Biofluid Core

生物流体核心

• 批准号: 10228128
• 负责人: ADAM L. BOXER
• 金额: $ 2.83万
• 依托单位: MAYO CLINIC ROCHESTER
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-09-15 至 2024-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10228128
• 关键词: Academia; Address; Alzheimer' s disease related dementia; Biological Markers; Blood; Blood specimen; C9ORF72; Cataloging; Catalogs; 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

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：生物流体芯 额颞叶变性（FTLD）的成功治疗需要早期和准确的诊断， 以及在临床试验中监测新干预措施效果的能力。ALLFTD的首要目标是 生成具有临床相关数据的潜在临床试验参与者的良好特征人群 试验入选和分层标准，血液或CSF中的液体生物标志物数据对此有用。此外，委员会认为， 随着越来越多的FTLD临床试验迫在眉睫，迫切需要改进现有的流体 生物标志物，并开发具有改进的灵敏度和特异性的新工具，以阐明 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是管理 生物样本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 学术界和工业界的合作。生物流体核心将由FTLD生物标志物专家伦纳德博士领导 Petrucelli博士和Adam Boxer博士，以及CSF神经退行性生物标志物发现和分析方面的专家。 安妮·费根，以实现以下目标：（1）监督生物流体标本的收集，处理，储存和 与临床核心、行政核心和国家集中储存库合作共享， 阿尔茨海默病和相关痴呆症（NCRAD）生物库。(2)执行标准品测量 所有参与者的CSF和血液中的神经变性生物标志物，以及 特定f-FTLD人群中的遗传相关蛋白。(3)支持外部合作，以确定新的 FTLD治疗靶点和生物标志物。