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: 10672793
• 负责人: NAEL A MCCARTY
• 金额: $ 109.35万
• 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 2028-06-30
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10672793
• 关键词: Address; Aging; Basic Science; Behavior; Behavioral; Biomedical Engineering; Biomedical Research; Bone Diseases; Budgets; 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; Diet; Direct Costs; Disease; Disease Progression; Endocrinology; Ensure; Environment; Exercise; Extramural Activities; Functional disorder; Funding; Goals; Health; Hepatology; Homeostasis; Informatics; Institution; Knowledge; Life Style; Link; Liver diseases; Longevity; Longitudinal Studies; Lung; Metabolic; Mission; Molecular; National Institute of Diabetes and Digestive and Kidney Diseases; Nutritional; Nutritional status; Outcome; Patients; Persons; Phenotype; Population; Ramp; Research; Research Personnel; Research Project Grants; Research Support; Resources; Science; Scientist; Sleep; Technology; Therapeutic; Toddler; Translations; Universities; base; biomedical scientist; comorbidity; cystic fibrosis related diabetes; data integration; data warehouse; gastrointestinal; glucose metabolism; health equity; innovation; member; mutant; new therapeutic target; novel; novel therapeutic intervention; nutrition; pandemic disease; preventive intervention; programs; small molecule therapeutics; success; translational potential

PROJECT SUMMARY – Overall This renewal proposal seeks to continue 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. Funded with partial budget less than two years ago, the Center has rapidly ramped up activities during these pandemic times and already has had great impact on the CF research community in Georgia. Center research funded by this P30 grant focuses on non-pulmonary aspects of CF disease central to the NIDDK mission, including CF-related diabetes (CFRD), CF-related liver and bone disease, and the impact of CF on nutritional status and GI function. Unique to our Center is the study of the relationship between these co-morbidities and lifestyle/behavioral choices such as diet, exercise, and sleep. The Center's research is focused on and enabled by the ~900 patients followed at the participating institutions. The research base has grown to include 73 investigators, across basic and clinical departments at several institutions, with >$27M in direct costs for CF-related research in the current year. These investigators are addressing issues that are becoming ever more important to the health of people with CF, as successes with small-molecule therapeutics enable 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 integrate data from multiple platforms spanning from basic science to clinical research informatics to enable longitudinal study of patients with the goal of identifying factors associated with disease progression and advancing our understanding of the CF therapeutic landscape. These clinical and research data are incorporated into the novel Georgia CF Data Warehouse.  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 aspects of CF who will bring new expertise that will advance new perspectives on pathophysiology and treatment, and launch new collaborations. We will accomplish these aims by the coordinated activities of three biomedical research Cores:  Diabetes, Endocrinology, & GI/Hepatology Core  Nutrition, Lifestyle, & Behaviors Core  Clinical Research & Informatics Core These research cores are supported by an Administrative Core which ensures the efficient use of resources and strengthens the intellectual environment for research through a robust Enrichment Program. The successful Pilot & Feasibility Program proposes two new projects; both prior projects already have received extramural support. Continued support of the Core Center will ensure 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 该更新提案旨在继续格鲁吉亚囊性纤维化研究和翻译核心 中心（格鲁吉亚CF核心中心），埃默里大学，亚特兰大，奥古斯塔的儿童保健中心 大学和格鲁吉亚理工学院。不到两年前， 中心在这些大流行时期迅速增加了活动，已经对 CF研究社区在格鲁吉亚。该P30赠款资助的中心研究重点是非肺部 NIDDK使命的核心CF疾病方面，包括CF相关糖尿病（CFRD）、CF相关肝脏 以及CF对营养状况和GI功能的影响。我们中心的独特之处在于 这些合并症与生活方式/行为选择（如饮食、锻炼和 睡吧该中心的研究集中在参与研究的约900名患者身上， 机构职能体系该研究基地已发展到包括73名研究人员，横跨基础和临床部门， 几个机构，本年度CF相关研究的直接成本超过2700万美元。这些研究者 正在解决对CF患者的健康变得越来越重要的问题， 与小分子治疗剂一起使用能够使CF群体“老化”。在光谱的另一端是 在幼儿和幼儿中进行的关于这些合并症的发生的研究，这些合并症有可能 制定早期预防措施。我们整合了来自多个平台的数据， 临床研究信息学，以实现对患者的纵向研究， 与疾病进展相关，并促进我们对CF治疗前景的理解。这些 临床和研究数据被整合到新型的格鲁吉亚CF数据仓库中。 目标1：为CF生物医学研究提供一个强大的，协调良好的环境。 目标2：促进和扩大CF非肺部并发症的资助研究。 目标3：吸引新的研究者专注于CF的非肺部方面，他们将带来新的专业知识 这将推进病理生理学和治疗的新观点，并开展新的合作。 我们将通过三个生物医学研究核心的协调活动来实现这些目标： 糖尿病、内分泌学和GI/肝病学核心 营养、生活方式和行为核心 这些研究中心由一个行政中心提供支持，行政中心确保有效利用资源， 通过强有力的强化计划加强研究的知识环境。试点成功 可行性方案提出了两个新项目;两个先前的项目已经得到了校外支持。 核心中心的持续支持将确保一个专注于非肺部CF的充满活力的研究企业 疾病，发现新的疾病机制，有可能转化为治疗策略。