This review examines how scalable non-mammalian model systems (C. elegans, Drosophila, zebrafish, and Xenopus) are being used to study the function of >100 high-confidence autism spectrum disorder (ASD) risk genes, identify common biological pathways, and discover pharmacological targets.
Studies in these systems revealed conserved roles for ASD genes in learning, sensory processing, sleep, and social behavior circuits, and identified shared pathways including neurogenesis, microtubule biology, and mitochondrial function; pharmacological suppressor screens notably implicated estrogenic compounds as modulators of ASD gene-associated phenotypes.
All findings are derived from non-mammalian organisms, so translational relevance to human ASD biology requires validation in mammalian and human cellular systems. As a review, it does not present new primary experimental data.
No direct clinical action is warranted yet, but clinicians should be aware that large-scale cross-species functional screens are beginning to define convergent drug targets — including estrogenic compounds — that may inform future precision medicine approaches for ASD subgroups.
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