Lumacaftor (BioDeep_00000858628)
代谢物信息卡片
VX-809(Lumacaftor)
化学式: C24H18F2N2O5 (452.11837219999995)
中文名称: 芦马卡托
谱图信息:
最多检出来源 () 0%
分子结构信息
SMILES: CC1=C(N=C(C=C1)NC(=O)C2(CC2)C3=CC4=C(C=C3)OC(O4)(F)F)C5=CC(=CC=C5)C(=O)O
InChI: InChI=1S/C24H18F2N2O5/c1-13-5-8-19(27-20(13)14-3-2-4-15(11-14)21(29)30)28-22(31)23(9-10-23)16-6-7-17-18(12-16)33-24(25,26)32-17/h2-8,11-12H,9-10H2,1H3,(H,29,30)(H,27,28,31)
数据库引用编号
6 个数据库交叉引用编号
- ChEBI: CHEBI:90951
- KEGGdrug: D10134
- PubChem: 16678941
- DrugBank: DB09280
- ChEMBL: CHEMBL2103870
- CAS: 936727-05-8
分类词条
相关代谢途径
Reactome(0)
BioCyc(0)
PlantCyc(0)
代谢反应
0 个相关的代谢反应过程信息。
Reactome(0)
BioCyc(0)
WikiPathways(0)
Plant Reactome(0)
INOH(0)
PlantCyc(0)
COVID-19 Disease Map(0)
PathBank(0)
PharmGKB(0)
0 个相关的物种来源信息
在这里通过桑基图来展示出与当前的这个代谢物在我们的BioDeep知识库中具有相关联信息的其他代谢物。在这里进行关联的信息来源主要有：
- PubMed: 来源于PubMed文献库中的文献信息，我们通过自然语言数据挖掘得到的在同一篇文献中被同时提及的相关代谢物列表，这个列表按照代谢物同时出现的文献数量降序排序，取前10个代谢物作为相关研究中关联性很高的代谢物集合展示在桑基图中。
- NCBI Taxonomy: 通过文献数据挖掘，得到的代谢物物种来源信息关联。这个关联信息同样按照出现的次数降序排序，取前10个代谢物作为高关联度的代谢物集合展示在桑吉图上。
- Chemical Taxonomy: 在物质分类上处于同一个分类集合中的其他代谢物
- Chemical Reaction: 在化学反应过程中，存在为当前代谢物相关联的生化反应过程中的反应底物或者反应产物的关联代谢物信息。
点击图上的相关代谢物的名称，可以跳转到相关代谢物的信息页面。
文献列表
- Parniya Arooj, David V Morrissy, Yvonne McCarthy, Tamara Vagg, Mairead McCarthy, Claire Fleming, Mary Daly, Joseph A Eustace, Desmond M Murphy, B J Plant. ROCK STUDY in CF: sustained anti-inflammatory effects of lumacaftor-ivacaftor in sputum and peripheral blood samples of adult patients with cystic fibrosis-an observational study.
BMJ open respiratory research.
2023 May; 10(1):. doi:
10.1136/bmjresp-2022-001590. [PMID: 37130650] - Mathias Schenkel, Dorna Ravamehr-Lake, Tomasz Czerniak, James P Saenz, Georg Krainer, Michael Schlierf, Charles M Deber. Impact of cholesterol and Lumacaftor on the folding of CFTR helical hairpins.
Biochimica et biophysica acta. Biomembranes.
2023 01; 1865(1):184078. doi:
10.1016/j.bbamem.2022.184078. [PMID: 36279907] - Clarissa Braccia, Josie A Christopher, Oliver M Crook, Lisa M Breckels, Rayner M L Queiroz, Nara Liessi, Valeria Tomati, Valeria Capurro, Tiziano Bandiera, Simona Baldassari, Nicoletta Pedemonte, Kathryn S Lilley, Andrea Armirotti. CFTR Rescue by Lumacaftor (VX-809) Induces an Extensive Reorganization of Mitochondria in the Cystic Fibrosis Bronchial Epithelium.
