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, Madeline Burke, Timothy W. Yu, Tojo Nakayama, Ruing Hu, 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.
bioRxiv · Oct 24, 2024 · PreprintNeuroscience