UAB CF Research and Translation Core Center

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DESCRIPTION (provided by applicant): The UAB P30 Research and Translation Core Center consolidates a large number of externally funded cystic fibrosis (CF) research programs on our campus. During the first funding period, the P30 has made important contributions to multiple UAB laboratories pursuing research relevant to CFTR cellular and Structural biology and CF pathogenesis. By virtue of the NIH Center, translational research at our Institution has advanced significantly in the past four years The richness of CF basic science at UAB has grown in parallel with this translational expansion. The P30 Center has allowed investigators at UAB and collaborating sites to improve understanding of cystic fibrosis disease mechanism and furnished novel opportunities to aggressively apply this Information towards experimental therapeutics. This NIH Center includes three Scientific Cores that help organize efforts of CF faculty towards the common and essential goal of helping Individuals with CF. The Cores include: Core A: Cell Model and Assay Core (KL Kirk, PI); Core B: Animal Models Core (DM Bedwell, PI); and Core C: Clinical and Translational Core (SM Rowe, PI). Each Core provides leading-edge assays, specialized reagents and valved expertise. The P30 has also engaged new investigators through a Pilot and Feasibility mechanism integral to Center vitality. 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 proposed: Project 1: SG Aller, PI. "Structure-based correction of the major defect in cystic fibrosis"; and Project 2: GA Caldwell, PI. "Investigation of torsinA modulation as a therapeutic strategy for cystic fibrosis". Through these scientific initiatives, th P30 has enhanced a collaborative environment for cystic fibrosis research at UAB, and is well positioned to continue in this capacity in the future.