Formulation of Synthetic Vectors for Cystic Fibrosis Gene Therapy

Following the identification and subsequent cloning of the gene that is defective in patients with cystic fibrosis (CF), there has been a substantial effort to evaluate gene therapy as a modality for treating this disease. As the primary cause of mortality in CF is respiratory dysfunction, the lung has been the major focus for genetic therapy intervention. Although this organ is relatively accessible for gene transduction-for example, by use of aerosols-it has become evident that there are several barriers to efficient transduction of the airway epithelia. These barriers are often further exacerbated in a diseased CF lung.