Cells.
2022 06; 11(12):. doi:
10.3390/cells11121938. [PMID: 35741067] - David Drummond, Jérémy Dana, Laureline Berteloot, Elena K Schneider-Futschik, Frédérique Chedevergne, Céline Bailly-Botuha, Thao Nguyen-Khoa, Mathieu Cornet, Muriel Le Bourgeois, Dominique Debray, Muriel Girard, Isabelle Sermet-Gaudelus. Lumacaftor-ivacaftor effects on cystic fibrosis-related liver involvement in adolescents with homozygous F508 del-CFTR.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.
2022 03; 21(2):212-219. doi:
10.1016/j.jcf.2021.07.018. [PMID: 34454846] - Katharina Habler, Anne-Sophie Kalla, Michael Rychlik, Mathias Bruegel, Daniel Teupser, Susanne Nährig, Michael Vogeser, Michael Paal. Isotope dilution LC-MS/MS quantification of the cystic fibrosis transmembrane conductance regulator (CFTR) modulators ivacaftor, lumacaftor, tezacaftor, elexacaftor, and their major metabolites in human serum.
Clinical chemistry and laboratory medicine.
2022 01; 60(1):82-91. doi:
10.1515/cclm-2021-0724. [PMID: 34668357] - Nesrine Baatallah, Ahmad Elbahnsi, Jean-Paul Mornon, Benoit Chevalier, Iwona Pranke, Nathalie Servel, Renaud Zelli, Jean-Luc Décout, Aleksander Edelman, Isabelle Sermet-Gaudelus, Isabelle Callebaut, Alexandre Hinzpeter. Pharmacological chaperones improve intra-domain stability and inter-domain assembly via distinct binding sites to rescue misfolded CFTR.
Cellular and molecular life sciences : CMLS.
2021 Dec; 78(23):7813-7829. doi:
10.1007/s00018-021-03994-5. [PMID: 34714360] - Murali K Yanda, Liudmila Cebotaru. VX-809 mitigates disease in a mouse model of autosomal dominant polycystic kidney disease bearing the R3277C human mutation.
FASEB journal : official publication of the Federation of American Societies for Experimental Biology.
2021 11; 35(11):e21987. doi:
10.1096/fj.202101315r. [PMID: 34662459] - C Colombo, A Foppiani, A Bisogno, S Gambazza, V Daccò, E Nazzari, A Leone, A Giana, A Mari, A Battezzati. Lumacaftor/ivacaftor in cystic fibrosis: effects on glucose metabolism and insulin secretion.
Journal of endocrinological investigation.
2021 Oct; 44(10):2213-2218. doi:
10.1007/s40618-021-01525-4. [PMID: 33586024] - Chiara Brandas, Alessandra Ludovico, Alice Parodi, Oscar Moran, Enrico Millo, Elena Cichero, Debora Baroni. NBD2 Is Required for the Rescue of Mutant F508del CFTR by a Thiazole-Based Molecule: A Class II Corrector for the Multi-Drug Therapy of Cystic Fibrosis.
Biomolecules.
2021 09; 11(10):. doi:
10.3390/biom11101417. [PMID: 34680050] - Steffie E M Vonk, Marloes van der Meer-Vos, Lieuwe D J Bos, Anne H Neerincx, Christof J Majoor, Anke-Hilse Maitland-van der Zee, Ron A A Mathôt, E Marleen Kemper. Quantitative Method for the Analysis of Ivacaftor, Hydroxymethyl Ivacaftor, Ivacaftor Carboxylate, Lumacaftor, and Tezacaftor in Plasma and Sputum Using Liquid Chromatography With Tandem Mass Spectrometry and Its Clinical Applicability.
Therapeutic drug monitoring.
