Today’s post is dedicated to cystic fibrosis transmembrane conductance regulator (CFTR) which is an ion transporter that regulates mucus hydration, viscosity, and acidity of the airway epithelial surface.
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In cystic fibrosis, CFTR dysfunction causes morbidity or mortality. Significant progress has been made in developing CFTR modulator therapy, CFTR-rescuing drugs have already transformed cystic fibrosis (CF) from a fatal disease to a treatable chronic condition. However, new-generation drugs able to bind CFTR with higher specificity/affinity and exert stronger therapeutic benefits and fewer side effects are still awaited. Another important goal is to determine the impact of CFTR-dependent cell signaling on CF disease pathogenesis.
Recently computational methods and biosensors have become indispensable tools in the process of drug discovery for many important human pathologies. They have been used only piecemeal in CF so far, calling for their appropriate integration with well-tried CF biochemical and cell-based models to speed up the discovery of new CFTR-rescuing drugs.