Center for Cystic Fibrosis Translational Research in the Post-CFTR modulator Era

Seattle Children'S Hospital, Seattle WA

Investigators

Linked publications & trials

Abstract

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 proposal updates our already robust P30 center program, with a large basic and clinical research base at these institutions extremely robust CF related infrastructure funding, comprising 19 departments and/or divisions across 5 Schools. The UWSOM has a long history of excellence in CF-related translational research and microbiology, with particular emphasis on the gastrointestinal microbiome, CF related diabetes (CFRD), and kidney disease 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 to promote creative, interdisciplinary and clinically-impactful CF research. 2) Provide expertise to researchers nationally to design innovative clinical studies that determine best practices 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 nationally. The center will be led by Multi-PDs Christopher H. Goss, MD MSc, Professor of Medicine and Pediatrics, and Lucas Hoffman, MD PhD, Professor of Pediatrics and Adjunct Professor of Microbiology, CF investigators both internationally recognized in their respective areas of clinical research and CF microbiology and gut microbiome. They will be supported by other key leadership: Kathleen Ramos MD, MSc, Associate Professor of Medicine; Charles Frevert, DVM, ScD, Professor of Department of Comparative Medicine; Pradeep Singh, MD, Professor of Medicine and Microbiology; Pavan Bhatraju, MD, MSc, Associate Professor of Medicine; Stephen Salipante MD, PhD, Professor of Laboratory Medicine; Sonya Heltshe, PhD, Professor of Pediatrics; and Sina Gharib, MD, Professor of Medicine. The center will consist of an Administrative Core (Goss and Hoffman, Directors) and three biomedical cores: The Clinical and Specimen (Goss and Ramos, Directors with Frevert and Hoffman), Genomics (Salipante, Director) and Biostatistical and Informatics Cores (Heltshe and Gharib, Directors). The CFRTC will operate a Pilot and Feasibility (P&F) program led by Drs. Singh and Bhatraju. Three newly approved P&F projects are included in the current proposal chosen through a competitive process and approved by the Internal Advisory Board.