Clinical Translation Core

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ABSTRACT The development and validation of biomarkers that reflect different aspects of clinical disease and cystic fibrosis are of paramount importance to continued efforts to understand disease biology and the development of new therapeutics. Here, we propose the creation of a new Clinical Translation Core that will greatly facilitate these efforts. The collection of human samples including sputum, bronchoscopic specimens, exhaled breath condensate, nasal epithelia, and rectal biopsies will be expertly collected by trained personnel and linked to standardize clinical data. In addition to supporting needs of specific projects, collection of commonly used specimens (e.g. sputum and BAL) will occur in an ongoing fashion to build a specimen repository. The Clinical Translational Core will also advance the development of novel imaging biomarkers, including mucociliary clearance and 19F/1H-MRI thoracic imaging. These modalities will provide important insights into not only lung structure but also spatial representation of key physiologic functions. Finally, this Core will assist other CF RTCC investigators by providing expert guidance and assistance with trial design and conduct, subject recruitment, data management and analysis. As a whole, the clinical translation core will support a large number of investigators who are focused on understanding CF disease pathogenesis and developing new therapeutic approaches while also bringing new, needed endpoints to CF clinical research.