2021 08; 43(4):555-563. doi:
10.1097/ftd.0000000000000829. [PMID: 33165217] - Christopher J Day, Benjamin Bailly, Patrice Guillon, Larissa Dirr, Freda E-C Jen, Belinda L Spillings, Johnson Mak, Mark von Itzstein, Thomas Haselhorst, Michael P Jennings. Multidisciplinary Approaches Identify Compounds that Bind to Human ACE2 or SARS-CoV-2 Spike Protein as Candidates to Block SARS-CoV-2-ACE2 Receptor Interactions.
mBio.
2021 03; 12(2):. doi:
10.1128/mbio.03681-20. [PMID: 33785634] - Miquéias Lopes-Pacheco, Iris A L Silva, Mark J Turner, Graeme W Carlile, Elvira Sondo, David Y Thomas, Nicoletta Pedemonte, John W Hanrahan, Margarida D Amaral. Characterization of the mechanism of action of RDR01752, a novel corrector of F508del-CFTR.
Biochemical pharmacology.
2020 10; 180(?):114133. doi:
10.1016/j.bcp.2020.114133. [PMID: 32628927] - Alfonso Trezza, Daniele Iovinelli, Annalisa Santucci, Filippo Prischi, Ottavia Spiga. An integrated drug repurposing strategy for the rapid identification of potential SARS-CoV-2 viral inhibitors.
Scientific reports.
2020 08; 10(1):13866. doi:
10.1038/s41598-020-70863-9. [PMID: 32807895] - Fanny Degrugillier, Abdel Aissat, Virginie Prulière-Escabasse, Lucie Bizard, Benjamin Simonneau, Xavier Decrouy, Chong Jiang, Daniela Rotin, Pascale Fanen, Stéphanie Simon. Phosphorylation of the Chaperone-Like HspB5 Rescues Trafficking and Function of F508del-CFTR.
International journal of molecular sciences.
2020 Jul; 21(14):. doi:
10.3390/ijms21144844. [PMID: 32650630] - Inna Uliyakina, Hugo M Botelho, Ana C da Paula, Sara Afonso, Miguel J Lobo, Verónica Felício, Carlos M Farinha, Margarida D Amaral. Full Rescue of F508del-CFTR Processing and Function by CFTR Modulators Can Be Achieved by Removal of Two Regulatory Regions.
International journal of molecular sciences.
2020 Jun; 21(12):. doi:
10.3390/ijms21124524. [PMID: 32630527] - Giulia Mancini, Nicoletta Loberto, Debora Olioso, Maria Cristina Dechecchi, Giulio Cabrini, Laura Mauri, Rosaria Bassi, Domitilla Schiumarini, Elena Chiricozzi, Giuseppe Lippi, Emanuela Pesce, Sandro Sonnino, Nicoletta Pedemonte, Anna Tamanini, Massimo Aureli. GM1 as Adjuvant of Innovative Therapies for Cystic Fibrosis Disease.
International journal of molecular sciences.
2020 Jun; 21(12):. doi:
10.3390/ijms21124486. [PMID: 32599772] - Michele Fiore, Cristiana Picco, Oscar Moran. Correctors modify the bicarbonate permeability of F508del-CFTR.
Scientific reports.
2020 05; 10(1):8440. doi:
10.1038/s41598-020-65287-4. [PMID: 32439937] - Kirsten A Cottrill, Carlos M Farinha, Nael A McCarty. The bidirectional relationship between CFTR and lipids.
Communications biology.
2020 04; 3(1):179. doi:
10.1038/s42003-020-0909-1. [PMID: 32313074] - Heledd H Jarosz-Griffiths, Thomas Scambler, Chi H Wong, Samuel Lara-Reyna, Jonathan Holbrook, Fabio Martinon, Sinisa Savic, Paul Whitaker, Christine Etherington, Giulia Spoletini, Ian Clifton, Anil Mehta, Michael F McDermott, Daniel Peckham. Different CFTR modulator combinations downregulate inflammation differently in cystic fibrosis.
eLife.
