Scientists may soon be able to treat autism with CRISPR gene editing

CRISPR-Cas9 gene editing is capable of some pretty impressive feats, from creating more efficient crops to malaria-resistant mosquitoes. Now researchers at the University of California, Berkeley and other institutes have discovered another potentially life-altering application: Editing out the genetic traits commonly associated with autism. While it’s so far been demonstrated on mice only, it raises the possibility of revolutionizing autism treatment for millions of people around the world.

“This study shows the first time demonstration of the rescue autistic symptoms using gene editing in autism mouse model,” Hye Young Lee, an assistant professor of cellular and integrative physiology at the University of Texas Health Science Center at San Antonio, told Digital Trends. “On the top of that, we used nonviral way to deliver to do it, which supports the potential therapeutic treatment for brain disorders.”

The work involved injecting the CRISPR complex into a specific brain region in mice, via special nanoparticles. This so-called “striatum” brain region is known to mediate the forming of habits, including the repetitive behaviors often seen with autism spectrum disorder (ASD.) The Cas9 disabled a specific receptor gene, which resulted in the dampening of exaggerated signaling between cells, thereby reducing repetitive behavior. In the case of mice, this meant a 30 percent decrease in compulsive digging and 70 percent drop in jumping, both of these behaviors associated with autistic behavior.

As another important development in the study, researchers discovered a way to ship the CRISPR particles long distances (in this case from Berkeley to San Antonio, Texas), as well as manufacturing them in a reproducible manner. This eliminates a key bottleneck affecting many projects involving nanotechnologies.

Could a similar treatment to this one day be used 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,” Lee said. In the future, the researchers think it might also be possible to inject these particles into the central nervous system by way of the spinal cord, rather than having to inject them directly into the brain.

A paper describing the work was recently published in the journal Nature Biomedical Engineering.