cftr

CFTR corrector 12 is a CFTR corrector that rescues all mutant proteins except M760R ABCA3, and can be used to study cystic fibrosis.

CFTR corrector 4 is a potent and orally available transmembrane conductance regulator (CFTR) for cystic fibrosis and is a potent (R,R) type active enantiomer. CFTR corrector 4 increases CFTR levels on the cell surface and is a potential compound for the study of cystic fibrosis.

CFTR corrector 8 is a highly effective modulator of the cystic fibrosis transmembrane conductance regulator (CFTR). This compound is specifically designed for utilization in research related to cystic fibrosis, a genetic disorder primarily affecting the lungs and digestive system [1].

CFTR(inh)-172 (CFTR Inhibitor-172) is a voltage-independent, selective CFTR inhibitor.

CFTR corrector 2 (FDL169) is a novel and potent CFTR (Cystic fibrosis transmembrane conductance regulator) corrector for treating cystic fibrosis (CF) patients who carry the F508del mutation.

CFTR Corrector 9, a cystic fibrosis transmembrane conductance regulator (CFTR) modulator, is employed in the research of cystic fibrosis (CF) and related CFTR-associated disorders [1].

CFTR corrector 6 is a highly effective CFTR potentiator utilized in research on cystic fibrosis (CF) and related CFTR-associated disorders[1]. This potent compound exhibits potentiation activity on the Cystic Fibrosis Transmembrane conductance Regulator (CFTR), rendering it beneficial for investigations in CF and other associated conditions.

PPQ-102 (CFTR Inhibitor), an effective CFTR inhibitor, can completely inhibit CFTR chloride current (IC50: 90 nM).

Elexacaftor (VX-445) is a modulator of cystic fibrosis transmembrane conductance regulator (CFTR) and it also facilitates the processing and trafficking of CFTR to increase the amount of CFTR at the cell surface.

Glibenclamide (Glyburide) is an antidiabetic sulfonylurea derivative with actions similar to those of chlorpropamide.

Lumacaftor (VRT 826809) 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).

Ataluren (PTC124) 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.

Ivacaftor (VX-770) (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.

Corrector C4, a corrector commonly used to study cystic fibrosis mutants, works by alleviating the interaction between CFTR transmembrane domain mutants and protein homeostasis.

WAY-326766 increases ion transport through mutant CFTR and can be used to alter the lifespan of eukaryotes.WAY-326766 can be used to treat cystic fibrosis (CF).

VRT-532 (CFpot-532) is an effective modulator of CFTR and is commonly used in studies of cystic fibrosis (CF) caused by CFTR defects. Modification of CFTR by this small molecule modulator can increase arylsulfatase B (ARSB), which is necessary to reduce the accumulation of sulphate glycosaminosaccharide (gag), thus reducing the accumulation of 4-chondroitin sulfate in cystic fibrosis.

Bamocaftor is a CFTR channel (DeltaF508-CFTR mutation) corrector for CF transmembrane conductance regulators designed to restore F508del-CFTR protein function. Combined use of tezacaftor and VX-561 for the treatment of cystic fibrosis patients with F508del/MF.

Cavosonstat (N91115) is an orally active S-nitrosoglutathione reductase (GSNOR) inhibitor that promotes cystic fibrosis transmembrane conductance regulator (CFTR) maturation and plasma membrane stabilization. As a CFTR stabilizer, Cavosonstat can be used to study cystic fibrosis.

BPO-27 racemate (BPO-27 (racemate)) is an effective CFTR inhibitor with IC50 of 8 nM.

GlyH-101 is a cell-permeable glycinyl hydrazone compound that blocks CFTR with Ki of 1.4 uM.

Ouabain octahydrate (Acocantherine) is a cardiac glycoside that inhibits Na(+)/K(+) ATPase. Ouabain octahydrate regulates transcription of MDR (increase, Pgp) and MRP (increase MRP1 and decrease CFTR, cyctic fibrosis transport receptor or cAMP-activated Cl- channel) genes, also alters localization of MRP1.

Tezacaftor (VX661) is a small molecule that can be used as a corrector of the cystic fibrosis transmembrane conductance regulator (CFTR) gene function.

GLPG1837 (ABBV-974) is an effective CFTR potentiator, with EC50s of 3 nM and 339 nM for F508del and G551D CFTR, respectively.

Nesolicaftor (PTI-428) specifically enhances cystic fibrosis transmembrane conductance regulator protein synthesis.

Icenticaftor (QBW251) is an orally active potentiator of the CFTR channel.

IOWH-032 (IOWH032) , a synthetic CFTR inhibitor, has been investigated for the treatment of cholera, diarrhea, and secretory diarrhea.

3,4-Dephostatin is an inhibitor of the interactions of CFTR-associated ligand (CAL) and PSD-95/Dlg1/ZO-1 (PDZ) domain.

Corrector C18 is a class I mutation corrector which rescues CFTR mutations in nucleotide-binding domain 1 (NBD1) by modulating proteostasis.

Tezacaftor-d4 is a deuterated compound of Tezacaftor. Tezacaftor has a CAS number of 1152311-62-0. VX-661 is a small molecule that can be used as a corrector of the cystic fibrosis transmembrane conductance regulator (CFTR) gene function.

Ivacaftor hydrate is an orally bioavailable CFTR potentiator. It also is used for cystic fibrosis treatment.

