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Biomedical Research Core 2 - Rodent Models & Drug Testing Core

生物医学研究核心 2 - 啮齿动物模型

基本信息

批准号：
10059767
负责人：
Pamela Vivian Tran
金额：
$ 23.06万
依托单位：
UNIVERSITY OF KANSAS MEDICAL CENTER
依托单位国家：
美国
项目类别：
财政年份：
2020
资助国家：
美国
起止时间：
2020-07-15 至
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10059767
关键词：
Address Agreement Alleles Amino Acids Animals Autosomal Dominant Polycystic Kidney Biocompatible Materials Biological Assay Biological Markers Biomedical Research Breeding C-terminal Catalogs Cell Culture Techniques Cell Nucleus Cell model Cells Cilia Co-Immunoprecipitations Communities Complement Consult Consultations Contracts Cryopreservation Cyst Data Data Analyses Databases Development Dietary Intervention Disease Drug Design Drug Modelings Drug or chemical Tissue Distribution Educational workshop Engineering Essential Drugs Exhibits Genes Genetic Models Genetic study Goals In Vitro Institutes Institution Investigation Kansas Kidney Knock-out Laboratories Learning Legal Maps Medical Medical center Modeling Molecular Target Multiprotein Complexes Mus Mutant Strains Mice Mutation N-terminal Organ Culture Techniques Outcome Pattern Pharmaceutical Preparations Pre-Clinical Model Process Reagent Renal Tissue Research Research Personnel Resources Rodent Model S-nitro-N-acetylpenicillamine Services Translations Universities Visualization Work biomarker identification commercialization design drug testing experimental study gene therapy human disease in vivo innovation insight material transfer agreement mouse model next generation novel polycystic kidney disease 1 protein pre-clinical ranpirnase repository response service intervention single-cell RNA sequencing structural genomics testing services therapeutic evaluation therapeutic target tool trafficking transcriptome sequencing transcriptomics

项目摘要

Project Summary – BRC2 The goal of the Rodent Models & Drug Testing Core is to combine the development of innovative mouse models and exploration of cell-specific gene networks with essential drug-testing services to propel the PKD community forward in our understanding of polycystin function, and in identification and evaluation of therapeutic targets. Novel in vivo tools will be provided to address significant barriers in the field, such as the paucity of genetic models that provide insight into potential outcomes of gene therapy, the lack of in vivo reagents to visualize subcellular localization of endogenous polycystins, and the lack of a pre-clinical model that exhibits the autosomal dominant inheritance of the human disease. Using the unparalleled expertise of KUMC in drug design, drug testing and commercialization, a drug testing service will be offered to meet the demands of much needed drug studies in the field. Additionally, single nucleus transcriptomic maps of kidneys of commonly used mouse models of ADPKD will be provided to help with the identification of biomarkers and therapeutic targets. The specific aims are to: 1) develop next-generation mouse models to study the genetics and function of Pkd1 and other cystogenic genes; 2) maintain a live-animal repository of PKD rodent models and tissue for distribution; 3) provide drug testing and dietary intervention services in PKD rodent models; and 4) provide longitudinal single- nucleus RNA-seq transcriptomics maps from fast- and slow-progressing Pkd1-mutant mice. Further, the Core will provide consultation services and participate in an annual workshop to help researchers learn about the various utilities conferred by different PKD models for better understanding of disease processes and for the design of pre-clinical drug-testing studies. Collectively, these services will benefit investigators studying any aspect of the disease, and will transform investigations of polycystin function, disease mechanisms, and treatments for the national PKD research community.

项目摘要 – BRC2 啮齿动物模型和药物测试核心的目标是将创新小鼠的开发结合起来细胞特异性基因网络的模型和探索以及基本药物测试服务以推动 PKD 社区在我们对多囊蛋白功能的理解以及治疗方法的识别和评估方面取得了进步目标。将提供新颖的体内工具来解决该领域的重大障碍，例如缺乏遗传模型可以深入了解基因治疗的潜在结果，但缺乏体内试剂可视化内源性多囊蛋白的亚细胞定位，并且缺乏展示内源性多囊蛋白的临床前模型人类疾病的常染色体显性遗传。利用 KUMC 在药物方面无与伦比的专业知识设计、药物测试和商业化，将提供药物测试服务，以满足许多人的需求需要该领域的药物研究。此外，常用肾脏的单核转录组图谱将提供 ADPKD 小鼠模型来帮助识别生物标志物和治疗靶点。具体目标是：1）开发下一代小鼠模型来研究Pkd1的遗传学和功能和其他囊原基因； 2) 维护 PKD 啮齿动物模型和组织的活体储存库以供分发； 3）为多囊肾啮齿动物模型提供药物检测和饮食干预服务； 4）提供纵向单快速和慢速进展 Pkd1 突变小鼠的核 RNA-seq 转录组图谱。此外，核心将提供咨询服务并参加年度研讨会，帮助研究人员了解不同 PKD 模型赋予的各种实用性，可以更好地了解疾病过程并促进临床前药物测试研究的设计。总的来说，这些服务将有利于研究人员研究任何疾病的一个方面，并将改变多囊蛋白功能、疾病机制和疾病的研究国家 PKD 研究界的治疗方法。