Cystic Fibrosis Translational Research in the Post-CFTR Modulator Era

P30囊性纤维化(CF)研究和翻译核心中心(CFRTC)位于 华盛顿大学医学院及其附属机构西雅图儿童医院 研究院(SCRI)。建议的P30中心计划增强了现有的强大的基础和临床 这些机构的研究基础在当前研究资金中的直接成本总计超过3700万美元(年为2370万美元 与发展基金有关的资金占资金总额的%)，以及额外的2,900万美元与林业相关的基础设施 提供资金，包括在4个部门和/或部门的19个部门和/或司的58名大学和妇女研究所及其附属调查员 学校(医学51所，公共卫生3所，药房2所，牙科1所)。UWSOM有着悠久的历史 精通CF相关的翻译研究和微生物学，尤其关注胃肠道 微生物组以及过去5年与慢性肾衰相关的糖尿病(CFRD)。这个P30的研究将集中在 四个具体目标：1)加强NIDDK的研究核心和其他基础设施 感兴趣的领域，促进创造性、跨学科和临床影响的CF研究。2)提供 为世界各地的研究人员提供专业知识，以设计创新的临床研究，以确定最佳实践和 开发新的治疗方法来纠正功能失调的CFTR及其次要后果。3) 指导成功的试点项目和可行性资助项目，吸引年轻人和“新手” 研究人员，强调支持研究与CF相关的糖尿病、胃肠道疾病、肾脏疾病的研究人员 生理学和其他肺外疾病的表现。4)培养和指导下一代 实验室、临床和生物统计科学在本地和全美的领先地位。 该中心将由儿科学教授邦妮·拉姆齐医学博士和普拉迪普·辛格共同领导 医学博士，微生物学和医学教授，CF调查人员，都是国际公认的 各领域的临床研究和细菌发病机制。他们将得到其他关键因素的支持 领导：卢卡斯·霍夫曼医学博士，儿科学和微生物学教授，DVM，SCD， 比较医学系教授，医学博士，公共卫生硕士，医学教授和 儿科学，Stephen Salipante医学博士，Colin Manoil博士，实验室医学副教授， 基因组科学教授和儿科学副教授大卫·尼科尔斯。中心将由以下人员组成 一个行政核心(拉姆齐和辛格，董事)和三个生物医学核心，宿主-微生物(霍夫曼 和Frevert，董事)，基因组学(Salipante和Manoil，董事)和临床翻译(Goss和 拉姆齐，董事)。CFRTC将运行一个试点和可行性(P&F)计划，由辛格和 尼科尔斯。三个新批准的P&F项目包括在当前的提案中，通过竞争性的 经内部咨询委员会核准。

This P30 Cystic Fibrosis (CF) Research and Translational Core Center (CFRTC) is 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 enhances an existing robust basic and clinical research base at these institutions totaling over $37 million direct costs in current research funding ($23.7 in DDK related funding representing 64% of total) and an additional $29 million in CF related infrastructure funding and includes 58 UW/SCRI and affiliate investigators in 19 departments and/or divisions across 4 Schools (Medicine: 51, Public Health: 3, Pharmacy: 2, Dentistry: 1). The UWSOM has a long history of excellence in CF-related translational research and microbiology with particular focus on gastrointestinal microbiome as well as CF related Diabetes (CFRD) in the past 5 years. The research for this P30 will focus on four Specific Aims: 1) Enhance the research cores and other infrastructure for CF research in NIDDK interest areas that promotes creative, interdisciplinary and clinically-impactful CF research. 2) Provide expertise to researchers world-wide to design innovative clinical studies that determine best practice and develop new therapeutic approaches to correct dysfunctional CFTR and its secondary consequences. 3) Direct a successful pilot and feasibility grant program that attracts young and “new to CF” investigators, emphasizing support for investigators studying CF-related diabetes, GI disease, renal physiology, and other extra-pulmonary disease manifestations. 4) Train and mentor the next generation of laboratory, clinical, and biostatistical science leaders in CF locally and across the US. The center will be led by Co-PDs, Bonnie Ramsey, MD, Professor of Pediatrics and Pradeep Singh MD, Professor of Microbiology and Medicine, CF investigators both internationally recognized in their respective areas of clinical research and bacterial pathogenesis. They will be supported by other key leadership: Lucas Hoffman MD, PhD, Professor of Pediatrics and Microbiology, Charles Frevert, DVM, ScD, Professor of Department of Comparative Medicine, Christopher Goss MD, MPH, Professor of Medicine and Pediatrics, Stephen Salipante MD, PhD, Associate Professor of Laboratory Medicine, Colin Manoil PhD, Professor of Genome Sciences and David Nichols, Associate Professor of Pediatrics. The center will consist of an Administrative Core (Ramsey and Singh, Directors) and three biomedical cores, Host-Microbe (Hoffman and Frevert, Directors), Genomics (Salipante and Manoil, Directors) and Clinical Translational (Goss and Ramsey, Directors). The CFRTC will operate a Pilot and Feasibility (P&F) program led by Drs. Singh and Nichols. Three newly approved P&F projects are included in the current proposal chosen through a competitive process and approved by the Internal Advisory Board.