Gene Therapy – The Brave New World

Although the theoretical basis for gene therapy has been available for some years, it has been a struggle to turn theory successfully into practice. The initial concepts of using viruses to transport genes into patient cells have met with repeated difficulties, so that new strategies are needed. We have developed methods for integrating genes into the chromosomes of human cells at a limited number of positions and with resulting high gene expression. This novel technology, based on the use of phage integrase enzymes, appears to have the makings of a new platform technology that can make gene therapy work. The lab has applied the technology to numerous disease targets with success and is proceeding along a course, which will be described, to develop the technology further and pursue clinical applications.