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：
10237338
负责人：
Bonnie W Ramsey
金额：
$ 121.48万
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/10237338
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.

该 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) 及其附属机构西雅图儿童医院研究所 (SCRI)。拟议的 P30 中心计划增强了现有的强大基础和临床能力。这些机构的研究基地目前的研究经费直接费用总计超过 3700 万美元（23.7 美元） DDK 相关资金占总额的 64%）以及 CF 相关基础设施的额外 2900 万美元资助，包括 58 名 UW/SCRI 和 4 个部门 19 个部门和/或部门的附属研究人员学校（医学：51 所，公共卫生：3 所，药学：2 所，牙科：1 所）。在 CF 相关转化研究和微生物学方面表现出色，特别关注胃肠道过去 5 年的微生物组以及 CF 相关糖尿病 (CFRD) 的研究将重点关注。四个具体目标： 1) 加强 NIDDK 的 CF 研究的研究核心和其他基础设施 2) 提供促进创造性、跨学科和具有临床影响力的CF研究的兴趣领域。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 及其继发后果 3)。指导成功的试点和可行性资助计划，吸引年轻人和“CF 新手” 研究人员，强调支持研究 CF 相关糖尿病、胃肠道疾病、肾病的研究人员生理学和其他肺外疾病表现 4) 培训和指导下一代。当地和美国各地 CF 实验室、临床和生物统计科学领域的领导者。该中心将由联合主任、儿科教授 Bonnie Ramsey 医学博士和 Pradeep Singh 领导医学博士、微生物学和医学教授、CF 研究人员均在其研究领域获得国际认可他们将得到其他关键领域的临床研究和细菌发病机制的支持。领导：Lucas Hoffman MD、PhD、儿科和微生物学教授 Charles Frevert、DVM、ScD、比较医学教授、克里斯托弗·戈斯医学博士、公共卫生硕士、医学教授儿科，Stephen Salipante 医学博士、博士、检验医学副教授、Colin Manoil 博士、该中心将由基因组科学教授和儿科副教授 David Nichols 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）和临床转化（Goss 和 Ramsey 董事）。CFRTC 将实施由 Singh 和 Dr. Ramsey 领导的试点和可行性 (P&F) 计划。 Nichols. 三个新批准的 P&F 项目包含在通过竞争选出的当前提案中。程序并由内部顾问委员会批准。