2020 03; 9(?):. doi:
10.7554/elife.54556. [PMID: 32118580] - Georg Krainer, Mathias Schenkel, Andreas Hartmann, Dorna Ravamehr-Lake, Charles M Deber, Michael Schlierf. CFTR transmembrane segments are impaired in their conformational adaptability by a pathogenic loop mutation and dynamically stabilized by Lumacaftor.
The Journal of biological chemistry.
2020 02; 295(7):1985-1991. doi:
10.1074/jbc.ac119.011360. [PMID: 31882543] - Murali K Yanda, Boyoung Cha, Cristina V Cebotaru, Liudmila Cebotaru. Pharmacological reversal of renal cysts from secretion to absorption suggests a potential therapeutic strategy for managing autosomal dominant polycystic kidney disease.
The Journal of biological chemistry.
2019 11; 294(45):17090-17104. doi:
10.1074/jbc.ra119.010320. [PMID: 31570523] - Giulia Amico, Chiara Brandas, Oscar Moran, Debora Baroni. Unravelling the Regions of Mutant F508del-CFTR More Susceptible to the Action of Four Cystic Fibrosis Correctors.
International journal of molecular sciences.
2019 Nov; 20(21):. doi:
10.3390/ijms20215463. [PMID: 31683989] - Ana M Matos, Francisco R Pinto, Patrícia Barros, Margarida D Amaral, Rainer Pepperkok, Paulo Matos. Inhibition of calpain 1 restores plasma membrane stability to pharmacologically rescued Phe508del-CFTR variant.
The Journal of biological chemistry.
2019 09; 294(36):13396-13410. doi:
10.1074/jbc.ra119.008738. [PMID: 31324722] - Samuel J Bose, Marcel J C Bijvelds, Yiting Wang, Jia Liu, Zhiwei Cai, Alice G M Bot, Hugo R de Jonge, David N Sheppard. Differential thermostability and response to cystic fibrosis transmembrane conductance regulator potentiators of human and mouse F508del-CFTR.
American journal of physiology. Lung cellular and molecular physiology.
2019 07; 317(1):L71-L86. doi:
10.1152/ajplung.00034.2019. [PMID: 30969810] - Frédéric Anglès, Darren M Hutt, William E Balch. HDAC inhibitors rescue multiple disease-causing CFTR variants.
Human molecular genetics.
2019 06; 28(12):1982-2000. doi:
10.1093/hmg/ddz026. [PMID: 30753450] - John J McNamara, Susanna A McColley, Gautham Marigowda, Fang Liu, Simon Tian, Caroline A Owen, David Stiles, Chonghua Li, David Waltz, Linda T Wang, Gregory S Sawicki. Safety, pharmacokinetics, and pharmacodynamics of lumacaftor and ivacaftor combination therapy in children aged 2-5 years with cystic fibrosis homozygous for F508del-CFTR: an open-label phase 3 study.
The Lancet. Respiratory medicine.
2019 04; 7(4):325-335. doi:
10.1016/s2213-2600(18)30460-0. [PMID: 30686767] - O B Garbuzenko, N Kbah, A Kuzmov, N Pogrebnyak, V Pozharov, T Minko. Inhalation treatment of cystic fibrosis with lumacaftor and ivacaftor co-delivered by nanostructured lipid carriers.
Journal of controlled release : official journal of the Controlled Release Society.
2019 02; 296(?):225-231. doi:
10.1016/j.jconrel.2019.01.025. [PMID: 30677435] - Qiangni Liu, Inna Sabirzhanova, Emily Anne Smith Bergbower, Murali Yanda, William G Guggino, Liudmila Cebotaru. The CFTR Corrector, VX-809 (Lumacaftor), Rescues ABCA4 Trafficking Mutants: a Potential Treatment for Stargardt Disease.
Cellular physiology and biochemistry : international journal of experimental cellular physiology, biochemistry, and pharmacology.
