Modifying disease in cystic fibrosis
- 1 November 2013
- journal article
- review article
- Published by Ovid Technologies (Wolters Kluwer Health) in Current Opinion in Pulmonary Medicine
- Vol. 19 (6), 645-651
- https://doi.org/10.1097/mcp.0b013e328365ab5f
Abstract
Recent therapies directed at proximal targets within cystic fibrosis (CF) pathophysiology hold potential to modulate disease. This review highlights recent clinical trials and future therapies focused on these early steps of disease. Recent approval of a CF transmembrane conductance regulator (CFTR) protein modulator, ivacaftor (Kalydeco), has ignited a wave of investigations for other modulators directed at CFTR mutation classes. Gene replacement therapy continues to be pursued at a slower pace in early phase clinical trials. Airway surface liquid strategies such as dry-powder mannitol and alternate ion channel regulation are discussed as genotype-independent methods of early modulation. The breadth of therapies for early targets of CF holds considerable hope to modify the natural history of this disease. Ongoing focus to develop novel markers of early disease state is paramount. The progress of drug development requires concurrent attention on a spectrum of targets to achieve maximal impact.This publication has 53 references indexed in Scilit:
- Adenoviral Gene Transfer Corrects the Ion Transport Defect in the Sinus Epithelia of a Porcine CF ModelMolecular Therapy, 2013
- Gene therapy in cystic fibrosisTranslational Research, 2013
- Assessing the Disease-Liability of Mutations in CFTRCold Spring Harbor Perspectives in Medicine, 2012
- Expert opinion in biological therapy: update on developments in lung gene transferExpert Opinion on Biological Therapy, 2012
- Assessment of F/HN-Pseudotyped Lentivirus as a Clinically Relevant Vector for Lung Gene TherapyAmerican Journal of Respiratory and Critical Care Medicine, 2012
- Progress in cystic fibrosis and the CF Therapeutics Development NetworkThorax, 2012
- Progress in Gene and Cell Therapy for Cystic Fibrosis Lung DiseaseCurrent Pharmaceutical Design, 2012
- Amniotic Mesenchymal Stem Cells: A New Source for Hepatocyte-Like Cells and Induction of CFTR Expression by Coculture with Cystic Fibrosis Airway Epithelial CellsJournal of Biomedicine and Biotechnology, 2012
- Cystic FibrosisThe New England Journal of Medicine, 2005
- Identification of the Cystic Fibrosis Gene: Genetic AnalysisScience, 1989