Scientists at UCSF demonstrated that CRISPR activation (CRISPRa) can boost expression of the functional SCN2A gene copy in a mouse model of SCN2A haploinsufficiency, a leading cause of neurodevelopmental disorders including autism and epilepsy. Published in Nature, the gene upregulation restored synaptic maturation, prevented seizures, and improved neurocognitive outcomes even when administered at developmental stages comparable to childhood. The study underscores CRISPRa’s therapeutic potential for haploinsufficiency disorders by enhancing endogenous gene expression without direct genome editing. These findings offer promising avenues for translational interventions targeting genetic causes of autism spectrum disorders and related conditions.