Literature DB >> 25083129

CFTR Modulators for the Treatment of Cystic Fibrosis.

Rebecca S Pettit, Chris Fellner.   

Abstract

Defects in a single gene lead to the defective proteins that cause cystic fibrosis, making the disease an ideal candidate for mutation-targeted therapy. Although ivacaftor is currently the only FDA-approved CFTR modifier, others are in development.

Entities:  

Keywords:  4PBA; CFTR; CFTR correctors; CFTR modulators; CFTR potentiators; VRT-532; VX-661; ataluren; cystic fibrosis; cystic fibrosis transmembrane conductance regulator (CFTR); ivacaftor; lumacaftor

Year:  2014        PMID： 25083129      PMCID： PMC4103577     

Source DB:  PubMed          Journal:  P T        ISSN： 1052-1372


  42 in total

1.  Specific rescue of cystic fibrosis transmembrane conductance regulator processing mutants using pharmacological chaperones.

Authors:  Ying Wang; M Claire Bartlett; Tip W Loo; David M Clarke
Journal:  Mol Pharmacol       Date:  2006-04-19       Impact factor: 4.436

Review 2.  Molecular mechanisms of CFTR chloride channel dysfunction in cystic fibrosis.

Authors:  M J Welsh; A E Smith
Journal:  Cell       Date:  1993-07-02       Impact factor: 41.582

3.  Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA.

Authors:  J R Riordan; J M Rommens; B Kerem; N Alon; R Rozmahel; Z Grzelczak; J Zielenski; S Lok; N Plavsic; J L Chou
Journal:  Science       Date:  1989-09-08       Impact factor: 47.728

4.  A pilot clinical trial of oral sodium 4-phenylbutyrate (Buphenyl) in deltaF508-homozygous cystic fibrosis patients: partial restoration of nasal epithelial CFTR function.

Authors:  R C Rubenstein; P L Zeitlin
Journal:  Am J Respir Crit Care Med       Date:  1998-02       Impact factor: 21.405

5.  Drug evaluation: PTC-124--a potential treatment of cystic fibrosis and Duchenne muscular dystrophy.

Authors:  Sherifa A Hamed
Journal:  IDrugs       Date:  2006-11

6.  Assessment of clinical response to ivacaftor with lung clearance index in cystic fibrosis patients with a G551D-CFTR mutation and preserved spirometry: a randomised controlled trial.

Authors:  Jane Davies; Helen Sheridan; Nicholas Bell; Steve Cunningham; Stephanie D Davis; J Stuart Elborn; Carlos E Milla; Timothy D Starner; Daniel J Weiner; Po-Shun Lee; Felix Ratjen
Journal:  Lancet Respir Med       Date:  2013-09-10       Impact factor: 30.700

7.  Fibrosis of the pancreas in infants and children; a statistical study of clinical and hereditary features.

Authors:  C U LOWE; C D MAY; S C REED
Journal:  Am J Dis Child       Date:  1949-09

8.  Lung disease at diagnosis in infants with cystic fibrosis detected by newborn screening.

Authors:  Peter D Sly; Siobhain Brennan; Catherine Gangell; Nicholas de Klerk; Conor Murray; Lauren Mott; Stephen M Stick; Philip J Robinson; Colin F Robertson; Sarath C Ranganathan
Journal:  Am J Respir Crit Care Med       Date:  2009-04-16       Impact factor: 21.405

9.  Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.

Authors:  Eitan Kerem; Michael W Konstan; Kris De Boeck; Frank J Accurso; Isabelle Sermet-Gaudelus; Michael Wilschanski; J Stuart Elborn; Paola Melotti; Inez Bronsveld; Isabelle Fajac; Anne Malfroot; Daniel B Rosenbluth; Patricia A Walker; Susanna A McColley; Christiane Knoop; Serena Quattrucci; Ernst Rietschel; Pamela L Zeitlin; Jay Barth; Gary L Elfring; Ellen M Welch; Arthur Branstrom; Robert J Spiegel; Stuart W Peltz; Temitayo Ajayi; Steven M Rowe
Journal:  Lancet Respir Med       Date:  2014-05-15       Impact factor: 30.700

10.  Effects of ivacaftor in patients with cystic fibrosis who carry the G551D mutation and have severe lung disease.

Authors:  Peter J Barry; Barry J Plant; Arjun Nair; Stephen Bicknell; Nicholas J Simmonds; Nicholas J Bell; Nadia T Shafi; Thomas Daniels; Susan Shelmerdine; Imogen Felton; Cedric Gunaratnam; Andrew M Jones; Alex R Horsley
Journal:  Chest       Date:  2014-07       Impact factor: 9.410
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  22 in total

