NeurosciencebioRxivPreprint
Gene therapy for targeting a prenatally enriched potassium channel associated with severe childhood epilepsy and premature death
Sean R Golinski, Karla Soriano, Alex C. Briegel…·Richard S. Smith
The paper demonstrates that ASO therapy targeting KCNT1 reduces seizures in a severe childhood epilepsy condition by normalizing neuronal function in prenatally enriched neurons.