Changing the Paradigm – Treating the Basic Defect in Cystic Fibrosis
Published 2015 View Full Article
- Home
- Publications
- Publication Search
- Publication Details
Title
Changing the Paradigm – Treating the Basic Defect in Cystic Fibrosis
Authors
Keywords
Cystic fibrosis, Lung, Genetic, Mutation, Treatment, Chloride
Journal
INDIAN JOURNAL OF PEDIATRICS
Volume 82, Issue 8, Pages 727-736
Publisher
Springer Nature
Online
2015-06-16
DOI
10.1007/s12098-015-1786-3
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- Pharmacokinetics and tolerability of oral sildenafil in adults with cystic fibrosis lung disease
- (2015) J.L. Taylor-Cousar et al. Journal of Cystic Fibrosis
- CFTR and lung homeostasis
- (2014) James F. Collawn et al. AMERICAN JOURNAL OF PHYSIOLOGY-LUNG CELLULAR AND MOLECULAR PHYSIOLOGY
- CFTR and Anoctamin 1 (ANO1) contribute to cAMP amplified exocytosis and insulin secretion in human and murine pancreatic beta-cells
- (2014) Anna Edlund et al. BMC Medicine
- Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation
- (2014) Kris De Boeck et al. Journal of Cystic Fibrosis
- Some gating potentiators, including VX-770, diminish F508-CFTR functional expression
- (2014) G. Veit et al. Science Translational Medicine
- Potentiator ivacaftor abrogates pharmacological correction of F508 CFTR in cystic fibrosis
- (2014) D. M. Cholon et al. Science Translational Medicine
- Exon-Skipping Antisense Oligonucleotides to Correct Missplicing in Neurogenetic Diseases
- (2014) Kavitha Siva et al. Nucleic Acid Therapeutics
- 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
- Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp- CFTR mutation: a phase 3, open-label extension study (PERSIST)
- (2014) Edward F McKone et al. Lancet Respiratory Medicine
- Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with aG551DMutation
- (2013) Jane C. Davies et al. AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE
- High-Content siRNA Screen Reveals Global ENaC Regulators and Potential Cystic Fibrosis Therapy Targets
- (2013) Joana Almaça et al. CELL
- Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients
- (2013) Gerald Schwank et al. Cell Stem Cell
- WS7.3 VX-661, an investigational CFTR corrector, in combination with ivacaftor, a CFTR potentiator, in patients with CF and homozygous for the F508Del-CFTR mutation: Interim analysis
- (2013) S. Donaldson et al. Journal of Cystic Fibrosis
- VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1
- (2013) Hong Yu Ren et al. MOLECULAR BIOLOGY OF THE CELL
- Pharmacological Characterization of a Novel ENaCα siRNA (GSK2225745) With Potential for the Treatment of Cystic Fibrosis
- (2013) Kenneth L Clark et al. Molecular Therapy-Nucleic Acids
- 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
- Neutrophil Elastase Degrades Cystic Fibrosis Transmembrane Conductance Regulator via Calpains and Disables Channel FunctionIn VitroandIn Vivo
- (2012) Mathieu Le Gars et al. AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE
- Ivacaftor in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation
- (2012) Patrick A. Flume et al. CHEST
- Airway epithelial cells—Functional links between CFTR and anoctamin dependent Cl− secretion
- (2012) Karl Kunzelmann et al. INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY
- Long term effects of denufosol tetrasodium in patients with cystic fibrosis
- (2012) Felix Ratjen et al. Journal of Cystic Fibrosis
- A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation
- (2011) Bonnie W. Ramsey et al. NEW ENGLAND JOURNAL OF MEDICINE
- 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
- Ataluren (PTC124) Induces Cystic Fibrosis Transmembrane Conductance Regulator Protein Expression and Activity in Children with Nonsense Mutation Cystic Fibrosis
- (2010) Isabelle Sermet-Gaudelus et al. AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE
- Denufosol Tetrasodium in Patients with Cystic Fibrosis and Normal to Mildly Impaired Lung Function
- (2010) Frank J. Accurso et al. AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE
- Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTRMutation
- (2010) Frank J. Accurso et al. NEW ENGLAND JOURNAL OF MEDICINE
- 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
- Epithelial Sodium Channel Inhibition in Primary Human Bronchial Epithelia by Transfected siRNA
- (2008) Emanuela Caci et al. AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY
- Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial
- (2008) Eitan Kerem et al. LANCET
Publish scientific posters with Peeref
Peeref publishes scientific posters from all research disciplines. Our Diamond Open Access policy means free access to content and no publication fees for authors.
Learn MoreAdd your recorded webinar
Do you already have a recorded webinar? Grow your audience and get more views by easily listing your recording on Peeref.
Upload Now