Translational Studies

项目主任/首席研究员(最后、第一、中间)：Frizzell，Ray翻译研究核心 摘要 P30翻译研究核心将继续在促进临床研究方面发挥重要作用 中心调查人员的活动以及培养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，以进行调查 发病机制和评价新的药物或基因疗法治疗囊性纤维化通过提供 访问500多名患者，随后是安东尼奥·J·帕伦博和珍妮特·帕伦博地区CF中心。核心的 任务是支持和维护一个分析实验室，该实验室支持最先进的测量 炎性介质，执行生理结果测量并提供统计支持，以及 管理样例存储库。具体地说，核心将： 1.执行专门的分析分析，并提供动物模型方面的专业知识，以促进翻译CF 研究。专门的检测包括细胞因子和趋化因子的多重检测，以及c)定量聚合酶链式反应。 临床标本的RNAseq分析。 2.提供基础设施，作为临床试验的终点执行生理测量，并执行 新疗法的早期研究。翻译核心将继续促进)体内呼吸道 吸收/粘液纤毛清除扫描；b)鼻电位差(NPD)测量；c)肺 清除指数(LCI)测量。 3.提供生物统计方面的基础设施，以支持研究人员发起的新研究。 4.维护和扩大参与的慢性萎缩性胃炎患者的血清、痰和上皮细胞样本库 研究人员发起的研究和来自纵向生物标志物的研究。痰，血清， 将对支气管肺泡灌洗、呼吸道/鼻腔上皮细胞进行处理和储存 临床信息。 除了四个具体目标外，中心还将继续支持中心调查员发起的研究 旨在加深我们对疾病发病机制的理解，并开发新的生物标志物或新的治疗方法 治疗慢性阻塞性肺疾病中的寿命缩短的肺部疾病。重要的是，这个核心的人员还将培训其他人员 并将帮助协调各项目之间的研究工作。 PHS 398/2590(11/07版)页面续格式页面

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