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

• 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
• 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

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.

这个P30囊性纤维化（CF）研究和转化核心中心（CFRTC）位于 华盛顿大学医学院及其附属机构西雅图儿童医院 研究所（SCRI）。这一拟议的P30中心计划增强了现有的强大的基础和临床 在这些机构的研究基地，目前的研究经费中的直接费用总计超过3700万美元（2370万美元）。 DDK相关资金占总额的64%），另外2900万美元用于CF相关基础设施 资金，包括58个UW/SCRI和附属调查员在19个部门和/或部门跨越4 学校（医学：51，公共卫生：3，药学：2，牙科：1）。UWSOM历史悠久， 卓越的CF相关的转化研究和微生物学，特别关注胃肠道 微生物组以及CF相关糖尿病（CFRD）。P30的研究将集中在 四个具体目标：1）加强NIDDK CF研究的研究核心和其他基础设施 感兴趣的领域，促进创造性，跨学科和临床影响力的CF研究。2)提供 为全球研究人员提供专业知识，以设计创新的临床研究，确定最佳实践， 开发新的治疗方法来纠正功能失调的CFTR及其继发性后果。第三章 指导一个成功的试点和可行性资助计划，吸引年轻人和“新的CF” 研究者，强调支持研究CF相关糖尿病、GI疾病、肾功能衰竭、 生理学和其他肺外疾病表现。4)培训和指导下一代 实验室，临床和生物统计科学的领导者在CF本地和美国各地。 该中心将由共同PD，邦妮拉姆齐，医学博士，儿科教授和Pradeep辛格领导 医学博士，微生物学和医学教授，CF研究人员都在国际公认的， 临床研究和细菌致病机理的各自领域。他们将得到其他关键的支持 领导：Lucas霍夫曼医学博士，哲学博士，儿科和微生物学教授，Charles Frevert，DVM，ScD， 比较医学系教授，Christopher Goss医学博士，公共卫生硕士，医学教授， 儿科，Stephen Salipante医学博士，博士，实验室医学副教授，Colin Manoil博士， 基因组科学教授和儿科副教授大卫尼科尔斯。该中心将由 一个行政核心（Ramsey和Singh，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 Ramsey，Directors）. CFRTC将运行一个由辛格博士领导的试点和可行性（P&F）计划， 尼科尔斯.三个新批准的P&F项目包括在当前的建议书中， 并由内部咨询委员会批准。