AUG 7, 2026 · JOURNAL ARTICLE
Gene Therapy for Autism Spectrum Disorder: Preclinical Advances, Translational Barriers, and Ethical Dimensions — A Scoping Review
Neuropsychobiology
This scoping review synthesizes preclinical evidence showing molecular and behavioral improvements in rodent models of monogenic ASD, but explicitly notes absence of completed human trials and significant translational barriers, limiting practice applicability.
Reported
Translational studies identified…12
Gene targets identifiedUBE3A, MECP2, FMR1, SHANK3/2, SCN2A, SYNGAP1