Prime editing-based gene correction alleviates the hyperexcitable phenotype and seizures of a genetic epilepsy mouse model

Dear Editor, Epilepsy is one of the most common chronic neurological diseases,affecting ～70 million people worldwide,and is listed as one of the key neurological and psychiatric diseases by the World Health Organization because of its long disease course and high disability rate[1,2].Although more than 20 anti-seizure medications(ASMs)have been developed to attenuate seizures,～30％of people with epilepsy do not benefit from the current medical treatment options[3,4].Developmental and epileptic encephalopathy(DEE)is a group of severe pediatric epilepsies oftencaused by single-gene mutations and is characterized by drug-resistant seizures and significant developmental delay or regression,with developmental disability persisting even when anti-seizure medications provide sufficient seizure control[5,6].