The present application relates to the technical field of
medicine. Disclosed for the first time is a chemogenetic
gene therapy technology for treating
autism spectrum disorder. Specifically, provided in the present technology is a combined
drug treatment strategy, which comprises a
neuron-targeted intracranial injection and a small-molecule
agonist (the latter serving as a second
drug administered after
surgery). According to this strategy, the injection is precisely delivered to the posterior substantia innominata
brain region by means of stereotactic intracranial injection, and then the second
drug is administered after
surgery, so as to intervene in the social behavioral abnormalities associated with
autism spectrum disorder. The therapy can prolong the
attack latency, shorten the duration of aggressive behaviors, and reduce the frequency of aggressive episodes in model animals, while not interfering with basic vital activities and emotional states such as normal social interaction, fear and
anxiety. This strategy pioneers a novel approach for treating
autism spectrum disorder, which is expected to improve the
quality of life of patients and alleviate the burdens on the families thereof and society, and thus has broad prospects for clinical application.