Preclinical Models Core (Core A)

临床前模型核心（核心 A）

• 批准号: 10460931
• 负责人: GUILLERMO C OLIVER
• 金额: $ 26.62万
• 依托单位: NORTHWESTERN UNIVERSITY AT CHICAGO
• 依托单位国家: 美国
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-08-01 至 2023-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10460931
• 关键词: Biological Assay; Biological Models; Biological Products; CRISPR/Cas technology; Cardiovascular Physiology; Cell Line; Cells; Clone Cells; Communities; Consultations; Databases; Derivation procedure; Development; Diabetic Nephropathy; Diagnosis; Disease; Disease model; E-learning; Educational workshop; Emerging Technologies; Focal Segmental Glomerulosclerosis; Gene Targeting; Generations; Genes; Genetic; Housing; Human; Hypertension; Injections; Innovative Therapy; International; Kidney; Kidney Diseases; Kidney Transplantation; Lead; Leadership; Metabolism; Methodology; Methods; Minerals; Modeling; Molecular; Monitor; Mouse Strains; Mus; Names; Pathogenesis; Pathway interactions; Patients; Phenotype; Physiology; Polycystic Kidney Diseases; Postdoctoral Fellow; Pre-Clinical Model; Prevention; Renal function; Research; Research Personnel; Research Support; Resources; Rodent; Rodent Model; Role; Sampling; Service delivery model; Services; Students; System; Talents; Technical Expertise; Testing; Therapeutic; Tissue Sample; Training; Training Programs; Training and Education; Transgenic Mice; Translating; Translations; Validation; Vertebrates; base; biobank; blastocyst; bone; candidate validation; clinical translation; cost; design; disease phenotype; embryonic stem cell; experience; gene function; genome editing; human model; human tissue; humanized mouse; hypertensive; induced pluripotent stem cell; induced pluripotent stem cell technology; innovation; insight; meetings; mouse genetics; mouse model; new technology; non-genetic; novel therapeutics; outreach; pre-clinical; programs; ranpirnase; repository; synergism; therapeutic candidate; therapeutic development; therapeutic evaluation; therapeutic target; tool; transplant model; web portal; webinar

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.

项目摘要——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 培训计划并开发新方法，重点关注治疗方法的发现和早期验证 肾脏疾病的目标。尽管最近取得的突破使我们的技术取得了重大进展 了解多囊肾、局灶节段性肾病等肾脏疾病的发病机制 肾小球硬化症的诊断、管理和治愈这些疾病的新疗法的出现进展缓慢。 为肾病患者提供新疗法的关键一步是获得先进的技术 用于基础发现研究、新发现的途径和早期研究的临床前模型系统 目标验证。在 PMC，我们将利用我们才华横溢的人员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，并将他们聚集在一个 建立并提供便捷访问的行政保护伞：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，可快速设计和生成 新型遗传性肾病模型 3）非遗传性肾病模型，包括高血压、肾病等 移植模型和最先进的小鼠表型分析服务以及 4) 生成和移植服务 对来自肾病患者和 iPSC 肾脏的诱导多能干细胞 (iPSC) 进行基因组编辑 生物样本库。 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， 为肾脏研究人员提供基因组编辑、诱导多能干细胞 (iPSC) 技术和表型分析 实习生。将向全球肾脏界提供所有核心相关服务的外展和获取服务 通过名为 nephro-HUB 的在线门户网站。与企业无缝对接，形成强大协同效应 治疗开发和临床转化 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促进发现和 加速将其转化为肾脏患者的创新疗法。