Liposomal DNA vectors for cystic fibrosis gene therapy. Current applications, limitations, and future directions

Abstract

The etiology of cystic fibrosis (CF), current therapies, and recent experimental molecular based therapies are briefly described including the use of recombinant human deoxyribonuclease I (rhDNase), nonsteroidal antiinflammatory drugs (ibuprofen), the sodium channel blocking diuretic amiloride, and uridine triphosphate (UTP), a chloride secretagogue. The original approach to CF gene therapy using recombinant replication defective adenovirus and its potential benefits as well as shortcomings are also briefly discussed. More recently, the use of nonviral cationic liposome plasmid complexes to deliver cystic fibrosis transmembrane conductance regulator (CFTR) cDNA has been successfully demonstr..