Study Reverses Structural Autism Deficits in Brain Cells

Researchers from Shimane University, Kobe University, and Hyogo Medical University successfully reversed structural brain abnormalities and behavioral deficits linked to autism spectrum disorder (ASD) in a mouse model by restoring shortened axon initial segments (AIS) in neurons. Using a chemogenetic technique called DREADD, they normalized AIS length in the prefrontal cortex-to-dorsal raphe nucleus pathway, resulting in improved sociability and reduced repetitive behaviors, suggesting potential for new therapeutic approaches in humans.
A team of researchers led by Professor Masashi Fujitani and Assistant Professor Yoshinori Otani from Shimane University, in collaboration with Kobe University and Hyogo Medical University, has achieved a breakthrough in autism spectrum disorder (ASD) research. The study, published in *Cell Death & Disease* on May 19, 2026, identified structural abnormalities in the axon initial segment (AIS) of neurons in a mouse model carrying genetic duplications linked to human ASD (15q dup mice). The AIS, critical for generating electrical signals, was abnormally shortened in the neural circuit connecting the prefrontal cortex to the dorsal raphe nucleus, impairing neuronal excitability and contributing to ASD-like behaviors. To test reversibility, the researchers used a chemogenetic technique called DREADD to selectively activate the damaged neural pathway. This intervention successfully restored the shortened AIS to normal length, correcting the structural defect. Alongside the physical repair, the mice exhibited significant behavioral improvements, including increased sociability and reduced repetitive behaviors, mirroring key ASD symptoms. The findings demonstrate that neural plasticity deficits in ASD are not permanent and can be reversed through targeted interventions. This research, published in a peer-reviewed journal, establishes a foundation for developing new circuit-based therapies for ASD in humans. The study highlights the potential of chemogenetic approaches in addressing neurological disorders by restoring impaired brain function.
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