(R)-Elexacaftor, an enantiomer of Elexacaftor, functions as a modulator of the cystic fibrosis transmembrane conductance regulator (CFTR). It exhibits an EC50 of 0.29 µM for the CFTR dF508 mutation [1].

(Rac)-Tezacaftor ((Rac)-VX-661) is a racemic mixture of the F508del CFTR corrector, Tezacaftor, used in cystic fibrosis research [1].

CTP-656, a cystic fibrosis transmembrane conductance regulator (CFTR) channel activator, is used potentially for the treatment of cystic fibrosis.

Navocaftor, as a cystic fibrosis transmembrane regulator ( CFTR ), is a protein modulator (US 20200377491 Al, example 1).

C4 Ceramide is a bioactive sphingolipid and cell-permeable analog of naturally occurring ceramides. [1] [2] [3] It inhibits IL-4 production by 16% in EL4 T cells stimulated with phorbol 12-myristate 13-acetate when used at a concentration of 10 μM. [1] C4 Ceramide is cytotoxic to SK-BR-3 and MCF-7/Adr breast cancer cells (IC50s = 15.9 and 19.9 μM, respectively). [2] C4 Ceramide also increases maturation and stability of cystic fibrosis transmembrane conductance regulator (CFTR) proteins bearing the F508 deletion (F508del) mutation, enhances cAMP-activated chloride secretion, and suppresses secretion of IL-8 in primary epithelial cells isolated from patients with cystic fibrosis.[3]

1. Steviol (NSC-226902), a natural sweetener, it inhibits proliferation of the gastrointestinal cancer cells intensively. 2. Steviol can induce a significant increase in CYP3A29 expression. 3. Steviol inhibits the proliferation of the human osteosarcoma U2OS cell line in a dose- and time-dependent manner. 4. Steviol can treat polycystic kidney disease, it slowed cyst growth, in part, by reducing AQP2 transcription, promoted proteasome, and lysosome-mediated AQP2 degradation.

Zatonacaftor, a cystic fibrosis transmembrane regulator (CFTR) modulator, is utilized in the research of cystic fibrosis [1] [2].

Very high affinity ClC-2 blocker (apparent KD ~ 50 pM). Slows ClC-2 activation and inhibits slow-gating but does not inhibit open ClC-2 channels. Selective for ClC-2 over other ClC family members (ClC-0, ClC-1, ClC-3 and ClC-4), CFTR, GABAC, CaCC and KV1.

Kobusin, a Calmodulin inhibitor, shows mild antiplasmodial,anti-inflammatory, and cytotoxic activities; it may be beneficial for the treatment of neuro-inflammatory diseases through the inhibition of iNOS expression and peroxynitrite scavenging potential. Kobusin can mildly reduce gastrointestinal motility in mice, it activates CFTR and CaCCgie chloride channel activities in mouse colonic epithelia and shows inhibitory effects toward ANO1/CaCC-mediated short-circuit currents in ANO1/CaCC-expressing FRT cells.

SRI-41315, a chemical compound, effectively addresses premature termination codons (PTCs) associated with cystic fibrosis in immortalized and primary human bronchial epithelial cells. It induces a prolonged pause at stop codons, resulting in the restoration of cystic fibrosis transmembrane conductance regulator (CFTR) expression and function. This compound achieves PTC suppression by reducing the abundance of the termination factor eRF1. Additionally, SRI-41315 synergistically enhances CFTR activity by potentiating aminoglycoside-mediated readthrough [1].

(R)-Vanzacaftor ((R)-VX-121) is a cystic fibrosis transmembrane conductance regulator (CFTR) modulator [1].

RNF5 inhibitor inh-02, a potent E3 ubiquitin ligase RNF5/RMA1 inhibitor, demonstrates significant efficacy in rescuing F508del-CFTR with an EC50 of 2.2 uM in bronchial epithelial cells homozygous for the F508del mutation. This compound is valuable for cystic fibrosis research[1].

(R)-Olacaftor ((R)-VX-440), a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator, demonstrates promising potential in cystic fibrosis (CF) research.

(R)-Posenacaftor ((R)-PTI-801) sodium, the R enantiomer of Posenacaftor, is a cystic fibrosis transmembrane regulator (CFTR) protein modulator. It corrects the folding and trafficking of the CFTR protein, and is utilized in the research of cystic fibrosis (CF)[1].

Dirocaftor (PTI-808) is a CFTR potentiator that enhances CFTR protein function by activating chloride channels, with potential utility for cystic fibrosis (CF) research.

NJH-2-057 is an EN523 OTUB1 recruiter linked to lumacaftor, a agent used to treat cystic fibrosis that binds ΔF508-CFTR .

NJH-2-056 is a deubiquitinase-targeting chimera (DUBTAC) linking the OTUB1 recruiter EN523 to the CFTR chaperone lumacaftor. NJH-2-056 can be used for cystic fibrosis research [1] .

ABBV-2222 is a CFTR corrector which is studied for the treatment of cystic fibrosis (CF).

DNDS is a channel blocker of voltage-dependent cystic fibrosis transmembrane conductance regulator (CFTR).

CP-628006 is a small molecule CFTR potentiator that effectively restores ATP-dependent channel gating to G551D-CFTR, the mutant form found in cystic fibrosis.