Scientists from the Institute for Basic Science reduced the activity of the glycine transporter SLC6A20 and thereby restored the function of NMDA receptors—critical for communication between neurons.
In mouse models of autism with SHANK2 and SHANK3 mutations, social behavior and communication improved, and repetitive actions decreased.
The effect lasted for at least 8 weeks and was evident even in adult animals. A similar result was obtained in human brain organoids.
These are currently preclinical data. However, the approach is interesting because it does not attempt to "turn on" the gene anew, but gently corrects existing signaling pathways. In the long term, it may prove useful for other conditions associated with weakened NMDA receptor function.
Sources:
- Institute for Basic Science. "New autism therapy shows surprising benefits even in adult mice." ScienceDaily (August 7, 2026).
- Roh J.D. et al. "Glycine-modulating Slc6a20a-ASO restores NMDA receptor function in SHANK2 and SHANK3-mutant mice and cortical organoids." Nature Communications (2026). DOI: 10.1038/s41467-026-73881-9.
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