MIT scientists reverse autism, retardation in mice

Researchers from the Picower Institute for Learning and Memory at Massachusetts Institute of Technology have reversed mild retardation and severe autism in mice by inhibiting the enzyme called p21-activated kinase (PAK). Their study focused on Fragile X Syndrome (FXS)--a leading cause of retardation and autism--which is linked to a mutated X chromosome gene. The PAK enzyme controls the connections between neurons and the brain.

"Strikingly, PAK inhibition also restored electrical communication between neurons in the brains of the FXS mice, correcting their behavioral abnormalities in the process," co-author Susumu Tonegawa, 1987 Nobel laureate and Picower Professor of Biology and Neuroscience, said in a prepared statement. "Notably, due to an elegant genetic manipulation of method employed by the Picower Institute researchers, PAK inhibition in the FXS mice did not take place until a few weeks after appearance of disease symptoms. This implies that future treatment may still be effective even after symptoms are already pronounced."

- read the report from Forbes

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