Clinical Translation Core

摘要 反映临床疾病和囊性疾病不同方面的生物标志物的开发和验证 纤维化对于继续努力了解疾病生物学和疾病的发展至关重要。 新的治疗方法。在这里，我们建议创建一个新的临床翻译核心，它将极大地促进 这些努力。采集人体样本，包括痰、支气管镜标本、呼出气 凝结液、鼻上皮和直肠活检将由训练有素的人员专业收集，并与 规范临床数据。除了支持特定项目的需求外，还收集了常用的 标本(如痰和BAL)将以持续的方式出现，以建立一个标本库。《临床》 翻译核心还将推动包括粘液纤毛在内的新型成像生物标记物的发展 Clear和19F/1H-MRI胸部成像。这些模式将不仅为肺提供重要的见解 结构，以及关键生理功能的空间表示。最后，此核心将协助其他配置文件 RTCC调查员，通过在试验设计和实施方面提供专家指导和协助，主题 招聘、数据管理和分析。作为一个整体，临床翻译核心将支持一个大型 致力于了解CF病发病机制和开发新技术的研究人员数量 治疗方法的同时也为CF临床研究带来了新的、所需的终点。

ABSTRACT The development and validation of biomarkers that reflect different aspects of clinical disease and cystic fibrosis are of paramount importance to continued efforts to understand disease biology and the development of new therapeutics. Here, we propose the creation of a new Clinical Translation Core that will greatly facilitate these efforts. The collection of human samples including sputum, bronchoscopic specimens, exhaled breath condensate, nasal epithelia, and rectal biopsies will be expertly collected by trained personnel and linked to standardize clinical data. In addition to supporting needs of specific projects, collection of commonly used specimens (e.g. sputum and BAL) will occur in an ongoing fashion to build a specimen repository. The Clinical Translational Core will also advance the development of novel imaging biomarkers, including mucociliary clearance and 19F/1H-MRI thoracic imaging. These modalities will provide important insights into not only lung structure but also spatial representation of key physiologic functions. Finally, this Core will assist other CF RTCC investigators by providing expert guidance and assistance with trial design and conduct, subject recruitment, data management and analysis. As a whole, the clinical translation core will support a large number of investigators who are focused on understanding CF disease pathogenesis and developing new therapeutic approaches while also bringing new, needed endpoints to CF clinical research.