Two children with epilepsy were treated with gene therapy.

Welcome to the era of personalized gene therapies. We are privileged witnesses to a medical revolution that promises to cure previously intractable orphan diseases. Advances in this field have been accumulating in recent years, thanks in large part to the rise of the gene therapyThis has made it possible to treat patients suffering from sickle cell disease, ofhemophilia or even rare forms of deafnessThese therapies treat diseases caused by a mutated gene and generally involve introducing a healthy version of the gene to compensate for the problems caused by the mutation. But another approach is beginning to gain traction: blocking the expression of this mutated gene so that the healthy version (when one of the gene copies is healthy) takes over. This is how researchers from the University of California, San Diego, and Rush University in Chicago succeeded in treating a rare form of epilepsy in two children. Their achievement was published on July 21, 2026, in Nature MedicineIt also shows that individualized gene therapies can be developed from a common platform, which could help reduce the production costs of these innovative drugs.

Mutations that send neurons into a frenzy