Studies in mice show promise for human treatment helping reverse symptoms of autism

Source: Xinhua| 2017-10-28 14:36:06|Editor: liuxin
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LOS ANGELES, Oct. 27 (Xinhua) -- U.S. researchers have found a drug treatment that may reverse symptoms of a rare genetic condition associated with autism.

A research team from the University of California, Davis and the Massachusetts Institute of Technology made the finding in a new animal model study.

Human chromosome 16p11.2 deletion syndrome, caused by the absence of about 27 genes on chromosome 16 at position p11.2, is present at an estimated rate of three in 10,000 in the general population. People who have this condition may have impaired communication and social skills, as well as delayed intellectual development, recurrent seizures, and an increased risk for obesity.

In the new study, published in the latest issue of the journal Neuropsychopharmacology, researchers used two independently derived lines of mutant mice, each missing a chromosomal region analogous to human 16p11.2.

"Our collaborative teams found that treatment with the drug R-baclofen improved scores on several learning and memory tasks, and on a standard assay of social behavior, in 16p11.2 mutant mice," Jacqueline Crawley, researcher at the University of California and co-senior author of the paper along with MIT's Mark Bear, said in a statement.

Normal and mutant mice at labs were tested after receiving the drug in their drinking water on three tasks: novel object recognition, object location memory and contextual recognition learning and memory. Mice of each genotype that received no R-baclofen in their drinking water were used as controls in the experiments.

Drug-treated mutant mice scored better after treatment on each cognitive task than the untreated mutant mice, according to the study, which is supported by the Simons Foundation.

"Remarkably, R-baclofen also increased some scores on a standard assay of mouse social behaviors -- male-female reciprocal social interactions -- in the 16p11.2 mutant mice," Crawley said.

The comparable study, conducted on mice at MIT, showed the same signs of improvement in mice that had been given R-baclofen. Another study is now underway to find out if the results can be reproduced and are viable for further study.

"If the preclinical mouse data prove consistently promising, our translational studies may encourage a new clinical trial with the human Arbaclofen compound in people with 16p11.2 deletion syndrome," said Crawley.