Biofluid Core

摘要-ARTFL LEFFTDS纵向FTLD：生物流体核心 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 生成具有良好特征的潜在临床试验参与者群体，他们拥有与临床相关的数据 试验纳入和分层标准，血液或脑脊液中的流体生物标记物数据对其有用。此外， 随着越来越多的FTLD临床试验迫在眉睫，迫切需要提炼当前的液体 并开发新的工具，提高敏感性和特异性，以阐明下游的影响 干预措施。除了产生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 生物标记物和生物标记物数据共享以及帮助管理协作的生物标记物发现工作 与学术界和工业界合作。生物流体核心将由FTLD生物标志物专家Leonard博士领导 Petrucelli和Adam Boxer博士是发现和分析脑脊液神经退行性生物标记物的专家。 Anne Fagan，以实现以下目标：(1)监督生物流体样本的收集、处理、储存和 与临床核心、管理核心和国家中央资料库合作共享 阿尔茨海默病和相关痴呆(NCRAD)生物库。(2)执行标准测量 所有参与者脑脊液和血液中神经变性的生物标记物，并有针对性地测量 特定f-FTLD人群中的遗传相关蛋白质。(3)支持外部协作，发现新的 FTLD治疗靶点和生物标志物。

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.