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

UAB 儿童囊性肾病核心中心 (UAB-CCKDCC)

• 批准号: 10455717
• 负责人: Bradley K. Yoder
• 金额: $ 76.89万
• 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/10455717
• 关键词: Address; Affect; Animal Model; Autosomal Recessive Polycystic Kidney; Basic Science; Biocompatible Materials; Biological Assay; Cell model; Childhood; Cilia; Clinical; Clinical Data; Clinical Trials; Collaborations; Consumption; Cyst; Cystic Kidney Diseases; Cystic kidney; Data; Defect; Development; Disease; Disease Progression; Ensure; Environment; Etiology; Functional disorder; Future; Generations; Genetic; Genetic Models; Goals; Grant; Heart Diseases; Human; In Vitro; Individual; International; Kidney Diseases; Knowledge; Laboratories; Localized Disease; Meckel-Gruber syndrome; Medical Genetics; Medicine; Methodology; Mission; Modeling; Morbidity - disease rate; Mutation; National Institute of Diabetes and Digestive and Kidney Diseases; Nephronophthisis; Outcome; Pathogenesis; Pathogenicity; Pathway interactions; Patients; Pharmaceutical Preparations; Preclinical Testing; Productivity; Proteins; Reagent; Reporter; Reproducibility; Research; Research Activity; Research Personnel; Resource Development; Resources; Scientist; Seeds; Sensory; Services; Signal Transduction; Site; Standardization; System; Testing; Time; Toxicology; Training; Translational Research; Translations; Validation; Vision; Work; base; cost; cost effective; data repository; effective therapy; genetic information; improved; in vitro Bioassay; in vitro Model; in vivo; innovation; longitudinal analysis; member; mortality; multidisciplinary; next generation; pre-clinical; prevent; programs; protein function; ranpirnase; recruit; repository; screening; serial imaging; therapeutic development; tool; treatment strategy

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.

摘要（总体） UAB-儿童囊性肾病核心中心 (UAB-CCKDCC) 的总体目标是 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，旨在消除囊性肾病相关研究中阻碍进展的障碍 开发针对囊性肾病的改进和创新治疗策略。为了实现这一点 为了实现这一目标，UAB-CCKDCC 组建了一支由研究人员和临床医生组成的多学科团队，他们将 指挥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 这些核心的集体使命是支持、加速和扩大基础研究和转化研究活动 由 PKD 联盟成员执行。该中心将重点开发资源 分析纤毛囊肿疾病蛋白的功能、定位和相互作用以及这些功能的缺陷如何 有助于涉及囊肿发生和进展的致病机制。该中心将完成其 通过提供对 CCKD 患者的临床数据和生物材料的现成访问权限， CCKD 患者相关细胞和动物模型的开发和分布，并通过 开发方法来利用这些模型来确定候选疗法的功效以减缓 使用标准化、具有成本效益的纵向成像和分析策略进行疾病进展。这 中心提供的服务和资源以及各个机构之间的整合 核心将扩大研究活动，远远超出大多数单个实验室的能力，并将加速 通过提供高质量、 需要稳健且可重复的结果来确定未来临床试验中药物的优先顺序。