Scientists in South Korea have identified a potential new drug target, SLC6A20, that may help restore disrupted neural pathways associated with autism, showing promising results in mice and human brain models.
Scientists have identified a possible new way to restore a key brain-signalling pathway that is disrupted in some forms of autism, in work that showed effects in both genetically modified mice and laboratory-grown human brain organoids. The study, led by Eunjoon Kim at the Institute for Basic Science in South Korea and published in Nature Communications on 29 May 2026, points to the glycine transporter SLC6A20 as a potential drug target.
The research centres on NMDA receptors, which help brain cells communicate and are important for learning, memory and the flexibility of neural connections. When these receptors do not work properly, signalling can weaken in ways that have been linked to autism, schizophrenia and intellectual disability. But autism is highly varied, so any treatment aimed at this pathway would likely apply only to a subset of people rather than to all autistic people.
To test the idea, the researchers used antisense oligonucleotides, short strands of genetic material designed to reduce production of the SLC6A20 transporter. In mice carrying SHANK2 or SHANK3 mutations, the treatment restored NMDA receptor activity and improved some behaviours linked to social interaction, communication and repetitive actions. The benefit was seen in adult mice as well, which suggests that at least some of these signalling problems may remain modifiable later in life.
The team also examined human cortical organoids, miniature lab-grown models of the brain’s outer layer, and found that the same approach brought NMDA receptor function closer to normal in cells carrying SHANK2 or SHANK3 changes. According to the study, a single treatment in mice remained effective for at least eight weeks without obvious harmful effects during the period examined. That is encouraging, but it is still early-stage research.
Experts will still want to see more work on safety, dosing and delivery before any human trial. The findings do not amount to a cure, but they do offer a more precise strategy for targeting one biological pathway that may be disrupted in some autism-related conditions. If that line of research holds up, it could help move autism treatment towards approaches tailored to specific genetic and molecular profiles rather than one broad diagnosis.
Disclaimer: This content is for informational purposes only and is not intended to be a substitute for professional medical judgment, advice, diagnosis, or treatment.
