Experimental therapy may reverse autism spectrum–like disorder in mice

A new study suggests that an experimental drug treatment may reverse symptoms of Rett syndrome, an autism spectrum disorder that causes severe neurodevelopmental problems, Cold Spring Harbor Laboratory announced. The genetic disorder affects about one in 10,000 people in the United States, mostly girls, because boys tend not to survive past infancy with the illness. Now, in The Journal of Clinical Investigation, scientists report that the drugs altered signaling pathways in cells associated with the gene MECP2—known to malfunction in the illness—in mice. As a result, male mice lived longer, and females experienced less behavioral symptoms, which in humans can include losing acquired social skills, an impaired gait, and interrupted brain development. The findings offer hope that Rett syndrome’s symptoms may one day be treated and reversed rather than simply managed, the scientists say.

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