The rise, fall and resurgence of gene therapy

Twenty years ago, scientists and investors were giddy over the promise of gene therapy to cure a host of deadly diseases. But the tragic death of a young man from an experimental treatment in 1999 nearly ended a scientist’s career and the entire field. Now, according to an article in Chemical & Engineering News (C&EN), the weekly newsmagazine of the American Chemical Society, both the scientist and gene therapy are experiencing a resurgence. Conceptually, gene therapy is simple: Because many diseases are caused by defective genes, supplying healthy copies of those genes should provide a cure. However, the challenge lies in delivering the therapeutic genes to the appropriate cells in the body. In the 1990s, many researchers, including James Wilson at the University of Pennsylvania, were studying hollowed-out adenoviruses to package the curative genes and get them inside cells. Wilson led a clinical study that examined the use of adenovirus gene therapy to cure a rare liver disorder.