Nanomolar-potency ‘co-potentiator’ therapy for cystic fibrosis caused by a defined subset of minimal function CFTR mutants - Phuan, Puay-Wah; Tan, Joseph-Anthony; Rivera, Amber A.; Zlock, Lorna; Nielson, Dennis W.; Finkbeiner, Walter E.; Haggie, Peter M.; Verkman, Alan S. (Scientific reports, 2019)
Keywords: 631/154; 631/45/269/1147; Cell culture; Chloride conductance; Clonal deletion; Cystic fibrosis; Cystic Fibrosis - drug therapy; Cystic Fibrosis - genetics; Cystic Fibrosis - metabolism; Cystic Fibrosis Transmembrane Conductance Regulator - genetics; Cystic Fibrosis Transmembrane Conductance Regulator - metabolism; Drug Discovery; Drug Synergism; Epithelial cells; Gene deletion; High-Throughput Screening Assays; Humanities and Social Sciences; Humans; multidisciplinary; Mutation; Piperidine; Piperidines - therapeutic use; Pyrazoles - therapeutic use; Science; Science (multidisciplinary); Structure-Activity Relationship