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CFTR
T0429 Glucosamine Glucosamine is an amino sugar and a prominent precursor in the biochemical synthesis of glycosylated proteins and lipids. Supplemental glucosamine may help to rebuild cartilage and
T1805 Ataluren Ataluren is a novel, orally administered drug that targets nonsense mutations. Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in
T1874 PPQ-102 PPQ-102, an effective CFTR inhibitor, can completely inhibit CFTR chloride current (IC50: 90 nM).
T2263 VX661 VX-661 is a small molecule that can be used as a corrector of the cystic fibrosis transmembrane conductance regulator (CFTR) gene function.
T2355 CFTRinh-172 CFTRinh-172 is a voltage-independent, selective CFTR inhibitor.
T2451 GlyH-101 GlyH-101 is a cell-permeable glycinyl hydrazone compound that blocks CFTR with Ki of 1.4 uM.
T2486 IOWH-032 IOWH032, a synthetic CFTR inhibitor, has been investigated for the treatment of cholera, diarrhea, and secretory diarrhea.
T2588 Ivacaftor Ivacaftor (VX-770) is a potentiator of CFTR targeting G551D-CFTR (EC50: 100 nM) and F508del-CFTR (EC50: 25 nM) in Fisher rat thyroid cells, respectively.
T2595 Lumacaftor VX-809 is a CFTR modulator that corrects the folding and trafficking of CFTR protein. It enhances F508del-CFTR protein maturation in FRT cells (EC50: 100 nM).
T3135 KM11060 KM11060 is a novel corrector of the F508del-CFTR trafficking defect, Correcting F508del-CFTR trafficking, increasing the amount of functional CFTR at the plasma membrane.
T5312 FDL169 FDL-169 is a novel and potent CFTR (Cystic fibrosis transmembrane conductance regulator) corrector for treating cystic fibrosis (CF) patients who carry the F508del mutation.
T7083 GLPG1837 GLPG1837 is an effective CFTR potentiator, with EC50s of 3 nM and 339 nM for F508del and G551D CFTR, respectively.
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