In Silico Evaluation of Lumacaftor for Cystic Fibrosis

Cystic fibrosis is a common life-limiting autosomal recessive genetic disorder that causes severe damage to lungs, digestive system and other organs in the body. This disease is a result of mutation in a gene that encodes for chloride-conducting transmembrane channel called CFTR, which in turn regulates mucociliary clearance and anion transport in the airways. No effective curative options exist for treatment of Cystic fibrosis. The available drugs exhibit low therapeutic index and can only ease symptoms and reduce complications associated with it. This study aims to evaluate the effectiveness of one such FDA approved drug called Lumacaftor by molecular docking and other in silico studies. The most prevalent CFTR mutation causing cystic fibrosis, ΔF508, impairs folding of nucleotide binding domain 1 and stability of the interface between NBD1. Lumacaftor and drugs like Lumacaftor are modulators 0f F508del-CFTR are responsible for the partial correction of interfacial stability and cellular processing defects. Classification code: I