Science Spotlight: Silencing seizures by fixing gene mutations

Two independent groups published back-to-back papers in Science Translational Medicine showing that in vivo gene editing can correct epilepsy-causing mutations in SCN1A, the gene encoding the sodium channel Nav1.1, in mouse models of severe seizure disorders.

A University of Zurich team used prime editing to correct the the Nav1.1 K1270T mutation in a mouse model of generalized epilepsy with febrile seizures plus (GEFS⁺). The team delivered the therapy to the cerebral ventricles of neonatal mice using AAV vectors carrying an intein-split prime editor under the control of a neuron-specific promoter. ...