Preclinical Models Core (Core A)

项目总结-临床前模型核心 临床前模型核心（PMC）将提供最先进的工具和服务，教育和 培训计划，并开发新的方法，重点是发现和早期验证的治疗 肾脏疾病的靶点。尽管最近的突破导致了我们的重大进展， 了解肾脏疾病的发病机制，如多囊肾和局灶性节段性肾病 然而，由于肾小球硬化症，诊断、管理和治愈这些疾病的新疗法一直缓慢出现。 为肾病患者提供新治疗方法的关键一步是提供复杂的 用于基础发现研究的临床前模型系统，新发现的途径的询问和早期诊断 目标验证在PMC，我们将利用我们的人才丰富的经验和技术专长， 肾脏研究人员，同时利用大量的西北资源，并将他们聚集在一起， 行政保护伞，以建立和提供方便的访问：1）NUmousekidney住房超过50个独特的 遗传性肾病小鼠品系2）基因组编辑“礼宾”服务，以快速设计和生成 新的遗传性肾脏模型3）非遗传性肾脏疾病模型，包括高血压和肾脏疾病模型 移植模型和最先进的小鼠表型分析服务，以及4）生成和 来自肾脏疾病患者和诱导多能干细胞（iPSC）肾脏的诱导多能干细胞（iPSC）的基因组编辑 生物库PMC核心还将提供小鼠遗传学方面的实践研讨会和基于网络的培训， 基因组编辑，诱导多能干细胞（iPSC）技术和肾脏研究者的表型分析， 实习生将向全球肾脏社区提供所有核心相关服务的推广和访问 通过一个叫肾枢纽的在线门户网站无缝集成，与 NU-GoKIDNEY核心的治疗开发和临床转化，试点和可行性， 丰富计划和创新的肾脏治疗加速器计划将促进发现， 加速将其转化为肾病患者的创新疗法。

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.