Patient personalized translational tools in cystic fibrosis to transform data from bench to bed-side and back

Abstract

In this study, we report an example of laboratory models informing clinical care for rare CF mutation patient, with subsequent recapitulation of clinical benefit in a unique and disease relevant, human-derived in vivo model, effectively translating data from the lab to the clinic and back. This extensive work outlines a potential platform to identify patient specific therapies and to understand relevant developmental abnormalities associated with CF disease.