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针对治疗发现和早期验证的新方法肾脏疾病的靶标。尽管最近的突破导致了我们的重大进步了解肾脏疾病的发病机理，例如多囊性肾脏疾病和局灶性分段肾小球硬化，诊断，管理和治愈这些疾病的新疗法的出现缓慢。为肾脏患者提供新疗法的路径的关键步骤是可用用于基本发现研究的临床前模型系统，新确定的途径和早期的询问目标验证。在PMC中，我们将探讨我们才华横溢的丰富经验和技术专长肾脏调查人员在利用大量的西北资源并将其放到一个下方的同时行政伞建立并轻松访问：1）Numousekidney住房超过50个独特遗传肾脏病小鼠菌株2）一种基因组编辑“礼宾”服务，以快速设计和生成新的遗传肾模型3）非遗传肾脏疾病模型，包括高血压和肾脏移植模型和最先进的鼠标表型服务，以及4）一代人的服务来自肾脏疾病和IPSCKIDNEY患者的诱导多能干细胞（IPSC）的基因组编辑生物库。 PMC核心还将提供动手研讨会和基于网络的鼠标遗传学培训，基因组编辑，诱导多能干细胞（IPSC）技术以及对肾脏研究者和学员。全球肾脏社区将提供外联和访问所有与核心相关的服务通过一个名为Nephro-Hub的在线网络门户网站。无缝集成和与疗法开发和临床翻译Nu-Gokidney核心，飞行员和可行性以及富集计划和创新的肾脏治疗加速器计划将催化发现和加快对肾脏患者的创新疗法的翻译。