供质谱流式细胞术来分析人类样本。 2）A 基因组工程核心 (GEC) 通过利用技术进步促进功能研究 例如CRISPR/Cas9产生突变细胞系和基因敲除/敲入小鼠以及诱导多能干细胞 细胞（iPSC）； 3) 细胞成像核心 (CIC)，提供最先进的成像技术 建立健康和疾病模型。行政核心促进协作和协同 风湿病研究跨多个学科的相互作用，并促进新的指导 对风湿病相关研究感兴趣的研究人员。

OVERALL ABSTRACT The overarching Goal of the Washington University Rheumatic Diseases Research Resource-based Center (WU-RDRRC) is to advance research in rheumatic diseases by providing the infrastructure, resources and opportunities for multiple levels of inquiry and translating those discoveries into treatments for delivery to patients. The WU-RDRRC seeks to advance the health of patients with rheumatic disease by supporting enabling technology and promoting the basic and translational research interests of its members. These interests are organized around three major themes: (1) Basic mechanisms of inflammation and autoimmunity; (2) Translational genomics and biomarker discovery; and (3) Musculoskeletal and Regenerative Medicine. Our Central Hypothesis is that translational endeavors in rheumatic diseases research require a team approach performed in a vibrant environment supported by a cross-disciplinary team of experts and state-of- the-art services. Our Focus is to promote interactive approaches that will accelerate the pace of innovative discoveries and translate findings to improvements in treatments for patients with rheumatic diseases. To accomplish our goals we propose the following: 1) A Translational Research Core (TRC) that comprises separate but related services devoted to supporting translational research endeavors: i) an integrated rheumatic disease registry and biospecimen repository (Biobank) that can be searched through electronic biomedical data management systems to allow annotation, tracking and sharing of tissue and databases; ii) a Genomics & Bioinformatics Facility (GBF) powered by the most up-to-date Next-Generation Sequencing technologies and bioinformatics tools to enhance the secure use and analysis of human genomic data and iii) an ImmunoMonitoring Facility (IMF) that offers access to mass cytometry for analysis of human samples. 2) A Genome Engineering Core (GEC) that facilitates functional studies through the use of technological advances such as CRISPR/Cas9 to generate mutant cell lines and knockout/knock-in mice and induced Pluripotent Stem Cells (iPSCs); and 3) A Cellular Imaging Core (CIC) that provides state-of-the-art imaging technologies for building models of health and disease. The Administrative Core promotes collaborative and synergistic interactions in rheumatic diseases research across multiple disciplines and fosters the mentoring of new investigators interested in rheumatic disease-related research.