VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1
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Title
VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1
Authors
Keywords
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Journal
MOLECULAR BIOLOGY OF THE CELL
Volume 24, Issue 19, Pages 3016-3024
Publisher
American Society for Cell Biology (ASCB)
Online
2013-08-08
DOI
10.1091/mbc.e13-05-0240
References
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Note: Only part of the references are listed.- Rescuing Mutant CFTR: A Multi-task Approach to a Better Outcome in Treating Cystic Fibrosis
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- (2012) Juan L. Mendoza et al. CELL
- Correction of Both NBD1 Energetics and Domain Interface Is Required to Restore ΔF508 CFTR Folding and Function
- (2012) Wael M. Rabeh et al. CELL
- Correctors of ΔF508 CFTR restore global conformational maturation without thermally stabilizing the mutant protein
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- Increased folding and channel activity of a rare cystic fibrosis mutant with CFTR modulators
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- Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809
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- The endoplasmic reticulum–associated Hsp40 DNAJB12 and Hsc70 cooperate to facilitate RMA1 E3–dependent degradation of nascent CFTRΔF508
- (2010) Diane E. Grove et al. MOLECULAR BIOLOGY OF THE CELL
- Integrated biophysical studies implicate partial unfolding of NBD1 of CFTR in the molecular pathogenesis of F508del cystic fibrosis
- (2010) Chi Wang et al. PROTEIN SCIENCE
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- CFTR Function and Prospects for Therapy
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- Atomic model of human cystic fibrosis transmembrane conductance regulator: Membrane-spanning domains and coupling interfaces
- (2008) J.-P. Mornon et al. CELLULAR AND MOLECULAR LIFE SCIENCES
- Solubilizing Mutations Used to Crystallize One CFTR Domain Attenuate the Trafficking and Channel Defects Caused by the Major Cystic Fibrosis Mutation
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- Consensus on the use and interpretation of cystic fibrosis mutation analysis in clinical practice
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- Cystic fibrosis: impaired bicarbonate secretion and mucoviscidosis
- (2008) Paul M Quinton LANCET
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- (2008) Meredith F. N. Rosser et al. MOLECULAR BIOLOGY OF THE CELL
- Phenylalanine-508 mediates a cytoplasmic-membrane domain contact in the CFTR 3D structure crucial to assembly and channel function
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