Figure1. Diverse autism-risk genes converge on two opposing molecular brain states (IMAGE)
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Transcriptomic profiling of 17 ASD mouse models representing over 1,200 human autism-risk genes, combined with single-cell RNA sequencing of approximately one million brain cells, identified two opposing molecular brain states despite diverse genetic etiologies. Group 1 showed reduced synaptic gene expression, increased chromatin and mRNA-processing programs, more consistent molecular responses to fluoxetine and lithium, and stronger cell-type remodeling, whereas Group 2 displayed the opposite molecular profile, more variable drug responses, and more moderate cellular remodeling. Comparable transcriptomic subgroups were also observed in the human ASD cortex, supporting a new framework for molecular classification and precision treatment of autism.
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Institute for Basic Science
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