The Evolution of Cystic Fibrosis Care

Chest
Jessica E Pittman, Thomas Ferkol

Abstract

Cystic fibrosis (CF) is the most common life-limiting inherited illness of whites. Most of the morbidity and mortality in CF stems from impaired mucociliary clearance leading to chronic, progressive airways obstruction and damage. Significant progress has been made in the care of patients with CF, with advances focused on improving mucociliary clearance, minimizing inflammatory damage, and managing infections; these advances include new antimicrobial therapies, mucolytic and osmotic agents, and antiinflammatory treatments. More recently, researchers have targeted disease-causing mutations using therapies to promote gene transcription and improve channel function, which has led to impressive physiologic changes in some patients. As we develop more advanced, allele-directed therapies for the management of CF, it will become increasingly important to understand the specific genetic and environmental interactions that cause the significant heterogeneity of lung disease seen in the CF population. This understanding of CF endotypes will allow for more targeted, personalized therapies for future patients. This article reviews the genetic and molecular basis of CF lung disease, the treatments currently available, and novel therapies th...Continue Reading

References

Sep 1, 1976·The Journal of Pediatrics·R C SternL W Matthews
Apr 30, 1992·The New England Journal of Medicine·E KeremH Levison
Feb 3, 1983·Nature·P M Quinton
May 1, 1983·The Journal of Clinical Investigation·M KnowlesR Boucher
Jan 1, 1980·Respiration; International Review of Thoracic Diseases·P H WellerD J Matthew
Mar 30, 1995·The New England Journal of Medicine·M W KonstanP B Davis
Jan 1, 1995·Annual Review of Genetics·J Zielenski, L C Tsui
Feb 2, 1999·The Journal of Clinical Investigation·J J Wine
May 9, 2001·The Journal of Pediatrics·G M NixonK Grimwood
Sep 20, 2001·The Journal of Pediatrics·M RosenfeldB Ramsey
Dec 18, 2001·The Journal of Pediatrics·J M QuanUNKNOWN Pulmozyme Early Intervention Trial Study Group
May 10, 2002·Pediatric Pulmonology·C SchaedelL Holmberg
Oct 1, 1964·The Journal of Pediatrics·L W MATTHEWSS SPECTOR
Oct 2, 2003·JAMA : the Journal of the American Medical Association·Lisa SaimanUNKNOWN Macrolide Study Group
Oct 14, 2003·American Journal of Respiratory and Critical Care Medicine·Ronald L GibsonBonnie W Ramsey
Dec 13, 2003·American Journal of Respiratory and Critical Care Medicine·Shawn D AaronFrancis Chan
Jan 14, 2004·American Journal of Respiratory and Critical Care Medicine·Christopher H GossJoel D Kaufman
Jan 15, 2004·American Journal of Respiratory Medicine : Drugs, Devices, and Other Interventions·Mary Jayne Kennedy
Feb 3, 2005·JAMA : the Journal of the American Medical Association·Zhanhai LiMark L Splaingard
Jul 19, 2005·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·N HøibyT Pressler
Oct 7, 2005·The New England Journal of Medicine·Mitchell L DrummUNKNOWN Gene Modifier Study Group
Oct 7, 2005·Pediatric Pulmonology·David S ArmstrongKeith Grimwood
Jan 20, 2006·The New England Journal of Medicine·Mark R ElkinsUNKNOWN National Hypertonic Saline in Cystic Fibrosis (NHSCF) Study Group
Jan 20, 2006·The New England Journal of Medicine·Scott H DonaldsonRichard C Boucher
Feb 24, 2006·The Journal of Pediatrics·Thomas FerkolCarlos E Milla
Feb 17, 2007·American Journal of Respiratory and Critical Care Medicine·Stephanie D DavisMargaret W Leigh
Apr 21, 2007·American Journal of Respiratory and Critical Care Medicine·Robin R DeterdingUNKNOWN Cystic Fibrosis Therapeutics Development Network and the Inspire 08-103 Working Group
Jul 27, 2007·Proceedings of the American Thoracic Society·Stephanie D DavisMargaret Rosenfeld
Feb 7, 2008·Chest·Arnon ElizurThomas W Ferkol
Feb 23, 2008·The Journal of Clinical Investigation·Frank J Accurso, Marci K Sontag
Mar 15, 2008·Chest·Anna JaquesBrett Charlton
Jul 22, 2008·The Journal of Pediatrics·Philip M FarrellUNKNOWN Cystic Fibrosis Foundation

❮ Previous
Next ❯

Citations

Mar 16, 2016·Molecular and Cellular Pediatrics·Speranza EspositoLuigi Maiuri
Jul 25, 2015·American Journal of Respiratory and Critical Care Medicine·Thomas Ferkol, Paul Quinton
Jul 10, 2016·The Journal of Allergy and Clinical Immunology. in Practice·Daniel L Hamilos
Jul 30, 2016·Pediatric Clinics of North America·Danielle M Goetz, Shipra Singh
May 19, 2018·The European Respiratory Journal·Marieke van HorckEdward Dompeling
Mar 19, 2019·Pediatric Pulmonology·Kevin W SouthernSarath Ranganathan
Mar 29, 2019·Science Translational Medicine·Thomas W Ferkol
Apr 21, 2020·BioEssays : News and Reviews in Molecular, Cellular and Developmental Biology·Renee F ConwayJason R Spence
Jul 15, 2017·Annals of the American Thoracic Society·Jessica E PittmanUNKNOWN Australian Respiratory Early Surveillance Team for Cystic Fibrosis
Sep 29, 2017·Nature Communications·Florence SonnevilleOlivier Tabary
Jun 21, 2018·Frontiers in Pharmacology·Anastasia TchoukaevPhilippe Le Rouzic
Apr 10, 2018·Work : a Journal of Prevention, Assessment, and Rehabilitation·Pablo S SaldanaMary Ellen Young
Oct 19, 2020·International Forum of Allergy & Rhinology·Brent A Senior
Jan 6, 2021·Journal of Medicinal Chemistry·Steven E Van der PlasMartin Andrews

❮ Previous
Next ❯

Related Concepts

Related Feeds

Anti-inflammatory Treatments

A drug or substance that reduces inflammation (redness, swelling, and pain) in the body. Anti-inflammatory agents block certain substances in the body that cause inflammation and swelling. Discover the latest research on anti-inflammatory treatments here

CREs: Gene & Cell Therapy

Gene and cell therapy advances have shown promising outcomes for several diseases. The role of cis-regulatory elements (CREs) is crucial in the design of gene therapy vectors. Here is the latest research on CREs in gene and cell therapy.