Delivery of genes into the CF airway. Issue 10 (11th July 2014)

• Record Type: Journal Article
• Authors: Gill, Deborah R; Hyde, Stephen C
• Is Part Of: Thorax. Volume 69:Issue 10(2014)
• Page Start: 962
• Page End: 964
• Publication Date: 2014-07-11
• Subjects: Cystic Fibrosis; Chest -- Diseases -- Periodicals; Thorax; Chest -- Diseases; Periodicals; Periodicals; 617.54
• DOI: 10.1136/thoraxjnl-2014-205835
• Languages: English
• ISSNs: 0040-6376

Abstract

Abstract : Gene therapy was suggested as a potential treatment for cystic fibrosis (CF), even before the identification of the CFTR gene. Initial enthusiasm has been tempered as it became apparent that reintroduction of the CFTR gene into the cells of the lung is more difficult than anticipated. Here, we review the major gene delivery vectors evaluated clinically, and suggest that advances in either plasmid DNA design and/or hybrid lentivirus biology may finally facilitate lung gene transfer with efficiencies sufficient for CF gene therapy to offer clinical benefit.