Translational Research Center for Expedite Novel Therapies in Cystic Fibrosis

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DESCRIPTION, OVERALL (provided by applicant): This P30 Cystic Fibrosis (CF) Research and Translational Core Center (CFRTC) will be 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 will enhance an existing robust basic and clinical research base at these institutions totally over $20 million direct costs in external NIH and Cystic Fibrosis Foundation funding ($15 million clinical based, $6 million laboratory based) and including 31 UWSOM investigators in 7 departments and/or divisions. The UWSOM has a long history of excellence in CF related translational research as evidenced by successful development of inhaled tobramycin (TOBI(r)) and leadership in the CFF supported Therapeutics Development Network. The research for this P30 will focus on four emphasis areas: 1) provide resources and expertise to expedite promising new therapeutic approaches to correct absent or dysfunctional CFTR and its secondary consequences, 2) foster sharing of existing and expanding human and bacterial biorepositories to enhance development of new biomarkers and laboratory assays, 3) enhance understanding of bacterial pathogenesis in CF lung disease, and 4) enhance understanding of host inflammatory response and its secondary consequences in CF. The center will be led by co-PI's, Dr. Bonnie Ramsey and Dr. E. Peter Greenberg, CF investigators both nationally recognized in their respective areas of clinical research and bacterial pathogenesis. They will be supported by other core leaders: Dr. William Parks, a senior investigator in modulation of immunity of epithelial mucosa and Dr. Samuel Miller, a senior scientist in bacterial genomics and proteomics. The center will consist of an Administrative Core (Ramsey and Greenberg) and four biomedical cores, Microbiology (Greenberg, Director), Genomics (Miller, Director), Inflammation (Parks, Director) and Clinical Translational (Ramsey, Director). The core centers will operate a Pilot and Feasibility (P&F) program led by Drs. Colin Manoil, a bacterial geneticist, and Ronald Gibson, a CF clinical investigator. Three P&F projects are included in the current proposal chosen through a competitive process from nine applications. During the funding period, the core center will focus on development of several novel therapeutic approaches to improve overall health for patients with CF.