Cationic Lipid-Mediated Gene Delivery to the Airways

Cationic lipid-mediated delivery of therapeutic genes to the airways represents an attractive modality for treatment of a variety of inherited and acquired pulmonary diseases. This chapter discusses the cationic lipids for cystic fibrosis (CF) gene therapy. Because cationic lipids present a different safety profile than viral vectors, they are currently under active investigation for the treatment of several human indications including CF. While the results of recent clinical studies in CF subjects are encouraging, it is clear that there are still many hurdles to overcome and significant improvements to be made before cationic lipid-mediated gene transfer can be regarded as a viable therapy for CF. Although these tasks are challenging, it is likely that as the basic understanding of the processes governing cationic lipid-mediated gene delivery continues to improve, more efficacious vector systems that are compatible with use in the treatment of these chronic diseases will emerge. Attaining an effective treatment for CF would be more attainable when such vectors are identified.