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

摘要(核心C) UAB-儿童囊性肾脏疾病核心中心(UAB-CCKDCC)的主要任务是与 在U24中央协调站点(U24-CCS)和NIDDK的指导下，PKD联盟将 消除囊性肾脏疾病研究中的障碍，这些障碍正在减缓 改进治疗策略。UAB-CCKDCC已经组建了一个多学科的研究团队， 指导四个紧密集成的资源和面向服务的核心以及一个管理核心。这个 这些核心的共同使命是支持、加速和扩展PKD的基础和翻译研究 联盟成员通过提供CCKD患者的临床数据和生物材料，通过 用于病因分析的CCKD患者相关细胞和动物模型的建立和分布 参与囊泡形成和发展的机制，并通过开发简化的、成本- 为PKD联盟提供有效的渠道，以快速确定减缓囊肿生长的新药的疗效。 UAB-CCKDCC的体内生物测定和模型开发资源(核心C)的任务是 与CCKD相关的动物模型、活体生物传感器和报告系统的生成和分发 途径，以及由这些模型产生的生物试剂。待开发的模型包括生物传感器 多个生物中的记者需要研究CCKD和动物模型中涉及的信号通路 将根据数据生成的具有人类患者突变的广泛CCKD综合征 在UAB-CCKDCC和整个PKD财团的核心A中获得。核心C还将维持一家银行 这些模型中的生物材料用于分发，以促进先导研究，以快速检验新的假说。最后， 由Core C生成和维护的广谱CCKD和报告系统将很容易实现 可用于PKD联盟在UAB-CCKDCC的核心D进行的临床前试验以进行评估 采用高度标准化、经济高效的纵向成像和创新分析的候选疗法 战略。Core C提供的服务和资源及其与其他内核的集成 在UAB-CCKDCC中，将扩大研究活动，使其超出大多数单个实验室的能力范围，并将 通过提供高质量、健壮且可重现的结果来加速对可能的治疗方法的研究 迫切需要为未来的临床试验确定药物的优先顺序，以阻止PKD和其他囊性肾脏疾病。

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.