Researchers from UCSF have demonstrated a novel CRISPR activation (CRISPRa) approach that boosts expression of the functional SCN2A gene copy in a mouse model mimicking human neurodevelopmental disorders caused by SCN2A haploinsufficiency. Treatment restored synaptic function and prevented seizures, showing efficacy when administered at a developmental stage comparable to human childhood. This preclinical work, published in Nature, opens pathways for gene regulation therapeutics in neurodevelopmental conditions like autism and epilepsy.