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

摘要(管理核心) UAB-儿童囊性肾脏疾病核心中心(UAB-CCKDCC)的主要任务是 在U24中央协调站点(U24-CCS)的指导下，与PKD联盟合作 NIDDK，以消除囊性肾脏疾病研究中的障碍，这些障碍正在减缓 改进和创新囊性肾脏疾病的治疗策略。UAB- CCKDCC组建了一支由多学科研究人员组成的团队，每个团队都有强大的研究计划，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 这些核心的总体和集体使命是支持、加速和扩展基本和转换 由PKD联盟成员开展的研究活动。该中心将专注于发展 分析睫状囊性疾病蛋白功能、定位和相互作用的资源，并评估如何 这些功能的缺陷导致了囊性病变的发生和发展。 该中心将通过随时访问CCKD的临床数据和生物材料来完成其使命 通过与患者相关的CCKD细胞和动物模型的发展和分布，以及 通过方法学的发展，利用这些模型来确定候选疗法的疗效 使用标准化、经济高效的纵向成像和分析来减缓疾病进展 战略。中心提供的服务和资源随着我们 CORE将扩大研究活动，超出大多数单独实验室的能力范围。它将加速 对囊性肾脏疾病的病因和可能的治疗方法的研究步伐，通过提供高质量、强大的、 和可重现的结果。这些都是迫切需要的，以确定未来临床试验的药物优先顺序，以阻止PKD和 其他囊性肾脏疾病。 履行UAB-CKDCC使命的关键是行政核心。行政核心意志 提供中心的整体结构，以确保每个核心都在朝着共同的方向工作 目标是为CCKD开发更好的治疗方案。行政核心将负责财务 和科学监督，协调努力，确保每个核心的活动与另一个核心的活动保持一致 U54-PKD中心(RTCC)，每个核心的重点反映了U24-CCS确定的优先事项 和NIDDK。行政核心将建立基准并确保实现这些基准，该中心 提供给PKD联盟的资源和服务是经过身份验证的、健壮的 及时交付，不会因材料转让协议或其他法律障碍而延误。

ABSTRACT (ADMINISTRATIVE CORE) The primary mission of the UAB-Childhood Cystic Kidney Disease Core Center (UAB-CCKDCC) is to work with the PKD Consortium, under the guidance 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 and innovative treatment strategies for cystic kidney disorders. The UAB- CCKDCC has assembled a multidisciplinary team of researchers, each with strong research programs, who will direct four tightly integrated resource and service-oriented Cores along with an Administrative Core. The overarching and 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 to assess 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 of our Cores will expand research activities beyond that capable in most individual laboratories. It will accelerate the pace of research into causes and possible cures of cystic kidney diseases by providing for high quality, robust, and reproducible outcomes. These are critically needed to prioritize drugs for future clinical trials to halt PKD and other cystic kidney disorders. Essential to fulfilling the mission of the UAB-CKDCC is the Administrative Core. The Administrative Core will provide for the Center’s overall structure to make sure that each of the Cores is working toward the common goal of developing better treatments options for CCKD. The Administrative Core will be responsible for financial and scientific oversight, coordinate efforts to ensure that each Core’s activities are aligned with those of the other U54-PKD Centers (RTCC), and that the focus of each Core reflects the priorities established by the U24-CCS and NIDDK. The Administrative Core will establish benchmarks and ensure they are achieved, that Center resources and services being provided to the PKD Consortium, that they are authenticated, robust, and are delivered in a timely manner, and are not delayed by material transfer agreements or other legal hurdles.