Translational Research Center for Expedite Novel Therapies in Cystic Fibrosis

概述（由申请人提供）：该P30囊性纤维化（CF）研究和转化核心中心（CFRTC）将位于华盛顿大学医学院（UWSOM）及其附属机构西雅图儿童研究所（SCRI）。这个拟议的P30中心项目将加强这些机构现有的强大的基础和临床研究基础，NIH和囊性纤维化基金会的外部直接资金总额超过2000万美元（1500万美元临床资金，600万美元实验室资金），包括31名UWSOM研究人员在7个部门和/或部门。UWSOM在CF相关的转化研究方面有着悠久的卓越历史，成功开发了吸入妥布霉素（TOBI(r)），并在CFF支持的治疗学开发网络中处于领导地位。P30的研究将集中在四个重点领域：1)提供资源和专业知识，以加快有希望的新治疗方法，以纠正CFTR缺失或功能失调及其继发后果；2)促进现有和正在扩展的人类和细菌生物库的共享，以加强新的生物标志物和生物标志物的开发

DESCRIPTION, OVERALL (provided by applicant): This P30 Cystic Fibrosis (CF) Research and Translational Core Center (CFRTC) will be located at the University of Washington School of Medicine (UWSOM) and its affiliated institution, Seattle Children's Research Institute (SCRI). This proposed P30 center program will enhance an existing robust basic and clinical research base at these institutions totally over $20 million direct costs in external NIH and Cystic Fibrosis Foundation funding ($15 million clinical based, $6 million laboratory based) and including 31 UWSOM investigators in 7 departments and/or divisions. The UWSOM has a long history of excellence in CF related translational research as evidenced by successful development of inhaled tobramycin (TOBI(r)) and leadership in the CFF supported Therapeutics Development Network. The research for this P30 will focus on four emphasis areas: 1) provide resources and expertise to expedite promising new therapeutic approaches to correct absent or dysfunctional CFTR and its secondary consequences, 2) foster sharing of existing and expanding human and bacterial biorepositories to enhance development of new biomarkers and laboratory assays, 3) enhance understanding of bacterial pathogenesis in CF lung disease, and 4) enhance understanding of host inflammatory response and its secondary consequences in CF. The center will be led by co-PI's, Dr. Bonnie Ramsey and Dr. E. Peter Greenberg, CF investigators both nationally recognized in their respective areas of clinical research and bacterial pathogenesis. They will be supported by other core leaders: Dr. William Parks, a senior investigator in modulation of immunity of epithelial mucosa and Dr. Samuel Miller, a senior scientist in bacterial genomics and proteomics. The center will consist of an Administrative Core (Ramsey and Greenberg) and four biomedical cores, Microbiology (Greenberg, Director), Genomics (Miller, Director), Inflammation (Parks, Director) and Clinical Translational (Ramsey, Director). The core centers will operate a Pilot and Feasibility (P&F) program led by Drs. Colin Manoil, a bacterial geneticist, and Ronald Gibson, a CF clinical investigator. Three P&F projects are included in the current proposal chosen through a competitive process from nine applications. During the funding period, the core center will focus on development of several novel therapeutic approaches to improve overall health for patients with CF.