Article
Discovery of novel VX-809 hybrid derivatives as F508del-CFTR correctors by molecular modeling, chemical synthesis and biological assays.
European journal of medicinal chemistry - 15 Dec 2020
Parodi Alice, Righetti Giada, Pesce Emanuela, Salis Annalisa, Tasso Bruno, Urbinati Chiara, Tomati Valeria, Damonte Gianluca, Rusnati Marco, Pedemonte Nicoletta, Cichero Elena, Millo Enrico
Abstract excerpt
Cystic fibrosis (CF) is the autosomal recessive disorder most recurrent in Caucasian populations. It is caused by different mutations in the cystic fibrosis transmembrane regulator protein (CFTR) gene, with F508del being the most common. During the last years, small-molecule therapy chosen to contrast CF relied on compounds that correct CFTR misfolding and ER retention (correctors such as VX-809), or defective...
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