Disease genes: flattery and deception

Completion of the human genome project raises the possibility of genetically based treatments for a multitude of human diseases. As yet only a handful of patients have benefited clinically from this approach. Why gene transfer is such a complex issue is discussed in this article. Theoretically, the easiest diseases to treat are single gene recessive diseases, where, presumably, gene delivery to somatic cells is all that is required. Two prime candidates for gene therapy are severe combined immunodeficiency disease (SCID) and cystic fibrosis (CF). Attempts to treat both of these diseases by gene therapy commenced in the late 1980s. Some clinical benefit has been recorded with SCID, but none, as yet, has been recorded with CF.