New Autism Treatment Works in Adult Mice, Human Brain Cells

Scientists just discovered a way to improve autism symptoms in adult mice by fixing how their brain cells communicate, and the approach also worked in human brain tissue.

Researchers at South Korea's Institute for Basic Science targeted a specific protein called SLC6A20 that controls glycine levels in the brain. Glycine is a chemical that helps brain cells send signals to each other through receptors called NMDA receptors, which are crucial for learning, memory, and social behavior.

People with autism, schizophrenia, and some other brain conditions often have reduced NMDA receptor activity. For decades, scientists tried boosting these receptors by blocking a different protein called GlyT1, but that caused breathing problems and limited results because GlyT1 exists throughout the brainstem.

Director Eunjoon Kim and his team chose a smarter target. SLC6A20 appears mainly in brain regions involved in thinking and memory, like the cortex and hippocampus, making it safer to block without affecting essential functions like breathing.

The researchers tested their treatment in mice with mutations in SHANK2 and SHANK3 genes, which are major autism risk factors in humans. Using a therapy called antisense oligonucleotides, they reduced SLC6A20 activity and watched what happened.

The results surprised even the scientists. Treated mice showed better social interaction, improved communication, and fewer repetitive behaviors. The treatment worked in adult mice, not just young ones, challenging the long-held belief that brain development disorders can only be addressed early in life.

The Bright Side

The therapy didn't just add or remove proteins in the brain. Instead, it fixed how existing proteins were functioning by correcting abnormal patterns in their chemical switches, restoring natural brain signaling rather than forcing artificial changes.

The team then created miniature human brains in the lab using CRISPR technology, giving them the same SHANK2 and SHANK3 mutations found in people with autism. When they applied a human version of the treatment, the tiny brain organoids showed restored NMDA receptor activity similar to healthy tissue.

One dose lasted at least eight weeks in mice with no detected side effects. The treatment remained effective throughout that period, suggesting it could offer lasting benefits without frequent administration.

While this research focused on autism, reduced NMDA receptor activity also affects people with schizophrenia and certain intellectual disabilities. That means this approach could eventually help a much broader group of people.

The findings offer something rare in brain science: hope that the window for treatment extends far beyond childhood.