Gene therapy for inherited lung disorders: An insight into pulmonary defence

Abstract

This review summarizes the latest developments in viral and nonviral gene delivery systems to the lung, and the problems that have to be overcome. Gene delivery has the potential to offer effective treatment to patients with life-threatening lung diseases such as cystic fibrosis and α1-antitrypsin deficiency, and could modify gene-environment relationships in asthma and other respiratory diseases. Phase I clinical trials conducted in the early 1990s showed that in principle gene transfer to the lung was safe. Although the preliminary results gave encouraging laboratory data, gene expression from viral or nonviral gene delivery systems was too inefficient or transient to offer clinical benefi..