Abstract
Cystic fibrosis is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that lead to abnormalities in transepithelial ion transport in the airways of affected patients. Lung disease is the major contributor to morbidity and mortality in patients with cystic fibrosis but recommended therapeutic interventions so far have focussed on symptom control rather than treatment of the underlying causes of the disease. New therapies that are currently in pre-clinical and clinical testing include CFTR pharmacotherapy, drugs targeting other ion channels, and hydrators of the cystic fibrosis airways. The current status of these and other new developments in the treatment of cystic fibrosis are reviewed
Keywords: Cystic fibrosis, lung disease, therapy, mutations, transmembrane conductance regulator (CFTR) gene, airways, mucus retention, bacterial infection, neutrophilic inflammation, osmotic gradient
Current Pharmaceutical Design
Title: New Therapies in Cystic Fibrosis
Volume: 18 Issue: 5
Author(s): Felix Ratjen and Hartmut Grasemann
Affiliation:
Keywords: Cystic fibrosis, lung disease, therapy, mutations, transmembrane conductance regulator (CFTR) gene, airways, mucus retention, bacterial infection, neutrophilic inflammation, osmotic gradient
Abstract: Cystic fibrosis is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that lead to abnormalities in transepithelial ion transport in the airways of affected patients. Lung disease is the major contributor to morbidity and mortality in patients with cystic fibrosis but recommended therapeutic interventions so far have focussed on symptom control rather than treatment of the underlying causes of the disease. New therapies that are currently in pre-clinical and clinical testing include CFTR pharmacotherapy, drugs targeting other ion channels, and hydrators of the cystic fibrosis airways. The current status of these and other new developments in the treatment of cystic fibrosis are reviewed
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Cite this article as:
Ratjen Felix and Grasemann Hartmut, New Therapies in Cystic Fibrosis, Current Pharmaceutical Design 2012; 18 (5) . https://dx.doi.org/10.2174/138161212799315984
DOI https://dx.doi.org/10.2174/138161212799315984 |
Print ISSN 1381-6128 |
Publisher Name Bentham Science Publisher |
Online ISSN 1873-4286 |
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