Zebrafish Drug Screen Targets Autism Genes, Spotlighting Levocarnitine

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Yale researchers used larval zebrafish to map how 774 FDA-approved drugs affect behaviors tied to autism-risk genes, building an open database of 520 compounds and identifying levocarnitine as a top rescuer for SCN2A and DYRK1A mutations; they validated the effect in human stem-cell–derived neurons, laying groundwork for precision, gene-targeted drug discovery in autism.
One Compound Repairs Neurons with Autism Mutations Neuroscience News
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