Gene therapy for cystic fibrosis: an example for lung gene therapy

Abstract

Gene therapy is currently being evaluated for a wide range of acute and chronic lung diseases. The requirement of gene transfer into the individual cell types of the complex lung structure will very much depend on the target disease. Over the last decade, the gene therapy community has recognized that there is not even one vector that is good for all applications, but that the gene transfer agent has to be carefully chosen. Gene therapy is particularly attractive for diseases that currently do not have satisfactory treatment options and probably easier for monogenic disorders than for complex diseases. Cystic fibrosis (CF) fulfills these criteria and is therefore a good candidate for gene therapy-based treatment. This review will focus on CF as an example for lung gene therapy and discuss the progress made in this field over the last couple of years.

Figure 1

Schematic presentation of the lung showing conducting airways (blue) and alveolar gas exchange region (pink).

Figure 2

Ciliated airway epithelial cells partially covered in mucus (courtesy of Professor Peter Jeffery, Imperial College).

Figure 3

Schematic presentation of the pulmonary and bronchial circulation transporting gene transfer agents to the airway epithelium after intravenous injection (courtesy of Steve Smith, Department of Gene Therapy, Imperial College London).