New research pinpoints a safe way to reverse a core brain signaling problem in autism spectrum disorder (ASD) — by silencing a single gene in cognition-related brain regions.
The Research
Led by Director Kim Eunjoon at the Institute of Basic Science in South Korea, the team focused on a glycine transporter called SLC6A20. The NMDA receptor (NMDAR), critical for learning and memory, needs both glutamate and glycine to work properly. Previous attempts to boost glycine targeted GlyT1, a transporter abundant in the brainstem, causing dangerous respiratory and motor side effects.
The researchers instead used antisense oligonucleotides (ASOs) to suppress SLC6A20 in adult mice with mutations in the autism-risk genes SHANK2 and SHANK3. The ASO treatment restored NMDAR activity, normalized abnormal phosphorylation patterns in synaptic proteins, and reversed behavioral deficits — including poor social interaction, communication impairments, and repetitive behaviors — without brainstem side effects. A single dose maintained benefits for at least eight weeks.
To confirm human relevance, the team created human cortical organoids with SHANK mutations using CRISPR and applied a human-targeted SLC6A20-ASO, which also restored NMDAR function to near-normal levels.
Why It Matters
This finding is a major shift in precision neuroscience. Instead of blasting the whole brain with a risky drug, researchers can target a transporter that’s concentrated in the cortex and hippocampus — brain areas responsible for thinking, memory, and social behavior. This could eventually lead to safer therapies for autism, schizophrenia, and other conditions linked to NMDAR hypofunction.
What You Can Do
While this therapy is still experimental, you can support your own NMDA receptor health with lifestyle choices: regular aerobic exercise, adequate sleep, and omega-3 fatty acids (from fish or supplements) are all linked to better glutamate signaling. Stay curious about your own cognition — brain training and cognitive assessments can help you track your mental sharpness over time.
Source: Neuroscience News
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