Preclinical Models Core (Core A)

临床前模型核心（核心 A）

基本信息

批准号：
10203938
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
项目类别：
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/10203938
关键词：
Biological Biological Assay Biological Models 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 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

项目摘要

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，重点关注疗法的发现和早期验证尽管最近的突破已经导致我们在肾脏疾病方面取得了重大进展。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药物用于基础发现研究、新发现的途径和早期研究的临床前模型系统在 PMC 中，我们将利用我们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的遗传模型3）非遗传性肾病模型，包括高血压和肾病移植模型和最先进的小鼠表型分析服务以及 4) 生成和对来自肾病患者和 iPSC 肾脏的诱导多能干细胞 (iPSC) 进行基因组编辑 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) 技术和表型分析将向全球肾脏界提供所有核心相关服务的外展服务。通过名为 nephro-HUB 的在线门户网站无缝集成并具有强大的协同作用。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。