Gene therapy for a severe inherited blindness, which produced dramatic improvements last year in 12 children and young adults who received the treatment in a clinical trial, has cleared another hurdle. The same research team that conducted the human trial now reports that a study in animals has shown that a second injection of genes into the opposite, previously untreated eye is safe and effective, with no ...

In his own words, MIT neuroscientist Mark Bear admits he did not “wake up one day and say, ‘Hey, I’m going to cure autism.’” But, after decades of painstaking basic research on how the brain rewires itself in response to external cues, Bear has discovered a way to reverse the symptoms of Fragile X Syndrome, a disorder that can cause autism, mental retardation, and epilepsy. “It was a classic payoff of basic ...