UAB CF Research and Translation Core Center

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PROJECT SUMMARY/ABSTRACT The UAB P30 Research and Translation Core Center consolidates a large number of externally funded cystic fibrosis (CF) research programs on our campus, building an innovative environment to pursue, advance, and train in CF-related science. During the last funding period, the P30 made robust and important contributions to multiple UAB laboratories of our Research Base of over 70 investigators pursuing research relevant to CFTR modulation; CF pathogenesis; novel outcome measures for the genetic therapy era; infection and inflammation in the context of highly effective modulator therapy; and therapeutic translation for untreated CFTR mutations. By virtue of the NIH Center, translational research at our Institution has continued to accelerate in the past five years, as has the breadth of investigators. The richness of CF basic science at UAB has grown in parallel with this translational expansion, providing numerous opportunities to exploit in the next funding period, and the Center is strategically structured to take advantage of these opportunities. The P30 Center has allowed investigators at UAB and collaborating sites to improve understanding of CF disease mechanism and has furnished novel opportunities to aggressively apply this information towards experimental therapeutics, including genetic-based therapies that will continue to grow in emphasis. This NIH Center includes three Biomedical Research Cores that help to organize efforts of CF faculty towards the common and essential goal of helping individuals with CF and to train the next generation of CF leaders. The Cores include: Core A: Cell Model and Assay Core (GM Solomon and J Guimbellot, Co-PIs); Core B: Animal Models Core (DM Bedwell and S Birket, Co-PIs); and Core C: Clinical and Translational Core (SM Rowe and A Gaggar, Co-PIs). Each Core provides leading-edge assays, specialized reagents and techniques, and valued expertise. The P30 has also engaged new investigators through a Pilot and Feasibility mechanism integral to Center vitality and has a tight integration with the UAB Center for Clinical and Translational Science, enabling a robust training environment. In addition to providing a platform from which junior and senior scientists are brought into the field, Pilot Projects serve as a means of rapidly testing exciting advances, particularly from the perspective of clinical translation. Two Pilot and Feasibility Projects are in progress: Project 1: R Patel, PI. “Erythrocyte Hemolysis and Lung Disease in CF”; and Project 2: E Plaisance, PI. “Ketone Monoester Supplementation in CF: A Pilot and Feasibility Study”, capitalizing on the next era of innovative opportunities to transform the disease. Through these scientific initiatives, the P30 has added value to a collaborative environment for CF research at our Institution, and is well-positioned to continue in this capacity in the future, with a major focus on therapeutic translation.