Overview
- An Australian-led team reporting in Nature Communications identified chronic overactivity at excitatory synapses as a key driver of cognitive decline in Sanfilippo syndrome.
- Using lab-grown cortical neurons made from patient stem cells and fine-grained electrical recordings, the researchers saw normal early development shift into bursts of tightly synchronized firing as networks matured.
- The affected neurons showed heightened sensitivity to mild nutrient stress, which intensified the overactive signaling and suggests common illness could speed neurological decline.
- In cell experiments, certain existing medications restored the balance between excitatory and inhibitory signals, pointing to a practical treatment target at the synapse.
- Researchers are now screening approved drugs for possible repurposing with this preclinical model, and they have not yet reported animal studies or clinical trials.