Georgia Cystic Fibrosis Research and Translation Core Center

乔治亚州囊性纤维化研究与转化核心中心

• 批准号: 10260479
• 负责人: NAEL A MCCARTY
• 金额: $ 59.01万
• 依托单位: EMORY UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-09-10 至 2023-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10260479
• 关键词: Address; Aging; Basic Science; Behavior; Behavioral; Biomedical Engineering; Biomedical Research; Center Core Grants; Child; Child health care; Clinical; Clinical Informatics; Clinical Research; Collaborations; Communities; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Data; Development; Diabetes Mellitus; Direct Costs; Disease; Disease Progression; Elements; Ensure; Environment; Functional disorder; Funding; Goals; Health; Homeostasis; Informatics; Institutes; Institution; Life Style; Link; Liver diseases; Longitudinal Studies; Lung; Metabolic; Mission; Molecular; National Institute of Diabetes and Digestive and Kidney Diseases; Nutritional; Nutritional status; Outcome; Patients; Population; Research; Research Personnel; Research Support; Resources; Science; Scientist; Sleep; Source; Technology; Therapeutic; Toddler; Translations; Universities; base; biomedical scientist; comorbidity; cystic fibrosis related diabetes; diet and exercise; gastrointestinal; glucose metabolism; member; multiple omics; mutant; new therapeutic target; novel therapeutic intervention; nutrition; preventive intervention; program dissemination; programs; small molecule therapeutics; success

PROJECT SUMMARY – Overall This application proposes to establish the Georgia Cystic Fibrosis Research and Translation Core Center (the Georgia CF Core Center) at Emory University, Children’s Healthcare of Atlanta, Augusta University, and the Georgia Institute of Technology. This center will leverage activities at these institutions to solve critical problems associated with CF. Georgia CF Core Center research will focus on non-pulmonary aspects of CF disease central to the mission of the NIDDK, including CF-related diabetes (CFRD) and the impact of CF on nutritional status, GI function, and liver disease. Unique to our center will be the study of the relationship between these co-morbidities and lifestyle/behavioral choices such as diet, exercise, and sleep. The proposed basic, clinical, and translational CF research supported via this application is centered around the 900 patients followed at the participating institutions. The research base is comprised of 61 investigators, across both basic and clinical departments at four institutions, with a total of $15,150,207 in direct costs for CF-related research in the current year. These investigators will address issues that are becoming ever more important to the health of people with CF, as successes with small-molecule therapeutics makes it possible for us to anticipate an “aging” CF population. On the opposite end of the spectrum are studies in toddlers and young children on the genesis of these co-morbidities which have the potential for defining early preventive interventions. We will integrate data from multiple platforms spanning from basic science to ‘omics and clinical research informatics to enable longitudinal study of patients with the goal of identifying factors associated with disease progression and advancing our understanding of current CF therapeutic and lifestyle approaches.  Aim 1: To provide a robust, well-coordinated environment for CF biomedical research.  Aim 2: To facilitate and enable the expansion of the funded research in non-pulmonary complications of CF.  Aim 3: To attract new investigators focused on non-pulmonary complications of CF who will bring new expertise that will advance new perspectives on pathophysiology and treatment. We will accomplish these aims by the coordinated activities of four biomedical research Cores:  Diabetes, GI, and Nutrition Core  Lifestyle and Behaviors Core  Molecular Multi-Omics Core  Clinical Research and Informatics Core These research cores will be supported by an Administrative Core which will ensure the efficient use of resources. This core will also strengthen the intellectual environment for research through a robust Enrichment and Dissemination Program as well as a Pilot & Feasibility Program with two projects proposed. Support of the proposed P30 program will create a vigorous research enterprise focused on non-pulmonary CF disease, uncovering new disease mechanisms with potential for translation to therapeutic strategies.

项目概要——总体 该申请提议建立乔治亚州囊性纤维化研究和翻译核心 埃默里大学亚特兰大儿童保健中心（佐治亚 CF 核心中心），奥古斯塔 该中心将利用这些机构的活动来促进大学和佐治亚理工学院的发展。 解决与 CF 相关的关键问题。佐治亚 CF 核心中心的研究将集中于非肺部疾病。 CF 疾病的各个方面对 NIDDK 的使命至关重要，包括 CF 相关糖尿病 (CFRD) 和 CF 对营养状况、胃肠道功能和肝脏疾病的影响是我们中心的独特研究。 这些共病与生活方式/行为选择（例如饮食、运动和饮食）之间的关系 通过该应用程序支持的拟议基础、临床和转化 CF 研究以睡眠为中心。 该研究基地由 61 家机构组成，共跟踪了约 900 名患者。 四个机构的基础和临床部门的研究人员，总计 15,150,207 美元 这些研究人员将解决当年 CF 相关研究的直接成本。 随着小分子疗法的成功，对于 CF 患者的健康变得越来越重要 使我们有可能预测“老龄化”的 CF 人群。 对幼儿和幼儿进行的关于这些合并症的起源的研究有可能 我们将整合来自多个平台的数据，从基本的到早期的预防干预措施。 科学到“组学和临床研究信息学”，以实现对患者的纵向研究，其目标是 确定与疾病进展相关的因素并增进我们对当前 CF 的理解 治疗和生活方式方法。  目标 1：为 CF 生物医学研究提供一个强大、协调良好的环境。  目标 2：促进并扩大 CF 非肺部并发症的资助研究。 目标 3：吸引新的研究人员关注 CF 非肺部并发症，他们将带来新的研究成果 专业知识将推动病理生理学和治疗的新观点。 我们将通过四个生物医学研究核心的协调活动来实现这些目标：  糖尿病、胃肠道和营养核心  生活方式和行为核心  分子多组学核心  临床研究和信息学核心 这些研究核心将得到行政核心的支持，该核心将确保有效利用 该核心还将通过强有力的丰富来加强研究的智力环境。 和传播计划以及试点和可行性计划，其中提出了两个项目的支持。 拟议的 P30 计划将创建一个专注于非肺 CF 疾病的充满活力的研究企业， 揭示新的疾病机制，并有可能转化为治疗策略。