Gene therapy: Understanding the basics

The dawning of the new millennium has brought with it heretofore unimaginable advances in our understanding of what it means to be human. Building on the advances of the past and our understanding of normal physiology and disease mechanisms, we can add information gleaned from the initial phase of the Human Genome Project. Much remains to be learned. If we continue to invest effort into uncovering its vast potential, gene therapy will become a powerful weapon against active disease and advance the quest for true preventive medicine. However, in addition to designing more efficient, less immunogenic, and easily targeted vectors, we must create ways to regulate expression of the transgene. Perhaps some of these solutions will come through the use of stem cells as the ultimate vector.