Alopecia Areata Center for Research Translation (AACORT)

AACORT总体摘要 哥伦比亚大学医学中心的斑秃研究翻译中心（AAORT） （CNOC）专注于开发斑秃（AA）新疗法的单一目标。的使命 AACORT是利用我们最近的发现，开始解开AA的遗传基础和事实， 其发病机制与先天性和获得性免疫的失调有关， 转化研究的发现，将导致这种疾病的新疗法，包括重新利用 现有的药物。在过去的几年里，AACOT团队在理解 AA的遗传基础，以及启动一系列采用机制驱动的靶向JAK的临床试验 旨在逆转疾病的抑制剂。虽然这类药物对指导 尽管在AA的临床开发方面做出了努力，但还有更多的工作要做，因为很明显许多患者没有 对这些药物有反应。AACORT的目标是在我们已建立的翻译管道的基础上， 将我们的基础和临床前发现应用于临床。这个AACORT是围绕一个 中央研究项目，侧重于斑秃的转化科学，并处理 疾病的创新转化方面，包括启动因素，如微生物组;临床前 研究集中在抗原发现和测试新的治疗方法在体外和体内;和临床 相关研究旨在严格评估新药的有效性和安全性， AA患者该研究项目得到了翻译和临床资源核心（TRAC）的支持， 和数据分析和翻译应用（DATA）核心，汇集了一个优秀的干部， 来自整个机构的才华横溢，忠诚和科学多元化的教师。支持AACORT 由一个非常有能力和经验丰富的行政核心，提供其至关重要的组织 基础设施演进AACORT的目标是克服基础发现转化应用的障碍 有可能彻底改变这种迄今为止难以治疗和使人衰弱的疾病的管理。

Overall AACORT Abstract The Alopecia Areata Center of Research Translation (AACORT) at Columbia University Medical Center (CUMC) is focused on the singular goal of developing new treatments for alopecia areata (AA). The mission of the AACORT is to leverage our recent discoveries beginning to unravel the genetic basis of AA and the fact that its pathogenesis is linked to dysregulation of both innate and acquired immunity, and to synergize these findings into translational studies that will lead to novel therapies for this disease, including the repurposing of existing drugs. In the past few years, the AACORT team has made transformative progress in understanding the genetic basis of AA, as well as initiating a series of clinical trials employing mechanism-driven targeted JAK inhibitors aimed at reversing the disease. While this class of drugs is having a profound impact on guiding clinical development efforts in AA, there is much more work to be done, since clearly many patients do not respond to these drugs. The goal of this AACORT is to build upon our established translational pipeline to leverage our basic and preclinical discoveries into the clinical setting. This AACORT is centered around a central Research Project that focuses on the Translational Science of Alopecia Areata, and deals with innovative translational aspects of the disease, including initiating factors such as the microbiome; preclinical studies focused on antigen discovery and testing new therapeutic approaches in vitro and in vivo; and clinical correlative studies designed to rigorously assess the efficacy and safety of new drugs coming available for patients with AA. The Research Project is supported by the Translational and Clinical Resource Core (TRAC), and the Data Analysis and Translational Applications (DATA) Core, which bring together an outstanding cadre of talented, committed and scientifically diverse faculty from across the institution. The AACORT is supported by a highly capable and experienced Administrative Core that provides its vitally important organizational infrastructure. The goal of the AACORT is to surmount barriers to translational application of basic discoveries that have the potential to revolutionize the management of this heretofore intractable and debilitating disorder.