Target Information

Target General Infomation
Target ID
T55654
Former ID
TTDNS00605
Target Name
cystic fibrosis transmembrane conductance regulator
Gene Name
CFTR
Synonyms
ATPbinding cassette subfamily C member 7; Channel conductancecontrolling ATPase; Cystic fibrosis transmembrane conductance regulator; cAMPdependent chloride channel; CFTR
Target Type
Successful
Disease Cystic fibrosis [ICD9: 277; ICD10: E84]
Diarrhea [ICD9: 787.91; ICD10: A09, K59.1]
HIV-associated diarrhoea [ICD10: A09]
Function
Involved in the transport of chloride ions. May regulate bicarbonate secretion and salvage in epithelial cells by regulating the SLC4A7 transporter. Can inhibit the chloride channel activity of ANO1. Plays a role in the chloride and bicarbonate homeostasis during sperm epididymal maturation and capacitation.
BioChemical Class
Acid anhydrides hydrolase
UniProt ID
P13569
EC Number
EC 3.6.3.49
Sequence
MQRSPLEKASVVSKLFFSWTRPILRKGYRQRLELSDIYQIPSVDSADNLSEKLEREWDRE
LASKKNPKLINALRRCFFWRFMFYGIFLYLGEVTKAVQPLLLGRIIASYDPDNKEERSIA
IYLGIGLCLLFIVRTLLLHPAIFGLHHIGMQMRIAMFSLIYKKTLKLSSRVLDKISIGQL
VSLLSNNLNKFDEGLALAHFVWIAPLQVALLMGLIWELLQASAFCGLGFLIVLALFQAGL
GRMMMKYRDQRAGKISERLVITSEMIENIQSVKAYCWEEAMEKMIENLRQTELKLTRKAA
YVRYFNSSAFFFSGFFVVFLSVLPYALIKGIILRKIFTTISFCIVLRMAVTRQFPWAVQT
WYDSLGAINKIQDFLQKQEYKTLEYNLTTTEVVMENVTAFWEEGFGELFEKAKQNNNNRK
TSNGDDSLFFSNFSLLGTPVLKDINFKIERGQLLAVAGSTGAGKTSLLMVIMGELEPSEG
KIKHSGRISFCSQFSWIMPGTIKENIIFGVSYDEYRYRSVIKACQLEEDISKFAEKDNIV
LGEGGITLSGGQRARISLARAVYKDADLYLLDSPFGYLDVLTEKEIFESCVCKLMANKTR
ILVTSKMEHLKKADKILILHEGSSYFYGTFSELQNLQPDFSSKLMGCDSFDQFSAERRNS
ILTETLHRFSLEGDAPVSWTETKKQSFKQTGEFGEKRKNSILNPINSIRKFSIVQKTPLQ
MNGIEEDSDEPLERRLSLVPDSEQGEAILPRISVISTGPTLQARRRQSVLNLMTHSVNQG
QNIHRKTTASTRKVSLAPQANLTELDIYSRRLSQETGLEISEEINEEDLKECFFDDMESI
PAVTTWNTYLRYITVHKSLIFVLIWCLVIFLAEVAASLVVLWLLGNTPLQDKGNSTHSRN
NSYAVIITSTSSYYVFYIYVGVADTLLAMGFFRGLPLVHTLITVSKILHHKMLHSVLQAP
MSTLNTLKAGGILNRFSKDIAILDDLLPLTIFDFIQLLLIVIGAIAVVAVLQPYIFVATV
PVIVAFIMLRAYFLQTSQQLKQLESEGRSPIFTHLVTSLKGLWTLRAFGRQPYFETLFHK
ALNLHTANWFLYLSTLRWFQMRIEMIFVIFFIAVTFISILTTGEGEGRVGIILTLAMNIM
STLQWAVNSSIDVDSLMRSVSRVFKFIDMPTEGKPTKSTKPYKNGQLSKVMIIENSHVKK
DDIWPSGGQMTVKDLTAKYTEGGNAILENISFSISPGQRVGLLGRTGSGKSTLLSAFLRL
LNTEGEIQIDGVSWDSITLQQWRKAFGVIPQKVFIFSGTFRKNLDPYEQWSDQEIWKVAD
EVGLRSVIEQFPGKLDFVLVDGGCVLSHGHKQLMCLARSVLSKAKILLLDEPSAHLDPVT
YQIIRRTLKQAFADCTVILCEHRIEAMLECQQFLVIEENKVRQYDSIQKLLNERSLFRQA
ISPSDRVKLFPHRNSSKCKSKPQIAALKEETEEEVQDTRL
Drugs and Mode of Action
Drug(s) Crofelemer Drug Info Approved HIV-associated diarrhoea [530433], [532210], [542477]
Ivacaftor Drug Info Approved Cystic fibrosis [467679], [532210]
iOWH032 Drug Info Phase 2 Diarrhea [524714]
Lumacaftor Drug Info Phase 2 Cystic fibrosis [522612], [542504]