2019; 53(2):400-412. doi:
10.33594/000000146. [PMID: 31403270] - Jeffrey D Brand, Ahmed Lazrak, John E Trombley, Ren-Jay Shei, A Timothy Adewale, Jennifer L Tipper, Zhihong Yu, Amit R Ashtekar, Steven M Rowe, Sadis Matalon, Kevin S Harrod. Influenza-mediated reduction of lung epithelial ion channel activity leads to dysregulated pulmonary fluid homeostasis.
JCI insight.
2018 10; 3(20):. doi:
10.1172/jci.insight.123467. [PMID: 30333319] - Qiangni Liu, Inna Sabirzhanova, Murali K Yanda, Emily A S Bergbower, Clément Boinot, William B Guggino, Liudmila Cebotaru. Rescue of CFTR NBD2 mutants N1303K and S1235R is influenced by the functioning of the autophagosome.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.
2018 09; 17(5):582-594. doi:
10.1016/j.jcf.2018.05.016. [PMID: 29936070] - Ana M Matos, Andreia Gomes-Duarte, Márcia Faria, Patrícia Barros, Peter Jordan, Margarida D Amaral, Paulo Matos. Prolonged co-treatment with HGF sustains epithelial integrity and improves pharmacological rescue of Phe508del-CFTR.
Scientific reports.
2018 08; 8(1):13026. doi:
10.1038/s41598-018-31514-2. [PMID: 30158635] - Stephanie Chin, Maurita Hung, Amy Won, Yu-Sheng Wu, Saumel Ahmadi, Donghe Yang, Salma Elmallah, Krimo Toutah, C Michael Hamilton, Robert N Young, Russell D Viirre, Christopher M Yip, Christine E Bear. Lipophilicity of the Cystic Fibrosis Drug, Ivacaftor (VX-770), and Its Destabilizing Effect on the Major CF-causing Mutation: F508del.
Molecular pharmacology.
2018 08; 94(2):917-925. doi:
10.1124/mol.118.112177. [PMID: 29903751] - Murali K Yanda, Qiangni Liu, Liudmila Cebotaru. A potential strategy for reducing cysts in autosomal dominant polycystic kidney disease with a CFTR corrector.
The Journal of biological chemistry.
2018 07; 293(29):11513-11526. doi:
10.1074/jbc.ra118.001846. [PMID: 29875161] - Nara Liessi, Elena Cichero, Emanuela Pesce, Maria Arkel, Annalisa Salis, Valeria Tomati, Matteo Paccagnella, Gianluca Damonte, Bruno Tasso, Luis J V Galietta, Nicoletta Pedemonte, Paola Fossa, Enrico Millo. Synthesis and biological evaluation of novel thiazole- VX-809 hybrid derivatives as F508del correctors by QSAR-based filtering tools.
European journal of medicinal chemistry.
2018 Jan; 144(?):179-200. doi:
10.1016/j.ejmech.2017.12.030. [PMID: 29272749] - Xin Meng, Yiting Wang, Xiaomeng Wang, Joe A Wrennall, Tracy L Rimington, Hongyu Li, Zhiwei Cai, Robert C Ford, David N Sheppard. Two Small Molecules Restore Stability to a Subpopulation of the Cystic Fibrosis Transmembrane Conductance Regulator with the Predominant Disease-causing Mutation.
The Journal of biological chemistry.
2017 03; 292(9):3706-3719. doi:
10.1074/jbc.m116.751537. [PMID: 28087700] - Zhihui Zhang, Michael M Baksh, M G Finn, David K Heidary, Christopher I Richards. Direct Measurement of Trafficking of the Cystic Fibrosis Transmembrane Conductance Regulator to the Cell Surface and Binding to a Chemical Chaperone.
Biochemistry.
