A new gene transfer vector for the lung

Abstract

Several viruses have been exploited as gene transfer vectors, including retrovirus, adeno-virus and adeno-associated virus. Clinical trials assessing these vectors have shown that they can all mediate gene transfer but the efficiency of transfer, particularly of certain cell types, has been disappointing. This has been especially true of studies attempting gene transfer to the respiratory epithelium, the major target for gene therapy of cystic fibrosis. Any vector system targeting the respiratory epithelium must first overcome a mucus layer and mucocillary clearance, and must be able to utilize receptors on the apical surface of nondividing, well-differentiated cells. Although much work being conducted is attempting to improve the existing vector systems, one group has described the in vivo use of a new vector system that exploits a virus that has natural mechanisms that might overcome at least some of these barriers to gene transfer of the respiratory epithelium.