Medicine Zurich researchers repair epilepsy gene in the mouse brain
Zurich researchers have almost cured mice with a hereditary form of epilepsy. Using a new gene therapy, they almost completely prevented epileptic seizures in the mice.
13.05.2026, 20:00
SDA
This was reported by researchers led by the University of Zurich (UZH) in a study published on Wednesday evening in the journal "Science Translational Medicine".
The team treated mice suffering from a special form of epilepsy with febrile convulsions. In humans, the disease corresponds to the so-called GEFS+ syndrome, a rare hereditary form of epilepsy in which those affected often develop febrile convulsions in childhood.
Significant effect
The researchers succeeded in correcting the genetic defect using "prime editing", a relatively new form of gene editing. In the affected mice, the wrong genetic "letter" is located at a specific position in the "SCN1A" gene. For the treatment, the researchers used specially developed viruses to introduce "prime editing tools", including genetic repair instructions, directly into the brains of newborn mice. This corrected the genetic code in many nerve cells.
The effect was clear: in the control group, around 80 percent of the animals developed febrile convulsions. After the treatment, only around 15 percent did. The seizure rate thus approached the level of healthy mice. The survival rate of the animals rose from 80 to 100 percent.
Not yet for humans
However, the treatment is not yet suitable for use in humans. The therapy has only been tested on mice so far. According to the study, important hurdles remain before clinical application - such as possible immune reactions, administration into the human brain and a sufficiently even distribution of the gene correction in the brain.
"Even though these are preclinical data from a mouse model, our results open up new perspectives," said co-first author Lucas Kissling in a UZH press release on the study. The results could provide a basis for cause-based therapies - i.e. for treatments that directly target the cause of the disease: the defective gene.
