Personalized Cystic Fibrosis Therapy and Research Center

项目概要： 该P30应用程序的一个基本组件是个性化临床核心，它将是 人类细胞、组织、生物流体的主要来源[例如：汗液、血清、痰液、支气管肺泡灌洗液 （BALF），]和翻译研究的专业知识。个性化临床核心直接建立在当前临床 CCHMC，CSMC和UCCOM的研究优势和基础设施，包括CFF-TDN计划， CF-RDP计划，赞助商发起的研究，和机构承诺CF转化研究。 个性化临床核心将以关键方式扩展这些功能， 转化研究项目，并提供与尖端基础科学直接相关的患者。很长的- 这一核心的长期目标是支持基础科学CF研究与患者来源的样本从临床到 实验室和高质量的转化研究将实验室发现转移到临床。这将是 通过三个具体目标实现。具体目标1和2将利用现有的伙伴关系， 分别获取、储存和分发高质量胃肠道和呼吸道样本的基础设施， 来自CF和非CF对照，与相关患者数据配对。具体目标3将提供工具 在CF中执行尖端转化研究所必需的，包括新型疾病的专业知识和支持 标记和终点。 通过个性化临床核心结构集中这些功能将有助于推动 研究效率和标准化。此外，通过 个性化的临床核心将可供CF研究人员直接使用，并用于利用 个性化模型系统核心和CF-器官芯片核心。

PROJECT SUMMARY: A foundational component of this P30 application is the Personalized Clinical Core, which will be the primary source of human cells, tissues, biofluids [e.g.: sweat, serum, sputum, bronchoalveolar lavage fluid (BALF),] and translational research expertise. The Personalized Clinical Core builds directly on current clinical research strengths and infrastructure at CCHMC, CSMC and UCCOM including the CFF-TDN program, the CF-RDP program, investigator-initiated research, and institutional commitments to CF translational research. The Personalized Clinical Core will expand these capabilities in critical ways, operationalizing novel translational research projects and providing direct patient relevance to cutting-edge basic science. The long- term goal of this core is to support basic science CF research with patient-derived samples from the clinic into the laboratory, and high-quality translational research moving laboratory findings into the clinic. This will be achieved through three specific aims. Specific Aims 1 and 2 will capitalize on existing partnerships and infrastructure to obtain, bank, and distribute high-quality gastrointestinal and respiratory samples, respectively, from CF and non-CF controls, paired with relevant patient data. Specific Aim 3 will provide the tools necessary to execute cutting-edge translational studies in CF, including expertise and support in novel disease markers and endpoints. Centralizing these capabilities through the Personalized Clinical Core structure will help to drive research efficiencies and standardization. In addition, the samples obtained and processed through the Personalized Clinical Core will be available for direct use by CF researchers, and for projects utilizing the Personalized Model System Core and the CF-Organ-On-a-Chip Core.