A team of researchers at the University of California, Berkeley and other institutes have found that CRISPR gene editing can be used to reduce the symptoms of autism in mice. The milestone achievement is expected to emerge as a breakthrough in the treatment of autism among humans in the future.
Modern medical science has not found any treatment or cure autism yet and several clinical trials of small-molecule treatments targeting proteins that cause autism have failed. This new study report was published in the journal Nature Biomedical Engineering.
The findings have suggested that editing out the genetic traits, commonly associated with autism using CRISPR-Cas9 gene editing, will drastically reduce the symptoms of this disorder.
In the study, Hye Young Lee, an assistant professor of cellular and integrative physiology at the University of Texas Health Science Center said that it was the first case "where we were able to edit a causal gene for autism in the brain and show rescue of the behavioural symptoms."
During the study, researchers injected the CRISPR complex into a specific region in the mice brain via nanoparticles. These CRISPR complex were injected into the striatum region of the brain that is primarily responsible for moulding habits including the repetitive behaviours often seen in people with autism. The injected CRISPR complex apparently disabled a specific receptor gene which plays a crucial role to trigger repetitive actions.
Hye Young Lee revealed that tests on bigger animals should be done before implementing this treatment method in humans.
"Not right away, but it can be used for human treatment once we make sure it is safe to use, and once it is tested in bigger animals than mice," said Lee, digitaltrends.com reported.
Experts in medical science believe that it might be possible to inject CRISPR particles into the central nervous system of humans via the spinal cord, rather than directly injecting it to the brain.