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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 treat arthritis.
T1805 PTC124 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 patients aged 5 years and older who are able to walk.
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 CFTR(inh)-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 (VX-770) 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. The mechanism of action of Ivacaftor is as a Chloride Channel Activation Potentiator, and Cytochrome P450 3A4 Inhibitor, and Cytochrome P450 2C9 I
T2595 VX-809 VX-809 (Lumacaftor) acts to correct CFTR mutations common in cystic fibrosis by increasing mutant CFTR (F508del-CFTR) maturation (EC50: 0.1 μM) in Fisher rat thyroid cells.
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.
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