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ASD jab: Chinese scientists reach milestone in revolutionary gene therapy for autism

  • A genome editing system developed by Chinese scientists successfully modified a mutated gene linked to autism when tested on mice
  • It is the first effective treatment of mice with ASD-related mutations using base editing in the brain, according to the researchers

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Autism spectrum disorder can affect the ability to interact and communicate, as well as cause repetitive behaviours and intense interests. Photo: Shutterstock
Chinese scientists have reached a significant milestone in creating the first injection that can undo the signs of autism through genetic base editing within the brain.

The treatment, developed by researchers in Shanghai, showed positive results when tested on mice.

The team created a genome editing system, which successfully modified the DNA of mice that had been given a mutation found in some patients with autism spectrum disorder (ASD).
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Mice given the injection containing the editing system registered a decrease in ASD-associated behaviour.

In a paper on their research, published in the journal Nature Neuroscience on November 27, the scientists said the potential treatment method could not just be used for patients with ASD, but also other genetic neurodevelopmental disorders.

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ASD affects around 1 per cent of the global population. One in every 36 children in the United States is diagnosed with the disorder, according to the US Centres for Disease Control and Prevention (CDC).
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