Translational Research Center for Expedite Novel Therapies in Cystic Fibrosis

总体描述（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）。这项拟议的P30中心计划将加强这些机构现有的强大的基础和临床研究基础，外部NIH和囊性纤维化基金会资助的直接成本总计超过2000万美元（1500万美元临床基础，600万美元实验室基础），包括7个部门和/或部门的31名UWSOM研究人员。UWSOM在CF相关转化研究方面有着悠久的卓越历史，吸入妥布霉素（TOBI（r））的成功开发和CFF支持的治疗药物开发网络的领导地位证明了这一点。该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性后果，2）促进现有和扩大人类和细菌生物库的共享，以加强新生物标志物的开发， 实验室测定，3）增强对CF肺病中细菌发病机理的理解，和4）增强对CF中宿主炎症反应及其继发后果的理解。该中心将由共同PI的，博士邦妮拉姆齐和博士E。彼得格林伯格，CF研究人员都在各自的临床研究和细菌发病机制领域得到国家认可。他们将得到其他核心领导人的支持：上皮粘膜免疫调节高级研究员William Parks博士和细菌基因组学和蛋白质组学高级科学家Samuel米勒博士。该中心将包括一个行政核心（拉姆齐和格林伯格）和四个生物医学核心，微生物学（格林伯格，主任），基因组学（米勒，主任），炎症（公园，主任）和临床翻译（拉姆齐，主任）。核心中心将运行一个试点和可行性（P&F）计划，由细菌遗传学家Colin Manoil博士和CF临床研究员罗纳德吉布森博士领导。 三个P&F项目包括在当前的提案中，通过竞争过程从 九个应用在资助期间，核心中心将专注于开发几种新颖的 治疗方法，以改善CF患者的整体健康状况。

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.