Article
Innovative Strategy toward Mutant CFTR Rescue in Cystic Fibrosis: Design and Synthesis of Thiadiazole Inhibitors of the E3 Ligase RNF5.
Journal of medicinal chemistry - 27 Jul 2023
Brusa Irene, Sondo Elvira, Pesce Emanuela, Tomati Valeria, Gioia Dario, Falchi Federico, Balboni Beatrice, Ortega Martínez Jose Antonio, Veronesi Marina, Romeo Elisa, Margaroli Natasha, Recanatini Maurizio, Girotto Stefania, Pedemonte Nicoletta, Roberti Marinella, Cavalli Andrea
Abstract excerpt
In cystic fibrosis (CF), deletion of phenylalanine 508 (F508del) in the CF transmembrane conductance regulator (CFTR) is associated to misfolding and defective gating of the mutant channel. One of the most promising CF drug targets is the ubiquitin ligase RNF5, which promotes F508del-CFTR degradation. Recently, the first ever reported inhibitor of RNF5 was discovered, i.e., the 1,2,4-thiadiazol-5-ylidene inh-2....
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