Washington University Rheumatic Diseases Research Resource-based Center

总体摘要 WU-RDRRC旨在通过支持使风湿性疾病患者能够 技术和促进成员组织的基础、临床和转化性研究兴趣 围绕三大主题：1)阐明炎症和自身免疫的基本机制；2) 加快临床/翻译研究，为精确医学提供信息；以及3)推进基因组工程 和再生医学，以开发风湿病的新治疗方法。 我们假设，风湿病的翻译研究工作需要团队方法来培养 一个充满活力的环境，并得到跨学科专家组和尖端技术的支持。 在过去的四年里，这一运营理念和由WU-RDRRC支持的基础设施 完美定位我们的调查人员，以提高他们的研究效率和影响，使他们能够 快速解决新冠肺炎大流行期间出现的紧急问题，并帮助许多年轻人 研究人员获得了外部资金，并成功过渡到研究独立。吴氏- RDRRC建议通过以下方式继续推动风湿病研究：1)提供基础设施， 所需的教育和培训，以协助调查人员进行实验和科学设计 项目；2)组织和支持采用和应用创新技术和 调查人员无法以具有成本效益的方式获得的专业知识；3)提高兴趣 在风湿病研究中，通过参与多学科团队并将不同学科结合在一起 和临床科室，以解决翻译科学方面的障碍；4)培养下一代青少年 对风湿病相关领域研究感兴趣的研究人员。 为了实现我们的目标，我们提出了四个核心：1)一个生物库和表型核心，以收集高质量的 用于下游分子分析的生物谱学；2)产生生物工程的基因组工程核心 基于细胞的治疗，敲除/敲除小鼠，用于临床前疾病模型测试；3)细胞成像 提供用于建立健康和疾病模型的新型显微技术的核心；以及4)一个 促进风湿病之间的协作和协同作用的管理核心 研究人员以及对风湿病研究感兴趣的初级和新手研究人员的指导。 此外，我们将通过扩大获得新技术的机会来满足成员不断变化的需求，例如 空间转录学，通过将健康的社会决定因素纳入 临床数据收集，并加强进入医生-科学家途径的受训人员的多样性。

OVERALL ABSTRACT The WU-RDRRC seeks to advance the health of patients with rheumatic diseases by supporting enabling technology and promoting the members’ basic, clinical, and translational research interests that are organized around three major themes: 1) elucidating basic mechanisms of inflammation and autoimmunity; 2) accelerating clinical/translational research to inform precision medicine; and 3) advancing genome engineering and regenerative medicine to develop new treatment approaches for rheumatic diseases. We posit that translational research endeavors in rheumatic diseases require a team approach cultivated in a vibrant environment and supported by cross-disciplinary groups of experts and cutting-edge technologies. Over the past four years, this operational philosophy and the infrastructure supported by the WU-RDRRC perfectly positioned our investigators to increase the efficiency and impact of their research, allowed them to quickly address emergent issues that arose during the COVID-19 pandemic, and helped a number of young investigators obtain extramural funding and successfully transition to research independence. The WU- RDRRC propose to continue promoting rheumatic disease research by: 1) providing the infrastructure, education, and training needed to assist investigators with the experimental and scientific design of their projects; 2) organizing and supporting Core laboratories that will adopt and apply innovative technologies and expertise that would otherwise be unavailable to investigators in a cost-effective manner; 3) promoting interest in rheumatic diseases research by engaging multidisciplinary teams and bringing together diverse disciplines and clinical divisions to address roadblocks in translational science; 4) fostering the next generation of junior investigators who are interested in the study of rheumatic disease-related areas. To accomplish our goals we propose four cores: 1) a Biobank and Phenotyping Core to collect high-quality biospecimens for downstream molecular analysis; 2) a Genome Engineering Core to generate bioengineered cell-based therapies, knockin/knockout mice for testing in preclinical disease models; 3) a Cellular Imaging Core that offers novel microscopy technologies for building models of health and disease; and 4) an Administrative Core that promotes collaborative and synergistic interactions among rheumatic disease researchers and the mentoring of junior and new investigators interested in rheumatic disease research. In addition, we will address members’ evolving needs by expanding access to novel technologies such as spatial transcriptomics, and the changing societal landscape by incorporating social determinants of health into clinical data collection, and enhancing diversity of trainees entering the physician-scientist pathway.