2017 Jan; 56(1):240-249. doi:
10.1021/acs.biochem.6b00853. [PMID: 28001373] - Elena K Schneider, Felisa Reyes-Ortega, John W Wilson, Tom Kotsimbos, Dominic Keating, Jian Li, Tony Velkov. Development of HPLC and LC-MS/MS methods for the analysis of ivacaftor, its major metabolites and lumacaftor in plasma and sputum of cystic fibrosis patients treated with ORKAMBI or KALYDECO.
Journal of chromatography. B, Analytical technologies in the biomedical and life sciences.
2016 Dec; 1038(?):57-62. doi:
10.1016/j.jchromb.2016.10.026. [PMID: 27792891] - Anna Atlante, Maria Favia, Antonella Bobba, Lorenzo Guerra, Valeria Casavola, Stephan Joel Reshkin. Characterization of mitochondrial function in cells with impaired cystic fibrosis transmembrane conductance regulator (CFTR) function.
Journal of bioenergetics and biomembranes.
2016 06; 48(3):197-210. doi:
10.1007/s10863-016-9663-y. [PMID: 27146408] - Guido Veit, Kathryn Oliver, Pirjo M Apaja, Doranda Perdomo, Aurélien Bidaud-Meynard, Sheng-Ting Lin, Jingyu Guo, Mert Icyuz, Eric J Sorscher, John L Hartman, Gergely L Lukacs. Ribosomal Stalk Protein Silencing Partially Corrects the ΔF508-CFTR Functional Expression Defect.
PLoS biology.
2016 05; 14(5):e1002462. doi:
10.1371/journal.pbio.1002462. [PMID: 27168400] - Elizabeth Matthes, Julie Goepp, Graeme W Carlile, Yishan Luo, Kurt Dejgaard, Arnaud Billet, Renaud Robert, David Y Thomas, John W Hanrahan. Low free drug concentration prevents inhibition of F508del CFTR functional expression by the potentiator VX-770 (ivacaftor).
British journal of pharmacology.
2016 Feb; 173(3):459-70. doi:
10.1111/bph.13365. [PMID: 26492939] - Jinhong Park, Poonam Khloya, Yohan Seo, Satish Kumar, Ho K Lee, Dong-Kyu Jeon, Sungwoo Jo, Pawan K Sharma, Wan Namkung. Potentiation of ΔF508- and G551D-CFTR-Mediated Cl- Current by Novel Hydroxypyrazolines.
PloS one.
2016; 11(2):e0149131. doi:
10.1371/journal.pone.0149131. [PMID: 26863533] - John J Brewington, Gary L McPhail, John P Clancy. Lumacaftor alone and combined with ivacaftor: preclinical and clinical trial experience of F508del CFTR correction.
Expert review of respiratory medicine.
2016; 10(1):5-17. doi:
10.1586/17476348.2016.1122527. [PMID: 26581802] - Cláudia A Loureiro, Ana Margarida Matos, Ângela Dias-Alves, Joana F Pereira, Inna Uliyakina, Patrícia Barros, Margarida D Amaral, Paulo Matos. A molecular switch in the scaffold NHERF1 enables misfolded CFTR to evade the peripheral quality control checkpoint.
Science signaling.
2015 May; 8(377):ra48. doi:
10.1126/scisignal.aaa1580. [PMID: 25990958] - Réginald Philippe, Fabrice Antigny, Paul Buscaglia, Caroline Norez, Frédéric Becq, Maud Frieden, Olivier Mignen. SERCA and PMCA pumps contribute to the deregulation of Ca2+ homeostasis in human CF epithelial cells.
Biochimica et biophysica acta.
2015 May; 1853(5):892-903. doi:
10.1016/j.bbamcr.2015.01.010. [PMID: 25661196] - Debora Baroni, Olga Zegarra-Moran, Oscar Moran. Functional and pharmacological induced structural changes of the cystic fibrosis transmembrane conductance regulator in the membrane solved using SAXS.
Cellular and molecular life sciences : CMLS.
