693. Airway Pre-Treatment with a Novel Surfactant Compound Improves Standard Enhancement of Lentiviral Gene Transfer In Vivo

To achieve an effective and efficient gene therapy for cystic fibrosis lung disease, we have developed in vivo methods using airway pre-treatment with a standard lysophosphatidylcholine (LPC) mixture, prior to delivery of lentiviral vectors to increase gene expression. High level, long-lasting gene expression is produced in intact nasal airways of normal and cystic fibrosis mice. In this study we tested if novel related surfactants we have identified can increase the effectiveness of gene expression in a short-term gene transfer setting.