Preclinical Models Core (Core A)

项目摘要-临床前模型核心 临床前模型核心(PMC)将提供访问最先进的工具和服务、教育和 培训计划和开发新的方法，重点是发现和早期验证治疗 肾脏疾病的靶标。尽管最近取得的突破导致了我们在 对多囊肾病、局灶性节段性肾病等肾脏疾病发病机制的认识 肾小球硬化，诊断、管理和治愈这些疾病的新疗法出现得很慢。 为肾病患者提供新治疗方法的关键一步是提供先进的 基础发现研究、新发现途径的询问和早期临床前模型系统 目标验证。在PMC，我们将利用我们的人才的丰富经验和技术专长 肾脏研究人员，同时利用西北大学的大量资源，并将它们整合到一个 行政保护伞，以建立和提供方便的访问：1)NUMUSEN超过50个独特的住房 遗传性肾病小鼠品系2)快速设计和生成基因组编辑‘礼宾’服务 新的遗传性肾脏模型3)包括高血压和肾脏在内的非遗传性肾病模型 移植模型和最先进的小鼠表型服务以及4)代和 肾脏疾病患者诱导多能干细胞(IPSC)及其肾脏的基因组编辑 生物库。PMC Core还将提供动手研讨会和基于网络的小鼠遗传学培训， 基因组编辑，诱导多能干细胞(IPSC)技术和肾脏研究人员和 实习生。将向全球肾病界提供外联和获得所有与核心有关的服务 通过一个名为neroHub的在线门户网站。无缝集成，并与 治疗学的发展和临床翻译NU-GoKIDNEY核心，试点和可行性 浓缩计划和创新的肾脏治疗加速器计划将催化发现和 加快将其转化为肾脏患者的创新疗法。

PROJECT SUMMARY – PRECLINICAL MODELS CORE The Preclinical Models Core (PMC) will provide access to state-of-the art tools and services, education and training programs and develop new methodologies focused on the discovery and early validation of therapeutic targets for kidney diseases. Despite recent breakthroughs that have led to major advances in our understanding of the pathogenesis of kidney diseases such as polycystic kidney disease and focal segmental glomerulosclerosis, new therapies to diagnose, manage and cure these disorders have been slow to emerge. A key step in the path to provide new treatments for kidney patients is the availability of sophisticated preclinical model systems for basic discovery research, interrogation of newly identified pathways and early target validation. In the PMC, we will exploit the extensive experience and technical expertise of our talented kidney investigators while leveraging substantial Northwestern resources and bring them together under one administrative umbrella to establish and provide easy access to: 1) NUmousekidney housing over 50 unique genetic kidney disease mouse strains 2) a genome editing `concierge' service to rapidly design and generate new genetic renal models 3) non-genetic kidney disease models including hypertensive and renal transplantation models and state-of-the art mouse phenotyping services and 4) service for the generation and genomic editing of induced pluripotent stem cells (iPSC) from patients with kidney diseases and an iPSCkidney biobank. The PMC Core will also provide hands-on workshops and web-based training in mouse genetics, genome editing, induced pluripotent stem cell (iPSC) technologies and phenotyping to kidney investigators and trainees. Outreach and access to all Core-related services will be provided to the global kidney community through an online web portal named nephro-HUB. Seamless integration and strong synergy with the Therapeutics Development and Clinical Translation NU-GoKIDNEY Cores, the Pilot and Feasibility and Enrichment Programs and an innovative Kidney Cure Accelerator program will catalyse discoveries and accelerate their translation into innovative therapies for kidney patients.