UAB Childhood Cystic Kidney Disease Core Center (UAB-CCKDCC) - Clinical, Translational, and Biorepository Resource

UAB 儿童囊性肾病核心中心 (UAB-CCKDCC) - 临床、转化和生物样本库资源

• 批准号: 10685977
• 负责人: LISA MARIE GUAY-WOODFORD
• 金额: $ 15.47万
• 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/10685977
• 关键词: Abbreviations; Acceleration; Address; Adult; Affect; American; Americas; Autosomal Dominant Polycystic Kidney; Autosomal Recessive Polycystic Kidney; Award; Biocompatible Materials; Biological; Breast Fibrocystic Disease; Caregivers; Child; Childhood; Clinical; Clinical Data; Clinical Research; Collaborations; Conceptions; Cystic Kidney Diseases; DNA; Data; Databases; Development; Diagnostic; Disease; Disease Progression; Epithelial Cells; Foundations; Genetic; Genotype; Geography; Goals; Guidelines; Health; Human; Individual; International; Intervention; Interview; Intuition; Kidney; Kidney Diseases; Knowledge; Methodology; Modeling; Molecular; Morbidity - disease rate; National Institute of Diabetes and Digestive and Kidney Diseases; Natural History; Observational Study; Pathogenesis; Pathogenicity; Pathway interactions; Patient Preferences; Patient Recruitments; Patients; Phase; Prognostic Marker; Rare Diseases; Recruitment Activity; Research; Research Institute; Research Personnel; Resource Development; Resources; Scientist; Site; Specimen; Standardization; Tissue Banks; Tissues; Translational Research; Universities; Urine; Voice; Washington; Work; acronyms; biobank; clinical center; clinical database; clinical decision-making; clinically significant; cost effective; design; disease prognosis; effective therapy; evidence base; experience; genetic information; human tissue; individual patient; member; mortality; multidisciplinary; novel therapeutics; patient oriented; patient population; patient retention; predictive marker; prognostic; protein function; recruit; renal epithelium; repository; retention rate; serial imaging; targeted treatment; therapeutic development; tissue resource; translational study

ABSTRACT It is the goal of the UAB-Childhood Cystic Kidney Disease Core Center (CCKDCC) to work with the other U54 PKD Centers, under the direction of the U24 Coordinating Center Site (U24-CCS) to reduce obstacles in cystic kidney disease research leading to more rapid translational studies by PKD Consortium Members and development of novel therapeutics. The UAB-CCKDCC will help address the limitations associated with the small CCKD patient population available at individual intuitions by providing access to clinical data and biomaterial from human CCKD patients from across the Americas. The Center will focus on the development of resources to analyze cilio-cystic disease protein function, localization, and interactions. The UAB-CCKDCC has assembled a multidisciplinary team of researchers to carry out these goals and actively recruit new and established investigators to the field. The Center will generate and provide researchers with patient relevant models of CCKD and establish methodology to utilize these models to ascertain the efficacy of candidate therapies to slow disease progression using a standardized, cost-effective, and longitudinal imaging strategy. The resources provided by the CCKDCC will help address several of the most significant hurdles slowing the development of therapeutic approaches and strategically recruit new scientists into the field. The Clinical, Translational, and Biorepository Resource (Core A) will compile clinical data, genetic information, and biological specimens from patients with recessive HRFD, as well as launch the US node of ADPedKD, an international, multicenter observational study of childhood ADPKD. This centralized resource is designed to accelerate discovery research in childhood cystic kidney diseases (partnership with Cores B and C). Integration of these resources under a unique identifier, combined with rigorous, detailed interviews of patient/caregiver health research experiences will inform the development of new, targeted interventional strategies (partnership with Core D). The Core will coordinate efforts with other U54-RTCCs, under direction of the U24-CCS and NIDDK, to develop and make the resources available to the PKD Research Consortium.

抽象的 UAB-儿童囊性肾病核心中心 (CCKDCC) 的目标是与其他 U54 合作 PKD 中心，在 U24 协调中心站点 (U24-CCS) 的指导下，减少囊性障碍 肾脏疾病研究导致 PKD 联盟成员进行更快速的转化研究 新疗法的开发。 UAB-CCKDCC 将帮助解决与小型设备相关的限制 通过提供临床数据和生物材料，可以凭个人直觉获得 CCKD 患者群体 来自美洲各地的人类 CCKD 患者。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。 UAB-CCKDCC 已集结 一个由多学科研究人员组成的团队来实现这些目标并积极招募新的和已建立的研究人员 调查人员前往实地。该中心将生成并向研究人员提供 CCKD 患者相关模型 并建立利用这些模型来确定候选疗法减缓疾病的功效的方法 使用标准化、具有成本效益的纵向成像策略进行进展。提供的资源 CCKDCC 将帮助解决减缓治疗发展的几个最重要的障碍 方法并战略性地招募新科学家进入该领域。 临床、转化和生物样本库资源（核心 A）将编译临床数据、遗传信息、 以及隐性 HRFD 患者的生物样本，并启动 ADPedKD 美国节点，这是一个 儿童 ADPKD 的国际多中心观察研究。这种集中的资源旨在 加速儿童囊性肾病的发现研究（与 Cores B 和 C 合作）。一体化 这些资源具有唯一的标识符，并结合对患者/护理人员进行严格、详细的访谈 健康研究经验将为制定新的、有针对性的干预策略提供信息（伙伴关系 与核心D）。核心将在 U24-CCS 的指导下与其他 U54-RTCC 协调工作， NIDDK，开发并向 PKD 研究联盟提供资源。