Lab-grown mini-brains reveal insights into severe autism and potential treatment options

Researchers at Scripps Research Institute have created lab-grown "mini-brains" using stem cells from patients with a rare genetic form of autism. This study focuses on the MEF2C gene mutation, which disrupts brain development and is linked to severe autism symptoms. The team tested an experimental drug, NitroSynapsin, on these mini-brains. The drug helped restore balance in brain cell activity, reducing excessive neuron firing and correcting imbalances in excitatory and inhibitory signals. This suggests potential for future treatments targeting MEF2C-related autism. While the findings are promising, the mini-brains cannot fully replicate human brain complexity. Further research and human trials are needed to confirm the results and explore the drug's effectiveness in treating autism.