Researchers are hailing a breakthrough after they used cutting-edge gene editing to significantly reduce repetitive behavior associated with the disorder.
Scientists injected gold nanoparticles covered in a “forest” of DNA chains to alter the the genetic code of mouse models with a form of autism called fragile X syndrome (FXS).
The technique, CRISPR-Cas9 gene editing, resulted in a 30 percent drop in repetitive digging, and a 70 reduction in leaping, both indicative of autistic behavior.
Because the editing DNA was introduced attached to gold particles, the team were able to control how much of the crucial Cas9 protein was delivered, in contrast to previous failed attempts using viruses.
“There are no treatments or cures for autism yet, and many of the clinical trials of small-molecule treatments targeting proteins that cause autism have failed,” said study leader Hye Young Lee, an assistant professor of cellular and integrative physiology at the University of Texas Health Science Center at San Antonio.
“This is the first case where we were able to edit a causal gene for autism in the brain and show rescue of the behavioral symptoms.”
Autism spectrum disorder is a complex developmental disorder, with a strong genetic component, which manifests during childhood.
It is characterized by deficits in the domain of social interaction and communication, stereotyped behavior, delayed speech and language and can also be associated with intellectual disability.
There are around 700,000 people on the autism spectrum in the UK – more than one in 100 – and when including families, it is a part of daily life for 2.8 million people.
The results are published in the journal Nature Biomedical Engineering.
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