Translational Studies

项目负责人/主要研究者（最后一名、第一名、中间名）：Frizzell，Ray Translational Studies Core 摘要 P30转化研究核心将继续在促进临床研究方面发挥重要作用 中心研究人员的活动，并促进与学术机构或行业的合作者。 此外，核心将为新的临床前研究和临床试验提供基础设施， 发病机制和评估新的药理学或遗传疗法，以治疗囊性纤维化， 访问超过500名患者，其次是区域安东尼奥J和珍妮特帕伦博CF中心。该芯体的 使命是支持和维护一个分析实验室，该实验室支持最先进的 炎症介质，以执行生理结果测量并提供统计支持，以及 管理示例存储库。具体而言，核心将： 1.进行专业的分析测定，并提供动物模型方面的专业知识，以促进转化CF research.专门的测定包括用于细胞因子和趋化因子的基于多重的测定，以及 和临床样品的RNAseq分析。 2.提供基础设施，以执行生理测量作为临床试验的终点，并执行 新疗法的早期研究。平移核心将继续促进a）体内气道 吸收/粘膜纤毛清除扫描; B）鼻电位差（NPD）测量，和c）肺 清除指数（LCI）测量。 3.提供生物统计学基础设施，以支持CF中新的生物统计学发起的研究。 4.维持和扩大来自参与CF治疗的CF患者的血清、痰液和上皮细胞的样本库， 由制药商发起的研究和纵向生物标志物研究。痰液，血清， 支气管肺泡灌洗液和气道/鼻上皮细胞将被处理并与伴随的 临床信息。 除了四个具体目标外，核心将继续支持中心发起的研究 旨在进一步了解疾病的发病机制，并开发新的生物标志物或新的治疗方法， 治疗缩短寿命的肺病重要的是，该核心的人员还将培训其他人员， 并将帮助协调项目之间的研究工作。 PHS 398/2590（Rev. 11/07）

Program Director/Principal Investigator (Last, First, Middle): Frizzell, Ray Translational Studies Core Abstract The P30 Translational Studies Core will continue to perform a vital role in facilitating the clinical research activities of the Center investigators and in fostering collaborators with academic institutions or industry. Moreover the Core will provide the infrastructure for new pre-clinical studies and clinical trials to investigate pathogenesis and evaluate new pharmacological or genetic therapies to treat Cystic Fibrosis by providing access to over 500 patients followed by the regional Antonio J. and Janet Palumbo CF Center. The Core's mission is to support and maintain an analytical lab, which supports state-o/f-the-art measurements of inflammatory mediators, to perform physiological outcome measures and provide statistical support, and to manage a sample repository. Specifically, the Core will: 1. Perform specialized analytical assays and provide expertise in animal models to facilitate translational CF research. Specialized assays include multi-plex based assays for cytokines and chemokines, and c) qPCR and RNAseq analysis of clinical samples. 2. Provide the infrastructure to perform physiological measures as endpoints in clinical trials, and to execute early Phase studies of novel therapies. The Translational Core will continue to facilitate a) in vivo airway absorptive/mucociliary clearance scans; b) nasal potential difference (NPD) measurements, and c) lung clearance index (LCI) measurements. 3. Provide infrastructure in biostatistics to support novel investigator-initiated research in CF. 4. Maintain and expand a sample bank of serum, sputum, and epithelial cells from CF patients participating in investigator-initiated research and from a longitudinal biomarkers study. Samples of sputum, serum, bronchoalveolar lavage, and airway/nasal epithelial cells will be processed and stored with accompanying clinical information. In addition to the four Specific Objectives, the Core will continue to support Center investigator-initiated studies that aim to further our understanding of disease pathogenesis, and develop new biomarkers or novel therapies for the life-shortening lung disease in CF. Importantly, the personnel in this Core will also train other personnel and will help coordinate research efforts among projects. PHS 398/2590 (Rev. 11/07) Page Continuation Format Page