2015 Apr; 72(7):1363-75. doi:
10.1007/s00018-014-1747-4. [PMID: 25274064] - Bruce A Stanton, Bonita Coutermarsh, Roxanna Barnaby, Deborah Hogan. Pseudomonas aeruginosa Reduces VX-809 Stimulated F508del-CFTR Chloride Secretion by Airway Epithelial Cells.
PloS one.
2015; 10(5):e0127742. doi:
10.1371/journal.pone.0127742. [PMID: 26018799] - Puay-Wah Phuan, Guido Veit, Joseph Tan, Ariel Roldan, Walter E Finkbeiner, Gergely L Lukacs, A S Verkman. Synergy-based small-molecule screen using a human lung epithelial cell line yields ΔF508-CFTR correctors that augment VX-809 maximal efficacy.
Molecular pharmacology.
2014 Jul; 86(1):42-51. doi:
10.1124/mol.114.092478. [PMID: 24737137] - Kavisha Arora, Changsuk Moon, Weiqiang Zhang, Sunitha Yarlagadda, Himabindu Penmatsa, Aixia Ren, Chandrima Sinha, Anjaparavanda P Naren. Stabilizing rescued surface-localized δf508 CFTR by potentiation of its interaction with Na(+)/H(+) exchanger regulatory factor 1.
Biochemistry.
2014 Jul; 53(25):4169-79. doi:
10.1021/bi401263h. [PMID: 24945463] - Debora Baroni, Olga Zegarra-Moran, Agneta Svensson, Oscar Moran. Direct interaction of a CFTR potentiator and a CFTR corrector with phospholipid bilayers.
European biophysics journal : EBJ.
2014 Jul; 43(6-7):341-6. doi:
10.1007/s00249-014-0956-y. [PMID: 24771136] - Luis J V Galietta. Managing the underlying cause of cystic fibrosis: a future role for potentiators and correctors.
Paediatric drugs.
2013 Oct; 15(5):393-402. doi:
10.1007/s40272-013-0035-3. [PMID: 23757197] - Tip W Loo, M Claire Bartlett, David M Clarke. Corrector VX-809 stabilizes the first transmembrane domain of CFTR.
Biochemical pharmacology.
2013 Sep; 86(5):612-9. doi:
10.1016/j.bcp.2013.06.028. [PMID: 23835419] - Steven M Rowe, Alan S Verkman. Cystic fibrosis transmembrane regulator correctors and potentiators.
Cold Spring Harbor perspectives in medicine.
2013 Jul; 3(7):. doi:
10.1101/cshperspect.a009761. [PMID: 23818513] - Tsukasa Okiyoneda, Guido Veit, Johanna F Dekkers, Miklos Bagdany, Naoto Soya, Haijin Xu, Ariel Roldan, Alan S Verkman, Mark Kurth, Agnes Simon, Tamas Hegedus, Jeffrey M Beekman, Gergely L Lukacs. Mechanism-based corrector combination restores ΔF508-CFTR folding and function.
Nature chemical biology.
2013 Jul; 9(7):444-54. doi:
10.1038/nchembio.1253. [PMID: 23666117] - Carmen Y S Chu, Jennifer C King, Mattia Berrini, R Todd Alexander, Emmanuelle Cordat. Functional rescue of a kidney anion exchanger 1 trafficking mutant in renal epithelial cells.
PloS one.
2013; 8(2):e57062. doi:
10.1371/journal.pone.0057062. [PMID: 23460825] - Fredrick Van Goor, Sabine Hadida, Peter D J Grootenhuis, Bill Burton, Jeffrey H Stack, Kimberly S Straley, Caroline J Decker, Mark Miller, Jason McCartney, Eric R Olson, Jeffrey J Wine, Ray A Frizzell, Melissa Ashlock, Paul A Negulescu. Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809.
Proceedings of the National Academy of Sciences of the United States of America.
2011 Nov; 108(46):18843-8. doi:
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