Translational Research Center for Expedite Novel Therapies in Cystic Fibrosis

总体描述(由申请人提供)：这个P30囊性纤维化(CF)研究和翻译核心中心(CFRTC)将设在华盛顿大学医学院(UWSOM)及其附属机构西雅图儿童研究所(SCRI)。这项拟议的P30中心计划将增强这些机构现有强大的基础和临床研究基础，总共超过2000万美元的外部NIH和囊性纤维化基金会资金的直接成本(1500万美元基于临床，600万美元基于实验室)，并包括7个部门和/或部门的31名UWSOM研究人员。UWSOM在与CF相关的转化研究方面有着悠久的卓越历史，成功开发吸入型妥布霉素(TOBI(R))和在CFF支持的治疗开发网络中处于领先地位就是明证。P30的研究将集中在四个重点领域：1)提供资源和专业知识，以加速有前景的新治疗方法，以纠正缺失或功能失调的CFTR及其次要后果；2)促进现有和不断扩大的人类和细菌生物库的共享，以促进新生物标记物的开发和 实验室检测，3)加强对慢性肺疾病细菌发病机制的了解，以及4)加强对宿主炎症反应及其在慢性肺疾病中的继发性后果的了解。该中心将由共同派的邦尼·拉姆齐博士和E·彼得·格林伯格博士领导，他们都是在各自的临床研究和细菌发病机制领域得到国家认可的CF研究人员。他们将得到其他核心领导者的支持：威廉·帕克斯博士，上皮黏膜免疫调节方面的高级研究员，以及塞缪尔·米勒博士，细菌基因组学和蛋白质组学方面的高级科学家。该中心将由一个行政核心(Ramsey和Greenberg)和四个生物医学核心组成：微生物学(Greenberg，主任)、基因组学(Miller，主任)、炎症(Parks，主任)和临床翻译(Ramsey，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临床调查员Ronald Gibson领导。 目前的方案中包括三个P&F项目，这些项目是通过竞争程序从 九份申请。在资助期间，核心中心将专注于几部小说的开发 改善CF患者整体健康的治疗方法。

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.