Mouse Model and Biobank Core (MMBC)

项目摘要 在过去的十年中，小鼠模型已成为转化型多囊肾病（PKD）的主要模型。 这对世界各地的研究人员至关重要。小鼠模型已被用于建立分子生物学模型。 和PKD的细胞基础，以鉴定治疗靶点并在体内验证治疗靶点。总体 小鼠模型和生物库核心（MMBC）的目标是为研究人员消除障碍， PKD小鼠模型在他们的研究。MMBC将通过分发和维护 PKD小鼠模型和试剂，通过分享专业知识和执行和/或提供援助， 使用标准化方案在小鼠中进行临床前试验。此外，MMBC将开发创新鼠标 这些模型可用于了解疾病的发病机制和测试新类别的治疗药物。到 为了实现这些目标，我们提出了以下目标：1）为研究界提供一个遗传学上的 工程活小鼠生物资源2）生成和维护小鼠生物标本库3）协助 使用适当的PKD模型对有希望的候选疗法进行临床前测试4）提供个性化的 咨询和工具的研究人员谁使用PKD小鼠模型在他们的研究和5）生成和 分发一种新的Pkd 2小鼠模型，可用于靶向缺陷的药物的临床前测试。 信道处理和/或功能。总之，完成这些目标将允许小鼠模型和 生物库核心，以协助不同的社区研究人员进行高质量的研究，使用国家的， 本领域的小鼠模型，从而加速多囊肾疾病领域的发现。

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.