Limiting Damage: Fragile X symptoms modulated in mice
By cutting in half the activity of a gene, scientists corrected many symptoms of a genetic defect in mice analogous to fragile X syndrome, a leading cause of inherited mental retardation in people.
The research suggests a new target for drug therapy for the condition, which is currently untreatable. However, it remains uncertain whether such drugs would benefit adults who have the disease. And it would take years of clinical trials to show whether drugs could check the disease’s progress in infants with the genetic defect.