标题
Phenotypic profiling of CFTR modulators in patient-derived respiratory epithelia
作者
关键词
-
出版物
npj Genomic Medicine
Volume 2, Issue 1, Pages -
出版商
Springer Nature
发表日期
2017-04-10
DOI
10.1038/s41525-017-0015-6
参考文献
相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。- Discovery of Clinically Approved Agents That Promote Suppression of Cystic Fibrosis Transmembrane Conductance Regulator Nonsense Mutations
- (2016) Venkateshwar Mutyam et al. AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE
- Fatty Acid Cysteamine Conjugates as Novel and Potent Autophagy Activators That Enhance the Correction of Misfolded F508del-Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)
- (2016) Chi B. Vu et al. JOURNAL OF MEDICINAL CHEMISTRY
- From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations
- (2016) Gudio Veit et al. MOLECULAR BIOLOGY OF THE CELL
- Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis
- (2016) Johanna F. Dekkers et al. Science Translational Medicine
- Novel personalized therapies for cystic fibrosis: treating the basic defect in all patients
- (2015) M. D. Amaral JOURNAL OF INTERNAL MEDICINE
- Mucosal production of uric acid by airway epithelial cells contributes to particulate matter-induced allergic sensitization
- (2015) M J Gold et al. Mucosal Immunology
- Directed differentiation of cholangiocytes from human pluripotent stem cells
- (2015) Mina Ogawa et al. NATURE BIOTECHNOLOGY
- Efficient generation of functional CFTR-expressing airway epithelial cells from human pluripotent stem cells
- (2015) Amy P Wong et al. Nature Protocols
- Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR
- (2015) Claire E. Wainwright et al. NEW ENGLAND JOURNAL OF MEDICINE
- Testing gene therapy vectors in human primary nasal epithelial cultures
- (2015) Huibi Cao et al. Molecular Therapy-Methods & Clinical Development
- VX-809 and Related Corrector Compounds Exhibit Secondary Activity Stabilizing Active F508del-CFTR after Its Partial Rescue to the Cell Surface
- (2014) Paul D.W. Eckford et al. CHEMISTRY & BIOLOGY
- Clinical Pharmacogenetics Implementation Consortium (CPIC) Guidelines for Ivacaftor Therapy in the Context of CFTR Genotype
- (2014) J P Clancy et al. CLINICAL PHARMACOLOGY & THERAPEUTICS
- Combined effects of VX-770 and VX-809 on several functional abnormalities of F508del-CFTR channels
- (2014) Z. Kopeikin et al. Journal of Cystic Fibrosis
- Cystic fibrosis genetics: from molecular understanding to clinical application
- (2014) Garry R. Cutting NATURE REVIEWS GENETICS
- The major cystic fibrosis causing mutation exhibits defective propensity for phosphorylation
- (2014) Stan Pasyk et al. PROTEOMICS
- Some gating potentiators, including VX-770, diminish F508-CFTR functional expression
- (2014) G. Veit et al. Science Translational Medicine
- A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial
- (2014) Michael P Boyle et al. Lancet Respiratory Medicine
- Efficient generation of lung and airway epithelial cells from human pluripotent stem cells
- (2013) Sarah X L Huang et al. NATURE BIOTECHNOLOGY
- A functional CFTR assay using primary cystic fibrosis intestinal organoids
- (2013) Johanna F Dekkers et al. NATURE MEDICINE
- Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Potentiator VX-770 (Ivacaftor) Opens the Defective Channel Gate of Mutant CFTR in a Phosphorylation-dependent but ATP-independent Manner
- (2012) Paul D. W. Eckford et al. JOURNAL OF BIOLOGICAL CHEMISTRY
- Ivacaftor potentiation of multiple CFTR channels with gating mutations
- (2012) Haihui Yu et al. Journal of Cystic Fibrosis
- Directed differentiation of human pluripotent stem cells into mature airway epithelia expressing functional CFTR protein
- (2012) Amy P Wong et al. NATURE BIOTECHNOLOGY
- Functional Rescue of F508del-CFTR Using Small Molecule Correctors
- (2012) Steven Molinski et al. Frontiers in Pharmacology
- A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation
- (2011) Bonnie W. Ramsey et al. NEW ENGLAND JOURNAL OF MEDICINE
- 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
- Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770
- (2009) F. Van Goor et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Evidence for direct CFTR inhibition by CFTRinh-172 based on Arg347mutagenesis
- (2008) Emanuela Caci et al. BIOCHEMICAL JOURNAL
- High-content Functional Screen to Identify Proteins that Correct F508del-CFTR Function
- (2008) Agata M. Trzcińska-Daneluti et al. MOLECULAR & CELLULAR PROTEOMICS
- Multidrug resistance protein 4 (MRP4/ABCC4): a versatile efflux transporter for drugs and signalling molecules
- (2008) F RUSSEL et al. TRENDS IN PHARMACOLOGICAL SCIENCES
Become a Peeref-certified reviewer
The Peeref Institute provides free reviewer training that teaches the core competencies of the academic peer review process.
Get StartedAsk a Question. Answer a Question.
Quickly pose questions to the entire community. Debate answers and get clarity on the most important issues facing researchers.
Get Started