Biofluid Core

生物流体核心

基本信息

批准号：
10208704
负责人：
ADAM L. BOXER
金额：
$ 86.8万
依托单位：
MAYO CLINIC ROCHESTER
依托单位国家：
美国
项目类别：
财政年份：
2019
资助国家：
美国
起止时间：
2019-09-15 至 2025-06-30
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10208704
关键词：
Academia Address Alzheimer&apos s disease related dementia Biological Markers Blood Blood specimen C9ORF72 Cataloging Cell Line Clinic Clinical Clinical Pathology Clinical Trials Collaborations Collection Communities Complement 3b DNA Data Databases Diagnosis Dipeptides Disease Early Diagnosis Equipment and supply inventories Experimental Models Family Frontotemporal Lobar Degenerations Genes Goals High Prevalence Industry Industry Collaboration Inflammatory Interleukin-18 Interleukin-6 Intervention Life Light Liquid substance Measurement Measures Messenger RNA Monitor Mutation National Institute of Neurological Disorders and Stroke Nerve Degeneration PGRN gene Participant Pathologic Pathology Patients Peripheral Blood Mononuclear Cell Plasma Population Prevention trial Proteins Research Personnel Role Sampling Sensitivity and Specificity Specimen Standardization Stratification Syndrome System TNF gene Therapeutic Washington accurate diagnosis autosomal dominant mutation behavioral variant frontotemporal dementia biobank biomarker discovery carrier status clinical trial participant data management data sharing drug discovery high throughput screening improved induced pluripotent stem cell intervention effect neurofilament new therapeutic target novel pharmacodynamic biomarker repository sample collection targeted biomarker tau Proteins therapeutic development therapeutic target tool treatment effect

项目摘要

ABSTRACT – ARTFL LEFFTDS Longitudinal FTLD: BIOFLUID CORE Successful treatment of Frontotemporal Lobar Degeneration (FTLD) will require early and accurate diagnosis, and the ability to monitor the effects of novel interventions in clinical trials. An overarching goal of ALLFTD is to generate a well-characterized population of potential clinical trial participants who have data relevant to clinical trial inclusion and stratification criteria, for which fluid biomarker data from blood or CSF are useful. Moreover, as increasing numbers of clinical trials for FTLD are imminent, there is an urgent need to refine current fluid biomarkers and develop new tools with improved sensitivity and specificity to elucidate downstream effects of interventions. In addition to generating fluid biomarker data, a major role of the Biofluid Core will be to manage biospecimen and biomarker data sharing as well as to help manage collaborative biomarker discovery efforts with academia and industry. The Biofluid Core will be led by experts in FTLD biomarkers, Drs. Leonard Petrucelli and Adam Boxer, and an expert in CSF neurodegenerative biomarker discovery and analysis, Dr. Anne Fagan, to achieve the following aims: (1) Oversee biofluid specimen collection, processing, storage and sharing in collaboration with the Clinical Core, Administrative Core and the National Centralized Repository for Alzheimer's Disease and Related Dementias (NCRAD) biobank. (2) Perform measurements of standard biomarkers of neurodegeneration in CSF and blood in all participants, and targeted measurements of genetically relevant proteins in specific f-FTLD populations. (3) Support external collaborations to identify new FTLD therapeutic targets and biomarkers.

摘要 - Artfl Lefftds纵向FTLD：生物流体核心成功治疗额颞叶变性（FTLD）将需要尽早，准确的诊断，以及监测新干预措施在临床试验中的影响的能力。 Allftd的总体目标是产生具有与临床有关的数据的潜在临床试验参与者的特征良好的人群试验纳入和分层标准，对于血液或CSF的流体生物标志物数据是有用的。而且，由于即将进行的FTLD临床试验数量的越来越多，因此迫切需要优化当前的流体生物标志物并开发具有提高灵敏度和特异性的新工具，以阐明下游影响干预措施。除了生成流体生物标志物数据外，生物流体核心的主要作用将是管理生物循环和生物标志物数据共享以及帮助管理协作生物标志物发现工作与学术界和工业。生物流体核心将由FTLD生物标志物的专家领导。伦纳德 Petrucelli和Adam Boxer，以及CSF神经退行性生物标志物发现与分析的专家，博士安妮·法根（Anne Fagan 与临床核心，行政核心和国家集中式存储库共享阿尔茨海默氏病和相关痴呆症（NCRAD）生物库。（2）进行标准测量 CSF和所有参与者中神经退行性的生物标志物，并针对测量特定F-FTLD种群中与遗传相关的蛋白质。（3）支持外部协作以确定新的 FTLD治疗靶标和生物标志物。