One Gene, One Drug Affect Autism Behaviors in Mouse Model

"Harvard researchers found that deleting one gene in certain brain cells causes autism behaviors in mice. They also discovered that treating the mice with rapamycin, an immunosuppressant drug, prevents the symptoms. Autism Speaks partially funded the study, published this month in the journal Nature.

The findings represent an important step in figuring out brain pathways that cause autism, explains the study’s senior author, Mustafa Sahin, Ph.D., associate professor of neurology at Harvard University. It also represents a very early step in determining whether rapamycin or drugs like it can help individuals with autism.

Past research has associated autism with certain brain cells in the cerebellum, a region involved in coordinating brain activity. These cells, called Purkinje cells, play an essential role in normal brain function. Studies using post-mortem tissue show that many individuals with autism have fewer of these cells than is normal...

Deleting the gene (either one or both copies) caused all three of autism’s core behaviors:

· Abnormal social interactions. The mice spent less time with each other and more with inanimate objects.

· Repetitive behaviors. The mice spent abnormal amounts of time pursuing one activity.

· Abnormal communication. They emitted abnormal levels of ultrasonic vocalizations."

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