Translational Studies

点击翻译按钮获取中文摘要

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