Gene therapy for cystic fibrosis

Abstract

After a decade of intensive research, effective gene therapy for cystic fibrosis (CF) remains an elusive goal. There have, however, been tremendous advances in our understanding of the basic processes of CF lung disease and in the development of gene therapy protocols, as reflected in the number and diversity of relevant patents issued. A selection of pertinent patents (with a bias towards those granted by the WIPO) is reviewed. Solutions to the key challenge of efficiently and safely delivering therapeutic genes to the respiratory epithelium are actively being sought. A new generation of vehicles that more effectively target cells while minimising immune responses is emerging. Vehicles and vectors that promote extended transgene persistence and expression are being developed to overcome the problems of short-term expression that characterised early clinical trials. There is ample cause for continued optimism that CF gene therapy will become a clinical reality through incremental improvements in all stages of the therapeutic process from formulation to expression.

• cystic fibrosis (CF)
• cystic fibrosis transmembrane conductance regulator (CFTR)
• expression vectors
• gene therapy
• lipofection
• lung epithelium
• viral vehicles