Massachusetts Institute of Technology researchers have found that suppressing a particular enzyme has reversed some symptoms of autism and mental retardation in mice.
An enzyme is a protein that triggers a chemical reaction, and this one, called p21-activated kinase (PAK), affects neural connections in the brain. Suppressing it can counteract the effects of Fragile X Syndrome (FXS), the leading cause of retardation and a genetic autism, MIT said Sunday.
"Our study suggests that inhibiting a certain enzyme in the brain could be an effective therapy for countering the debilitating symptoms of FXS in children, and possibly in autistic kids as well," the study's co-author Mansuo L. Hayashi, a former MIT postdoctoral fellow now working at Merck Research Laboratories, said in a statement. Several chemical compounds today already are known to inhibit PAK's activity.
The Proceedings of the National Academy of Sciences is reporting the research this week.
The experiment involved mice genetically altered to suffer from FXS. Suppressing the enzyme's activity restored the structure of neurons and their electrical communication abilities, MIT said. The suppression also corrected behavioral abnormalities.