Researchers at the University of Zurich have used a novel gene editing approach to treat mice with an inherited form of epilepsy. The study, published in the journal Nature Communications on May 13, 2026, demonstrated that delivering CRISPR components directly to the brain could correct a mutation in the Kcna1 gene, which is linked to a severe form of epilepsy in humans.
The team, led by Professor Sandra Siegert, injected a harmless virus carrying the gene editing machinery into the brains of young mice. The treatment targeted neurons in the hippocampus, a region often involved in seizures. After the procedure, the mice showed a significant reduction in the frequency and severity of fever-induced seizures compared to untreated controls.
According to the study, the edited mice also had improved survival rates. While all untreated mice died within 30 days of the fever challenge, 80% of the treated mice survived. The researchers noted that the gene editing was precise, with no off-target effects detected in the analyzed brain tissue.
This is the first time that in vivo gene editing has been successfully used to treat epilepsy in a mammalian model. The approach could pave the way for future therapies for genetic forms of epilepsy in humans, though the researchers caution that more safety and efficacy studies are needed before clinical trials can begin.
