Researchers have identified a possible new way to address autism spectrum disorder by targeting a glycine transporter called SLC6A20, according to ScienceDaily.
In the study, the team found that lowering the activity of this transporter helped restore NMDA receptor function. Those receptors are important for communication between brain cells and play a key role in learning, memory, and other cognitive processes.
The researchers tested the approach in mouse models with SHANK2 and SHANK3 mutations, which are linked to autism risk and some other neurodevelopmental disorders. Treatment with antisense oligonucleotides improved social interaction, social communication, and repetitive behaviors, and the benefits were seen in adult mice.
The team also tested the strategy in human cortical organoids made with CRISPR gene editing and found that an ASO aimed at the human SLC6A20 gene brought NMDA receptor activity close to normal. According to the researchers, one dose remained effective for at least eight weeks in mice and did not show detectable adverse effects during that time.
While the study focused on autism spectrum disorder, the researchers say the findings could also be relevant to other conditions linked to reduced NMDA receptor activity, including schizophrenia and some forms of intellectual disability.