1.  Nasospheroids permit measurements of CFTR-dependent fluid transport.

Authors:  Jennifer S Guimbellot; Justin M Leach; Imron G Chaudhry; Nancy L Quinney; Susan E Boyles; Michael Chua; Inmaculada Aban; Ilona Jaspers; Martina Gentzsch
Journal:  JCI Insight       Date:  2017-11-16

2.  Whole-blood transcriptomic responses to lumacaftor/ivacaftor therapy in cystic fibrosis.

Authors:  Benjamin T Kopp; James Fitch; Lisa Jaramillo; Chandra L Shrestha; Frank Robledo-Avila; Shuzhong Zhang; Sabrina Palacios; Fred Woodley; Don Hayes; Santiago Partida-Sanchez; Octavio Ramilo; Peter White; Asuncion Mejias
Journal:  J Cyst Fibros       Date:  2019-08-29       Impact factor: 5.482

3.  The COPD Pipeline XXVII.

Authors:  Nicholas Gross
Journal:  Chronic Obstr Pulm Dis       Date:  2015-04-21

4.  In vitro 3D culture lung model from expanded primary cystic fibrosis human airway cells.

Authors:  Rachael E Rayner; Jack Wellmerling; Wissam Osman; Sean Honesty; Maria Alfaro; Mark E Peeples; Estelle Cormet-Boyaka
Journal:  J Cyst Fibros       Date:  2020-06-18       Impact factor: 5.482

5.  Chronic β2AR stimulation limits CFTR activation in human airway epithelia.

Authors:  John J Brewington; Jessica Backstrom; Amanda Feldman; Elizabeth L Kramer; Jessica D Moncivaiz; Alicia J Ostmann; Xiaoting Zhu; L Jason Lu; John P Clancy
Journal:  JCI Insight       Date:  2018-02-22

Review 6.  Can Cystic Fibrosis Patients Finally Catch a Breath With Lumacaftor/Ivacaftor?

Authors:  E K Schneider; F Reyes-Ortega; J Li; T Velkov
Journal:  Clin Pharmacol Ther       Date:  2016-11-23       Impact factor: 6.875

7.  Predictive value of the modified Bhalla score for assessment of pulmonary exacerbations in adults with cystic fibrosis.

Authors:  Layla Diab-Cáceres; Rosa María Girón-Moreno; Elena García-Castillo; Maria Teresa Pastor-Sanz; Casilda Olveira; Marta María García-Clemente; Rosa Nieto-Royo; Concha Prados-Sánchez; Paloma Caballero-Sánchez; Maria Jose Olivera-Serrano; Alicia Padilla-Galo; Encarnacion Nava-Tomas; Amparo Esteban-Peris; Maria Fernández-Velilla; Maribel Torres; Rosa Mar Gómez-Punter; Julio Ancochea
Journal:  Eur Radiol       Date:  2020-08-01       Impact factor: 5.315

8.  Residual function of cystic fibrosis mutants predicts response to small molecule CFTR modulators.

Authors:  Sangwoo T Han; Andras Rab; Matthew J Pellicore; Emily F Davis; Allison F McCague; Taylor A Evans; Anya T Joynt; Zhongzhou Lu; Zhiwei Cai; Karen S Raraigh; Jeong S Hong; David N Sheppard; Eric J Sorscher; Garry R Cutting
Journal:  JCI Insight       Date:  2018-07-26

Review 9.  Targeted therapies to improve CFTR function in cystic fibrosis.

Authors:  Malcolm Brodlie; Iram J Haq; Katie Roberts; J Stuart Elborn
Journal:  Genome Med       Date:  2015-09-24       Impact factor: 11.117

10.  From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations.

Authors:  Gudio Veit; Radu G Avramescu; Annette N Chiang; Scott A Houck; Zhiwei Cai; Kathryn W Peters; Jeong S Hong; Harvey B Pollard; William B Guggino; William E Balch; William R Skach; Garry R Cutting; Raymond A Frizzell; David N Sheppard; Douglas M Cyr; Eric J Sorscher; Jeffrey L Brodsky; Gergely L Lukacs
Journal:  Mol Biol Cell       Date:  2016-02-01       Impact factor: 4.138
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