A new study in *Science* uncovers a previously overlooked molecular pathway that appears to drive some of the most severe forms of autism spectrum disorder. By combining genetic analysis of affected families with cutting‑edge brain‑cell imaging, the researchers identified a set of signaling proteins that malfunction in key neural circuits during early development. The findings not only fill a critical gap in the biological model of profound autism but also point to specific druggable targets that could be pursued in future therapies. If these targets prove viable, they may open the door to treatments that address core symptoms rather than just managing secondary behaviors.
Research published in the journal Science illuminates a "missing piece" in the biological understanding of profound autism and offers new targets for drug development.
