"Gene scissors" may reduce symptoms of autism

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U.S. researchers used gold nanoparticles to carry “genetic scissors” to modify an autism-related gene in the brains of experimental rats, which successfully reduced the stereotypic repetitiveness of experimental mice. Related papers were published in the British Journal of Natural and Biomedical Engineering.

Gene Scissors refers to the CRISPR-Cas9 technology, which can cut or replace gene fragments just like editing articles on a computer. A press communique issued by the University of California, Berkeley, said that the new study that the school’s staff participated in was the first successful use of genetic scissors in the editing of genes in the brain.

Repeating some meaningless actions or weird behavior for a long time is one of the typical symptoms of autism. Rats with fragile X syndrome repeat dug and jump and can be used as animal models to study autism in humans. Their repetitive behavior is related to hyperactivity of brain metabotropic glutamate receptor 5 (mGluR5) genes.

The researchers attached the "scissor" protein Cas9 and RNAs that localize to the surface of the gold nanoparticles, encapsulated with polymers, and delivered intracranially into the striatum of the brain of rats to modify the mGluR5 gene. The results showed that the mGluR5 protein in the striatum was reduced by about half, the repetitive excavation behavior of the experimental mice was reduced by 30%, and the repeat jumping behavior was reduced by 70%.

The causes of autism are very complicated. The Fragile X syndrome is only one of them, and effective treatments for experimental mice may not be effective for humans. Therefore, this result cannot be applied to the treatment of autism. However, it shows the potential of genetic scissors and gold nanoparticle carriers, and will advance research in related fields to find treatments for autism and many other brain-related diseases. (According to Xinhua News Agency)


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