Researchers at UCSF demonstrated that CRISPR activation of the functional SCN2A gene copy in mouse models restores synaptic maturation and mitigates seizure susceptibility, representing a breakthrough in treating neurodevelopmental disorders caused by SCN2A haploinsufficiency. This approach, focusing on upregulating the existing functional allele rather than editing mutations, showed efficacy even in juvenile brains, offering promising translational potential for patients with autism spectrum disorder and epilepsy linked to SCN2A defects.