VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1
出版年份 2013 全文链接
标题
VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1
作者
关键词
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出版物
MOLECULAR BIOLOGY OF THE CELL
Volume 24, Issue 19, Pages 3016-3024
出版商
American Society for Cell Biology (ASCB)
发表日期
2013-08-08
DOI
10.1091/mbc.e13-05-0240
参考文献
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注意:仅列出部分参考文献,下载原文获取全部文献信息。- Rescuing Mutant CFTR: A Multi-task Approach to a Better Outcome in Treating Cystic Fibrosis
- (2013) Margarida D. Amaral et al. CURRENT PHARMACEUTICAL DESIGN
- HGF Stimulation of Rac1 Signaling Enhances Pharmacological Correction of the Most Prevalent Cystic Fibrosis Mutant F508del-CFTR
- (2012) Sónia Moniz et al. ACS Chemical Biology
- Requirements for Efficient Correction of ΔF508 CFTR Revealed by Analyses of Evolved Sequences
- (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
- (2012) Lihua He et al. FASEB JOURNAL
- Increased folding and channel activity of a rare cystic fibrosis mutant with CFTR modulators
- (2011) Ray A. Caldwell et al. AMERICAN JOURNAL OF PHYSIOLOGY-LUNG CELLULAR AND MOLECULAR PHYSIOLOGY
- Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809
- (2011) F. Van Goor et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for theF508del-CFTRmutation
- (2011) J P Clancy et al. THORAX
- CFTR: folding, misfolding and correcting the ΔF508 conformational defect
- (2011) Gergely L. Lukacs et al. TRENDS IN MOLECULAR MEDICINE
- 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
- Structure and Dynamics of NBD1 from CFTR Characterized Using Crystallography and Hydrogen/Deuterium Exchange Mass Spectrometry
- (2009) H.A. Lewis et al. JOURNAL OF MOLECULAR BIOLOGY
- Cooperative Assembly and Misfolding of CFTR Domains In Vivo
- (2009) Kai Du et al. MOLECULAR BIOLOGY OF THE CELL
- CFTR Function and Prospects for Therapy
- (2008) John R. Riordan Annual Review of Biochemistry
- 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
- (2008) Luísa S. Pissarra et al. CHEMISTRY & BIOLOGY
- Consensus on the use and interpretation of cystic fibrosis mutation analysis in clinical practice
- (2008) C. Castellani et al. Journal of Cystic Fibrosis
- Cystic fibrosis: impaired bicarbonate secretion and mucoviscidosis
- (2008) Paul M Quinton LANCET
- Assembly and Misassembly of Cystic Fibrosis Transmembrane Conductance Regulator: Folding Defects Caused by Deletion of F508 Occur Before and After the Calnexin-dependent Association of Membrane Spanning Domain (MSD) 1 and MSD2
- (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
- (2008) A. W. R. Serohijos et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
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