Single-dose lentiviral gene transfer for lifetime airway gene expression.
Alice G Stocker, Karlea L Kremer, Rachel Koldej, Darren S Miller, Donald S Anson, David W Parsons
The Journal of Gene Medicine | Published : 2009
BACKGROUND: Cystic fibrosis (CF) is caused by a defect in cystic fibrosis transmembrane conductance regulator (CFTR) activity, often resulting in an incurable airway disease. Gene therapy into the conducting airway epithelium is a potential cure for CF; however, most gene vectors do not result in long-lived expression, and require re-dosing. Perversely, intrinsic host immune responses can then block renewed gene transfer. METHODS: To investigate whether persistent gene expression could be achieved after a single dosing event, thus avoiding the issue of blocking host responses, we used a gene transfer protocol that combined an airway pretreatment using lysophosphatidylcholine with a human imm..View full abstract