VX-809, a CFTR corrector, increases the cell surface density of functional F508del-CFTR in pre-clinical models of cystic fibrosis

Pediatric Pulmonology

Volumn 44, Issue SUPPL. 32, 2009, Pages 154-155

  Van Goor, Fredrick ^a   Hadida, Sabine ^a   Grootenhuis, Peter D ^a   Burton, Bill ^a   Stack, Jeffrey ^a   Cao, Dong ^a   Neuberger, Tim ^a   Singh, Ashvani ^a   Olson, Eric R ^a   Wine, Jeffrey J ^b   Frizzell, Raymond A ^c   Ashlock, Melissa ^d   Negulescu, Paul ^a  

^a VERTEX PHARMACEUTICALS   (United States)

^b STANFORD UNIVERSITY   (United States)

^c UNIVERSITY OF PITTSBURGH   (United States)

^d CYSTIC FIBROSIS FOUNDATION   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTIFIBROTIC AGENT; TRANSMEMBRANE CONDUCTANCE REGULATOR; UNCLASSIFIED DRUG; VX 770; VX 809;

ARTICLE; CELL DENSITY; DISEASE COURSE; DISEASE SEVERITY; DRUG ACTIVITY; DRUG BIOAVAILABILITY; DRUG EFFECT; HUMAN; IN VITRO STUDY; IN VIVO STUDY; LUNG ALVEOLUS EPITHELIUM; LUNG FIBROSIS; PATHOGENESIS; PRIORITY JOURNAL; PROTEIN FUNCTION;

EID: 70349778630     PISSN: 87556863     EISSN: 10990496     Source Type: Journal    
DOI: None     Document Type: Article

References (8)

1. Riordan JR, Rommens JM, Kerem B, Alon N, Rozmahel R, Grzelczak Z, Zielenski J, Lok S, Plavsic N, Chou JL, et al. Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA. Science 1989;245:1066-1073. (Pubitemid 19231814)
2. Welsh MJ, Smith AE. Molecular mechanisms of CFTR chloride channel dysfunction in cystic fibrosis. Cell 1993;73:1251-1254. (Pubitemid 23201140)
3. Boucher RC. Cystic fibrosis: a disease of vulnerability to airway surface dehydration. Trends in molecular medicine 2007;13:231-240. (Pubitemid 46836874)
4. Van Goor F, Straley KS, Cao D, Gonzalez J, Hadida S, Hazlewood A, Joubran J, Knapp T, Makings LR, Miller M, Neuberger T, Olson E, Panchenko V, Rader J, Singh A, Stack JH, Tung R, Grootenhuis PD, Negulescu P. Rescue of DeltaF508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules. Am J Physiol 2006;290:L1117-1130. (Pubitemid 43830856)
5. Davis PB, Drumm M, Konstan MW. Cystic fibrosis. Am J Resp Crit Care Med 1996;154:1229-1256.
6. McKone EF, Emerson SS, Edwards KL, Aitken ML. Effect of genotype on phenotype and mortality in cystic fibrosis: a retrospective cohort study. Lancet 2003;361:1671-1676.
7. Noone PG, Pue CA, Zhou Z, Friedman KJ, Wakeling EL, Ganeshananthan M, Simon RH, Silverman LM, Knowles MR. Lung disease associated with the IVS8 5T allele of the CFTR gene. Am J Resp Crit Care Med 2000;162:1919-1924. (Pubitemid 30947582)
8. Noone PG, Zhou Z, Silverman LM, Jowell PS, Knowles MR, Cohn JA. Cystic fibrosis gene mutations and pancreatitis risk: relation to epithelial ion transport and trypsin inhibitor gene mutations. Gastroenterology 2001;121:1310-1319. (Pubitemid 33140448)