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

基本信息

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
项目类别：
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

项目摘要

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-Childhood囊性肾脏疾病核心中心（CCKDCC）的目标是与其他U54合作 PKD中心，在U24协调中心站点（U24-CC）的指导下减少囊性障碍物肾脏疾病研究导致PKD财团成员和新型治疗学的发展。 UAB-CCKDCC将有助于解决与小的限制通过提供临床数据和生物材料的访问，可以在个人直觉上获得CCKD患者人群来自来自整个美洲的人类CCKD患者。该中心将集中于资源的发展分析Cilio-Cystic疾病蛋白功能，定位和相互作用。 UAB-CCKDCC已组装一个多学科的研究人员团队，旨在实现这些目标并积极招募新的并建立调查人员到该领域。该中心将生成并为研究人员提供CCKD的患者相关模型并建立方法来利用这些模型来确定候选疗法的疗效缓慢疾病采用标准化，具有成本效益和纵向成像策略的进展。提供的资源 CCKDCC将有助于解决一些最重大的障碍，以减缓治疗性的发展接近并战略性地招募新科学家进入该领域。临床，转化和生物座席资源（核心A）将编译临床数据，遗传信息，以及来自隐性HRFD患者的生物标本，以及启动ADPEDKD的美国节点国际儿童ADPKD的多中心观察研究。该集中资源旨在加速儿童囊性肾脏疾病的发现研究（与核心B和C的伙伴关系）。一体化这些资源在唯一的标识符下，再加上严格的，详细的患者/照料者访谈卫生研究经验将为开发新的，有针对性的介入策略（伙伴关系）与核心D）。核心将在U24-CC的指导下与其他U54-RTCC进行协调 NIDDK，开发和使PKD研究联盟的资源可用。