Cystic Fibrosis Translational Research in the Post-CFTR Modulator Era

后 CFTR 调节器时代的囊性纤维化转化研究

基本信息

批准号：
9982658
负责人：
Bonnie W Ramsey
金额：
$ 123.01万
依托单位：
SEATTLE CHILDREN'S HOSPITAL
依托单位国家：
美国
项目类别：
财政年份：
2010
资助国家：
美国
起止时间：
2010-07-06 至 2025-05-31
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/9982658
关键词：
Adult Affect Aging Area Basic Science Biological Biometry Child Chronic Clinical Clinical Research Collaborations Cystic Fibrosis Cystic Fibrosis Transmembrane Conductance Regulator Data Dentistry Development Direct Costs Disease Doctor of Philosophy Exocrine pancreas FDA approved Functional disorder Funding Future Genomics Genotype Germ-Free Goals Grant Growth Health Industry Inflammation Infrastructure Institution International Intestinal Diseases Islets of Langerhans Kidney Laboratories Leadership Link Lung Lung diseases Malignant neoplasm of gastrointestinal tract Measures Medicine Mentors Metabolic Methods Microbe Microbiology National Institute of Diabetes and Digestive and Kidney Diseases Nutritional Observational Study Outcome Measure Pathogenesis Patients Pediatrics Pharmaceutical Preparations Pharmacy facility Phenotype Physiological Physiology Population Process Public Health Recording of previous events Research Research Design Research Institute Research Personnel Resources Risk Science Scientist Specimen Statistical Methods Symptoms Training Translational Research Universities Vision Washington Work age group base care burden clinical effect clinical phenotype comparative cystic fibrosis mouse cystic fibrosis patients cystic fibrosis related diabetes design dissemination research drug development genome sciences gut microbiome improved infancy innovation insight interest kidney fibrosis medical schools member mid-career faculty next generation novel novel strategies novel therapeutic intervention novel therapeutics nutrition professor programs ranpirnase translational pipeline treatment effect

项目摘要

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.

P30囊性纤维化（CF）研究和转化核心中心（CFRTC）位于华盛顿大学医学院（UWSOM）及其分支机构机构西雅图儿童研究所（SCRI）。该建议的P30中心计划增强了现有的强大基本和临床这些机构的研究基础总计超过3700万美元的直接成本，目前的研究资金（23.7美元） DDK相关资金占总数的64％），另外2900万美元的CF相关基础设施资金，包括19个部门和/或4个部门的58个UW/SCRI和分支机构调查员学校（医学：51，公共卫生：3，药房：2，牙科：1）。 UWSOM有悠久的历史卓越与CF相关的转化研究和微生物学，特别关注胃肠道在过去的5年中，微生物组以及与CF相关的糖尿病（CFRD）。该P30的研究将重点放在四个具体目的：1）增强NIDDK CF研究的研究核心和其他基础设施促进创意，跨学科和临床影响的CF研究的兴趣领域。 2）提供全球研究人员的专业知识，以设计最佳实践和开发新的治疗方法，以纠正功能失调的CFTR及其次要后果。 3）指导一项成功的飞行员和可行性赠款计划，吸引了年轻和“新手CF” 调查人员强调支持研究CF相关糖尿病，胃肠道疾病，肾脏的研究人员生理学和其他肺外疾病表现。 4）火车和指导下一代 CF本地和整个美国的实验室，临床和生物统计学科学领导者。该中心将由Co-Pds，Bonnie Ramsey，医学博士，儿科教授和Pradeep Singh领导 MD，微生物学和医学教授，CF研究人员都在国际上认可其临床研究和细菌发病机理的各自领域。他们将得到其他钥匙的支持领导力：卢卡斯·霍夫曼（Lucas Hoffman）医学博士，博士，儿科和微生物学教授，查尔斯·弗雷弗特（Charles Frevert），DVM，SCD，比较医学系教授，克里斯托弗·戈斯（Christopher Goss）医学博士，MPH，医学教授和儿科，Stephen Salipante医学博士，博士，Colin Manoil博士实验室医学副教授，基因组科学教授和大卫·尼科尔斯（David Nichols），儿科副教授。中心将包括行政核心（Ramsey and Singh，董事）和三个生物医学核心，宿主 - 摩托车（Hoffman）和FREVERT，董事），基因组学（Salipante and Manoil，董事）和临床翻译（Goss和Goss和拉姆西，导演）。 CFRTC将运营由DRS领导的飞行员和可行性（P＆F）计划。辛格和尼科尔斯。通过竞争性选择的当前提案中包括三个新批准的P＆F项目过程并由内部顾问委员会批准。