UAB Childhood Cystic Kidney Disease Core Center (UAB-CCKDCC) - In Vivo Bioassay and Model Development Resource

摘要（核心C） UAB儿童囊性肾病核心中心（UAB-CCKDCC）的主要使命是与 PKD联合体在U24中央协调站（U24-CCS）和NIDDK的指导下， 消除囊性肾病研究中阻碍开发进展的障碍 改善治疗策略。UAB-CCKDCC组建了一个多学科研究团队， 指导四个紧密集成的资源和面向服务的核心沿着管理核心。的 这些核心的集体使命是支持、加速和扩大PKD的基础研究和转化研究 联盟成员通过提供CCKD患者的临床数据和生物材料， CCKD患者相关细胞和动物模型的开发和分布，以分析致病性 参与囊肿发生和进展的机制，并通过发展精简，成本- 为PKD联盟提供有效的管道，以快速确定新药减缓囊肿生长的功效。 UAB-CCKDCC中的体内生物测定和模型开发资源（核心C）的任务是 用于CCKD相关的动物模型、体内生物传感器和报告系统的产生和分发 途径和从这些模型产生的生物试剂。待开发的模型包括生物传感器 研究CCKD和动物模型中涉及的信号通路所需的多种生物体中的报告基因 用于基于数据生成的具有人类患者突变的广泛CCKD综合征 从UAB-CCKDCC的核心A和整个PKD联盟获得。核心C也将维持一个银行 从这些模型中提取生物材料进行分配，以促进试点研究，快速测试新的假设。最后， 由核心C产生和维护的广泛的CCKD和报告系统将很容易制造 可供PKD联盟用于在UAB-CCKDCC核心D中进行的临床前试验，以评价 候选疗法采用高度标准化、成本效益高的纵向成像和创新分析 战略布局核心C提供的服务和资源及其与其他核心的集成 在UAB-CCKDCC中，将扩展研究活动，超出大多数单个实验室的能力， 通过提供高质量、稳健和可重复的结果，加速对可能的治疗方法的研究， 因此，迫切需要优先考虑未来临床试验的药物，以阻止PKD和其他囊性肾病。

ABSTRACT (CORE C) The primary mission of the UAB-Childhood Cystic Kidney Disease Core Center (UAB-CCKDCC) is to work with the PKD Consortium, under the direction of the U24 Central Coordinating Site (U24-CCS) and NIDDK, to eliminate obstacles in cystic kidney disease research that are slowing progress toward the development of improved treatment strategies. The UAB-CCKDCC has assembled a multidisciplinary team of researchers that direct four tightly integrated resource and service-oriented Cores along with an Administrative Core. The collective mission of these Cores is to support, accelerate, and expand basic and translational research by PKD Consortium members by providing access to clinical data and biomaterial from CCKD patients, through the development and distribution of patient-relevant cell and animal models of CCKD to analyze pathogenic mechanisms involved in cyst initiation and progression, and through the development of streamlined, cost- effective pipelines for the PKD Consortium to rapidly ascertain the efficacy of new drugs to slow cyst growth. The In Vivo Bioassay and Model Development Resource (Core C) within the UAB-CCKDCC is tasked with generating and distributing animal models, in vivo biosensors and reporter systems for CCKD associated pathways, and biological reagents generated from these models. Models to be developed include biosensors and reporters in multiple organisms needed to study signaling pathways involved in CCKD and animal models for a wide range of CCKD syndromes with human patient mutations that will be generated based on data obtained in Core A in the UAB-CCKDCC and from the overall PKD Consortium. Core C will also maintain a bank of biomaterials from these models for distribution to promote pilot studies to rapidly test new hypotheses. Finally, the wide spectrum of CCKD and reporter systems generated and maintained by Core C will be made readily available to PKD Consortium for preclinical trails being conducted in Core D of the UAB-CCKDCC to evaluate candidate therapies using highly standardized, cost-effective, and longitudinal imaging and innovative analysis strategies. The services and resources being made available by Core C and it’s integration with the other Cores in the UAB-CCKDCC will expand research activities beyond what is capable in most individual labs and will accelerate research into possible cures by providing for high quality, robust, and reproducible outcomes that are critically needed to prioritize drugs for future clinical trials to halt PKD and other cystic kidney disorders.