VX-661 Drug Info Phase 2 Cystic fibrosis [525275]
Activator CBIQ Drug Info [543927]
phenylglycine-01 Drug Info [543927]
sulfonamide-01 Drug Info [543927]
UCCF-029 Drug Info [543927]
UCCF-339 Drug Info [543927]
UCCF-853 Drug Info [543927]
Inhibitor CFTR inhibitors Drug Info [543927]
iOWH032 Drug Info [531605]
Blocker (channel blocker) CFTRinh-172 Drug Info [543927]
GlyH-101 Drug Info [543927]
Modulator Crofelemer Drug Info [530433], [532210]
Ivacaftor Drug Info [551871]
Regulator Lumacaftor Drug Info [532863]
VX-661 Drug Info [532904]
Pathways
KEGG Pathway ABC transporters
cAMP signaling pathway
AMPK signaling pathway
Gastric acid secretion
Pancreatic secretion
Bile secretion
Vibrio cholerae infection
Reactome ABC-family proteins mediated transport
WikiPathways ABC-family proteins mediated transport
References
Ref 467679(http://www.guidetopharmacology.org/) Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Ligand id: 4342).
Ref 522612ClinicalTrials.gov (NCT00865904) Study of VX-809 in Cystic Fibrosis Subjects With the ???508-CFTR Gene Mutation. U.S. National Institutes of Health.
Ref 524714ClinicalTrials.gov (NCT02111304) Efficacy of iOWH032 in Dehydrating Cholera. U.S. National Institutes of Health.
Ref 525275ClinicalTrials.gov (NCT02508207) A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation.
Ref 530433Crofelemer, an antisecretory antidiarrheal proanthocyanidin oligomer extracted from Croton lechleri, targets two distinct intestinal chloride channels. Mol Pharmacol. 2010 Jan;77(1):69-78.
Ref 532210Nat Rev Drug Discov. 2013 Feb;12(2):87-90.
Ref 542477(http://www.guidetopharmacology.org/) Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Ligand id: 7453).
Ref 542504(http://www.guidetopharmacology.org/) Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Ligand id: 7481).
Ref 530433Crofelemer, an antisecretory antidiarrheal proanthocyanidin oligomer extracted from Croton lechleri, targets two distinct intestinal chloride channels. Mol Pharmacol. 2010 Jan;77(1):69-78.
Ref 531605Developing novel antisecretory drugs to treat infectious diarrhea. Future Med Chem. 2011 Aug;3(10):1317-25.
Ref 532210Nat Rev Drug Discov. 2013 Feb;12(2):87-90.
Ref 532863A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial. Lancet Respir Med. 2014 Jul;2(7):527-38.
Ref 532904CFTR Modulators for the Treatment of Cystic Fibrosis. P T. 2014 Jul;39(7):500-11.
Ref 543927(http://www.guidetopharmacology.org/) Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Target id: 707).
Ref 551871Drugs@FDA. U.S. Food and Drug Administration. U.S. Department of Health & Human Services. 2015

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