Project 1: Translational Science of Alopecia Areata

摘要 该研究项目是AACORT的主要研究指导方针。的总目标是 AACORT是对斑秃病理的综合研究，该研究 该项目正式确定为四个关键目标：1)微生物组的研究，2)蛋白质组研究 AA自身抗原，3)AA病理和药物发现的小鼠模型研究，4)和人类的研究 对再生障碍性贫血患者进行分子病理和药物反应。这四个目标将导致全面 微生物组、基因组、转录组和蛋白质组负荷的特征 AA病理学。这反过来将为生物标志物的开发提供基础，以进行跟踪 再生障碍性贫血的进展和治疗反应(基因组和分子)，以及鉴定 毒品目标和候选毒品。为了推动对这些目标的追求，研究项目 将作为核心预算资源调拨目标核心之间的关键调解人，负责设计和 获取用于分析的人类患者生物样本和数据核心，该数据核心将监督 对TRAC生成的所有数据进行注释、存储和分析。这将是研究之一 项目的作用是确定这两个核心的有针对性的生物目标，然后 获取数据并分析关键的分子驱动因素，然后研究项目就可以 验证作为他们正在进行的研究的补充。研究项目进行的实验，在 人类或小鼠模型，将不断与人类的生物信息学分析交叉核对 共同开发一种前所未有的再生障碍性贫血病理学系统模型。以这种方式，所有正在进行的 AACORT的研究将由该研究提出的中心生物学问题进行调解 项目，从而提供统一的目标，使AACORT可以利用其多个 分析管道。

ABSTRACT The Research Project serves as the principal research directive of the AACORT. The overall goal of the AACORT is the comprehensive study of the pathology of Alopecia Areata, which the Research Project formalizes into four key goals: 1) the study of the microbiome, 2) proteomic research into AA autoantigens, 3) mouse model studies of AA pathology and drug discovery, 4) and studies of human AA patients for molecular pathology and drug response. These four goals will result in the full characterization of the microbiome, genomic, transcriptomic, and proteomic loads that contribute to AA pathology. This, in turn, will provide the foundation for the development of biomarkers to track AA progression and response to treatment (both genomic and molecular), and the identification of drug targets and drug candidates. To drive the pursuit of these objectives, the Research Project will serve as a key mediator between the TRAC Core, which will be responsible for designing and acquiring human patient biosamples for analysis, and the DATA Core, which will oversee the annotation, storage, and analysis of all data generated by the TRAC. It will be one of the Research Project’s roles to identify targetable biological objectives to these two cores, which will then acquire the data and analyze it for the key molecular drivers that the Research Project can then validate as a complement to their ongoing research. Experiments pursued by the Research Project, in human or mouse models, will be continuously cross-checked with the bioinformatic analyses of human cohorts to develop an unprecedented systemic model of AA pathology. In this manner, all ongoing research in the AACORT will be mediated by the central biological questions posed by the Research Project, thereby providing uniform objectives for which the AACORT can leverage its multiple analytic pipelines.