Gene and cell therapy for cystic fibrosis

Since the discovery of the CF gene over a decade ago, several groups worldwide have explored the potential of gene therapy, the insertion of a normal copy of the gene into the respiratory epithelium. Both viral and synthetic gene transfer agents have been designed to this end, although problems with repeat application have occurred with the former have occurred. Here, we will briefly review success in clinical trials to date. Cell therapy, replacement of the CF cell with a healthy, normal cell, is at a much earlier stage of development, studies mainly being preclinical or observational. Some evidence of plasticity of, for example, bone marrow cells, has been demonstrated, although for the airway, levels of correction appear to be extremely low and a degree of damage seems to be a prerequisite. Whether this approach will be able to achieve clinical success remains to be seen.