Seeing Is Believing
5/2/2008 United Kingdom staff Economist.com The prospects for using genes as a therapy may be improving For around 40 years scientists have understood how genes work. They have known the structure of genes, how they replicate, how they are controlled and expressed and, crucially, how to manipulate them. Such knowledge has been the basis of a genetic revolution that offers the power to rewrite the material from which all living organisms are made. There has been great progress in realising some of this promise, in the form of genetically modified organisms. But ways to correct the genetic mistakes that cause many human diseases have been slower to arrive. Gene therapy has been plagued with problems—naivety, false promises, over-optimism and fatalities. Although thousands of patients have received gene therapy for a variety of conditions, only a few have shown any clinical benefit. Could that be about to change? There was news this week of a successful attempt to correct a faulty gene that leads to blindness. An international team of scientists, led by a group at the University of Pennsylvania, used a genetically engineered virus to introduce the correct version of a gene called RPE65 into six people suffering from a retinal disease known as Leber's congenital amaurosis. In four patients vision improved. Earlier work with the same technique on dogs suffering from a naturally occurring form of blindness has also been successful. Katherine High, of the Howard Hughes Medical Institute in Maryland, and one of the directors of the study, [...]