Gene Therapy, Successful Against Parkinson's, Continues on the Road to Redemption

Back in the 1980s, gene therapy was one of science's greatest hopes and hypes, and researchers predicted the technique would be used to cure a huge range of illnesses. During the 90s, many early gene therapy trials were effective or downright dangerous, some causing cancer or even death. But more recently, scientists who stuck with gene therapy have started to see positive results, with promising treatments for malformed hemoglobin, color blindness, and depression. (See the DISCOVER magazine feature "The Second Coming of Gene Therapy" for more.) Now, researchers have announced that they've successfully treated the symptoms of Parkinson's disease in a small group of people---a far cry from a cure, but still a step in the right direction. I Once Was Blind but Now I See The theory behind gene therapy is simple: A healthy gene hitches a ride into the patient's genome on a virus, replacing the genes responsible for some genetic disease or disorder. Actually doing this is more difficult, because humans have a little thing called an immune system that's remarkably efficient at finding and destroying foreign bodies. After the first U.S. death from gene therapy in 1999, and leukemia cases in France the same year, many started to think that gene therapy was more of a problem than an answer.