The Synthesis of Molecular Probes Based on Cystic Fibrosis Drugs for Biological Research
Philip Junor
Abstract:<p>Cystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. These mutations either impair trafficking to thecell membrane, reducefunction as ananion channel,orboth. Ivacaftor(a channel potentiator) was the first FDA-approved drug that treats the underlying cause of CF, specifically for patients with the G551D mutation. Shortly after, Orkambi, a combination of ivacaftor and the trafficking corrector l… Show more
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