TH 17 cell/IL-17a Pathway in Autism Spectrum Disorder


This technology is a new therapeutic and theranostic target for ASD.

Problem Addressed

Autism spectrum disorder (ASD) is a common, incurable developmental disorder that affects behavior and communication. ASD is incurable, and treatments are currently limited to symptom management through intensive behavioral and educational programs. There is therefore an enormous need for new ASD treatments.


These inventors discovered a unique neuron dysfunction that can be therapeutically targeted to reverse ASD-like behavioral symptoms in a mouse model. The ASD mouse model displays characteristic patches of disorganized cortex neurons that strongly correlate with a variety of behavioral defects. The neurons in these disorganized patches are hyperactivated, which causes the ASD-like behavioral defects of the mouse model. Notably, the inventors demonstrate that acutely reducing the neural activity in the disorganized area reverses the ASD-like behavioral symptoms in the mice. Similar to the mouse model, human ASD patients also frequently display disorganized cortex patches. Therefore, identifying cortex patches and quieting the neuron hyperactivity in these locations with pharmacologic or optogenetic techniques is a promising new treatment opportunity for ASD.


  • Novel therapeutic approach for ASD by calming hyperactivated cortex neurons
  • Hyperactive cortex patches provide a new diagnostic/theranostic strategy for ASD
  • Extensive in vivo data demonstrates success of this technology in ASD mouse model