UAB Childhood Cystic Kidney Disease Core Center (UAB-CCKDCC)

摘要(总体) UAB-儿童囊性肾脏疾病核心中心(UAB-CCKDCC)的总体目标是在 在U24中央协调站点(U24-CCS)的指导下，与PKD联盟建立伙伴关系 NIDDK，以消除囊性肾脏疾病相关研究中阻碍进展的障碍 改进和创新囊性肾脏疾病的治疗策略。要做到这一点 目标，UAB-CCKDCC已经组建了一个由研究人员和临床医生组成的多学科团队，他们将 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、加速和扩展基础研究和翻译研究活动 由PKD联盟成员执行。该中心将专注于开发资源以 分析睫状囊性疾病蛋白的功能、定位和相互作用，以及这些功能的缺陷 有助于囊性病变发生和发展的致病机制。该中心将完成其 使命通过提供CCKD患者的临床数据和生物材料，通过 CCKD患者相关细胞和动物模型的发展和分布，并通过 开发方法学，利用这些模型来确定减慢候选疗法的疗效 使用标准化、经济高效、纵向成像和分析策略的疾病进展。这个 中心提供的服务和资源，以及每个中心之间的集成 CORE将扩大研究活动，远远超出大多数单个实验室的能力范围，并将加速 通过提供高质量的研究来研究囊性肾脏疾病的原因和可能的治疗方法， 为未来的临床试验确定药物的优先顺序所需的稳健、可重复性的结果。

ABSTRACT (OVERALL) The overarching goal of the UAB-Childhood Cystic Kidney Disease Core Center (UAB-CCKDCC) is to work in partnership with the PKD Consortium, under the direction of the U24 Central Coordinating Site (U24-CCS) and NIDDK, to eliminate obstacles in cystic kidney disease related research that are hindering progress toward the development of improved and innovative treatment strategies for cystic kidney disorders. To accomplish this goal, the UAB-CCKDCC has assembled a multidisciplinary team of researchers and clinicians who will 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 activities being performed by PKD Consortium members. The Center will focus on the development of resources to analyze cilio-cystic disease protein function, localization, and interactions and how defects in these functions contribute to the pathogenic mechanisms involved in cyst initiation and progression. The Center will complete its mission through providing ready access to clinical data and biomaterial from CCKD patients, through the development and distribution of patient-relevant cell and animal models of CCKD, and through the development of methodology to utilize these models to ascertain the efficacy of candidate therapies to slow disease progression using a standardized, cost-effective, and longitudinal imaging and analysis strategies. The services and resources being made available by the Center along with the integration between each of the Cores will expand research activities well beyond that capable in most individual laboratories and will accelerate the pace of research into causes and possible cures of cystic kidney diseases by providing for high quality, robust, and reproducible outcomes needed to prioritize drugs for future clinical trials.