Researchers have developed a novel CRISPR-based gene therapy approach aiming to treat SCN2A haploinsufficiency, a genetic cause of neurodevelopmental disorders including autism and epilepsy. The approach boosts expression levels of SCN2A, which encodes a critical neuronal sodium channel. Preclinical models demonstrated potential reversal of neurological symptoms by correcting gene dosage deficits. This advancement highlights CRISPR's transformative impact on addressing rare genetic brain disorders through precise genome modulation.