Genomes and Genes
Fredrick Van Goor
Affiliation: Vertex Pharmaceuticals
- Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or functionFredrick Van Goor
Vertex Pharmaceuticals Incorporated, 130 Waverly St, Cambridge, MA, United States Vertex Pharmaceuticals Incorporated, 11010 Torreyana Road, San Diego, CA 92121, United States Electronic address
J Cyst Fibros 13:29-36. 2014..The aim of this in vitro study was to evaluate the effect of ivacaftor on mutant CFTR protein forms with defects in protein processing and/or channel function...
- Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770Fredrick Van Goor
Vertex Pharmaceuticals Incorporated, 11010 Torreyana Road, San Diego, CA 92121, USA
Proc Natl Acad Sci U S A 106:18825-30. 2009..These results support the hypothesis that pharmacological agents that restore or increase CFTR function can rescue epithelial cell function in human CF airway...
- Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809Fredrick Van Goor
Vertex Pharmaceuticals, San Diego, CA 92121, USA
Proc Natl Acad Sci U S A 108:18843-8. 2011..VX-809 was more efficacious and selective for CFTR than previously reported CFTR correctors. VX-809 represents a class of CFTR corrector that specifically addresses the underlying processing defect in F508del-CFTR...
- Ivacaftor potentiation of multiple CFTR channels with gating mutationsHaihui Yu
Vertex Pharmaceuticals Incorporated, Cambridge, MA 02139, USA
J Cyst Fibros 11:237-45. 2012..The aim of this in vitro study was to determine whether ivacaftor potentiates mutant CFTR with gating defects caused by other CFTR gating mutations...
- Rescue of DeltaF508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small moleculesFredrick Van Goor
Vertex Pharmaceuticals, 11010 Torreyana Road, San Diego, CA 92121, USA
Am J Physiol Lung Cell Mol Physiol 290:L1117-30. 2006..The CFTR-activating effects of the two mechanisms are additive and support the rationale of a drug discovery strategy based on rescue of the basic genetic defect responsible for CF...
- Use of primary cultures of human bronchial epithelial cells isolated from cystic fibrosis patients for the pre-clinical testing of CFTR modulatorsTimothy Neuberger
Vertex Pharmaceuticals Incorporated, San Diego, CA 92121, USA
Methods Mol Biol 741:39-54. 2011..It is expected that the pre-clinical testing of CFTR potentiators and correctors using epithelial cell cultures derived from CF patients will help to increase their likelihood of clinical efficacy...
- VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1Hong Yu Ren
Department of Cell Biology and University of North Carolina Cystic Fibrosis Center, School of Medicine, University of North Carolina, Chapel Hill, NC 27599 Vertex Pharmaceuticals, San Diego, CA 92121
Mol Biol Cell 24:3016-24. 2013..These data suggest that the use of VX-809 in combination with an additional CFTR corrector that suppresses folding defects downstream of MSD1 may further enhance CFTR function in people with F508del-CFTR. ..
- Discovery of N-(2,4-di-tert-butyl-5-hydroxyphenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide (VX-770, ivacaftor), a potent and orally bioavailable CFTR potentiatorSabine Hadida
Vertex Pharmaceuticals Incorporated, 11010 Torreyana Road, San Diego, California 92121, United States
J Med Chem 57:9776-95. 2014....