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Mouse Model and Biobank Core (MMBC)

小鼠模型和生物样本库核心 (MMBC)

基本信息

批准号：
10058979
负责人：
Patricia Outeda Garcia
金额：
$ 19.98万
依托单位：
UNIVERSITY OF MARYLAND BALTIMORE
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：
2020-08-24 至 2025-06-30
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10058979
关键词：
Alleles Biological Blood Collection Communities Complex Consultations Custom Disease Disease Progression Disease model Enterobacteria phage P1 Cre recombinase Familiarity Gene Silencing Gene Targeting Genetic Engineering Genotype Goals Grant Health Human Image Individual Institution LoxP-flanked allele Maryland Molecular Monitor Mus Pathogenesis Polycystic Kidney Diseases Preclinical Testing Proteins Protocols documentation Reagent Renal function Reporting Research Research Personnel Resources Sampling Services Signal Pathway Standardization Testing Therapeutic Agents Time Tissues Transgenic Organisms Translations United States National Institutes of Health Urine biobank congenic embryo cryopreservation experience human disease in vivo innovation investigator training mouse model novel pathogen preclinical trial prevent recombinase-mediated cassette exchange repository sperm cell targeted agent therapeutic target tool

项目摘要

Project Summary Over the last decade mouse models have become a mainstay of translational polycystic kidney disease (PKD) research and vital to investigators around the world. Mouse models have been used to establish the molecular and cellular basis of PKD, to identify therapeutic targets and to validate treatment targets in vivo. The overarching goal of the Mouse Models and Biobank Core (MMBC) is to dismantle barriers for investigators seeking to use PKD mouse models in their research. The MMBC will facilitate PKD research by distributing and maintaining PKD mouse models and reagents, by sharing expertise and by performing and/or providing assistance with preclinical trials in mice using standardized protocols. In addition, the MMBC will develop innovative mouse models that can be used to understand disease pathogenesis and to test new classes of therapeutic agents. To achieve these goals we propose the following aims: 1) Provide the research community with a genetically engineered live mouse bio-resource 2) Generate and maintain a repository of murine biospecimens 3) Assist in preclinical testing of promising candidate therapies using appropriate PKD models 4) Provide personalized consultation and tools for investigators who use PKD mouse models in their research and 5) Generate and distribute a novel Pkd2 mouse model that can be used for pre-clinical testing of agents that target defective channel processing and/or function. In summary, completion of these aims will allow the Mouse Models and Biobank Core to assist a diverse community of investigators in conducting high quality research using state-of- the-art mouse models thereby accelerating discovery in the field of polycystic kidney disease.

项目概要在过去的十年中，小鼠模型已成为转化性多囊肾病 (PKD) 的支柱研究对世界各地的研究人员至关重要。小鼠模型已被用来建立分子 PKD 的细胞基础，以确定治疗靶点并验证体内治疗靶点。首要的小鼠模型和生物库核心 (MMBC) 的目标是为寻求使用小鼠模型的研究人员消除障碍他们研究中的 PKD 小鼠模型。 MMBC 将通过分发和维护 PKD 研究来促进 PKD 小鼠模型和试剂，通过分享专业知识以及执行和/或提供帮助使用标准化方案在小鼠中进行临床前试验。此外，MMBC还将开发创新型鼠标可用于了解疾病发病机制和测试新型治疗剂的模型。到为了实现这些目标，我们提出以下目标：1）为研究界提供遗传工程化活体小鼠生物资源 2) 生成并维护小鼠生物样本库 3) 协助使用适当的 PKD 模型对有前景的候选疗法进行临床前测试 4) 提供个性化为在研究中使用 PKD 小鼠模型的研究人员提供咨询和工具，以及 5) 生成和分发一种新型 Pkd2 小鼠模型，可用于针对缺陷药物的临床前测试通道处理和/或功能。总之，完成这些目标将使小鼠模型和 Biobank Core 协助多元化的研究人员利用现状进行高质量的研究最先进的小鼠模型从而加速了多囊肾疾病领域的发现。