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

UAB 儿童囊性肾病核心中心 (UAB-CCKDCC) - 行政核心

• 批准号: 10685972
• 负责人: Bradley K. Yoder
• 金额: $ 32.27万
• 依托单位: UNIVERSITY OF ALABAMA AT BIRMINGHAM
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-07-20 至 2025-06-30
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10685972
• 关键词: Acceleration; Animal Model; Basic Science; Benchmarking; Biocompatible Materials; Biological Models; Biometry; Biosensor; Cell model; Childhood; Clinical; Clinical Data; Clinical Trials; Collaborations; Communities; Cyst; Cystic Kidney Diseases; Cystic kidney; Data Analyses; Defect; Development; Disease; Disease Progression; Ensure; Experimental Designs; Family; Future; Generations; Genetic; Genetic Materials; Genetic Models; Goals; In Vitro; Individual; Informatics; Interdisciplinary Study; Kidney; Kidney Diseases; Laboratories; Legal; Logistics; Methodology; Mission; Modeling; Mutation; National Institute of Diabetes and Digestive and Kidney Diseases; Outcome; Outcomes Research; Pathogenesis; Pathogenicity; Pathway interactions; Patients; Pharmaceutical Preparations; Phenotype; Preclinical Testing; Proteins; Reporter; Reproducibility; Reproducibility of Results; Research; Research Activity; Research Personnel; Resource Development; Resource Sharing; Resources; Scientist; Services; Site; Standardization; Structure; System; Testing; Training and Education; Translational Research; Validation; Vision; Work; clinical practice; cost effective; improved; in vivo; innovation; longitudinal analysis; material transfer agreement; member; multidisciplinary; programs; protein function; recruit; serial imaging; therapeutic evaluation; treatment strategy

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.

摘要（管理核心） UAB儿童囊性肾病核心中心（UAB-CCKDCC）的主要使命是 在U24中央协调中心（U24-CCS）的指导下，与PKD联盟合作， NIDDK，以消除阻碍囊性肾病研究进展的障碍， 开发针对囊性肾病的改进和创新治疗策略。UAB- CCKDCC组建了一个多学科的研究团队，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沿着管理核心。的 这些核心的总体和集体使命是支持、加速和扩展基础和转化 PKD联盟成员正在进行的研究活动。该中心将重点发展 资源来分析纤毛囊性疾病蛋白质的功能，定位和相互作用，并评估如何 这些功能的缺陷导致了囊肿发生和发展的致病机制。 该中心将通过提供随时访问CCKD的临床数据和生物材料来完成其使命 通过开发和分发患者相关细胞和CCKD动物模型， 通过开发利用这些模型的方法来确定候选疗法的疗效 使用标准化的、具有成本效益的纵向成像和分析来减缓疾病进展 战略布局中心沿着提供的服务和资源， 核心将扩大研究活动超出了大多数个别实验室的能力。将加快 通过提供高质量，强大， 和可重复的结果。这些是迫切需要的，以优先考虑未来临床试验的药物，以阻止PKD， 其他囊性肾疾病。 行政核心是履行阿拉伯联合酋长国-刚果民主共和国发展协调委员会使命的关键。行政核心将 提供中心的整体结构，以确保每个核心都朝着共同的目标努力， 目的是为CCKD开发更好的治疗方案。行政核心将负责财务 协调努力，确保每个核心的活动与其他核心的活动保持一致 U 54-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。行政核心将制定基准并确保实现这些基准， 向PKD联盟提供的资源和服务，它们是经过认证的，健壮的， 及时交付，并且不因材料转让协议或其他法律的障碍而延迟。