Gene therapy: the first decade

Gene therapy promises to revolutionize medicine by treating the causes of disease rather than the symptoms. We are nearing the end of the first decade of gene therapy, and this article summarizes the approaches taken, results achieved, lessons learned and important recent developments. The early results on the clinical efficacy of gene therapies were disappointing, largely because the available gene-transfer vectors proved to be inadequate. Recently, however, clinical benefit has been clearly demonstrated and great progress made in selecting and improving vectors. There is now every prospect that the second decade will see gene therapy live up to its enormous potential.