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Gene editing can curb autism symptoms

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Scientists have found that editing a gene in the brain can help decrease the repetitive behaviors – a symptom of autism spectrum disorders.

In the study, the team led by scientists at the University of Texas-San Antonio used nanoparticle carriers to inject a gene-editing enzyme called Cas9 into the striatum – a brain region associated with the formation of habits.

Enzymes are proteins that trigger biochemical reactions.

“The enzyme we used, Cas9, is like a pair of scissors. We were able to cut the genetic blueprint, DNA, at a location that causes the exaggerated repetitive behaviors,” said Hye Young Lee, Assistant Professor at the varsity.

In a mice model with fragile X syndrome – an inherited cause of autism spectrum disorders, the researchers targeted Cas9 at a molecule called mGluR5 that is excitatory – it increases communications between neurons.

The approach worked. The rodents’ digging behavior slowed by 30 percent and the leaping behavior was reduced by 70 percent, Lee said, in the paper described in the journal Nature Biomedical Engineering.

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