Target Optimization in Precision Treatment of KCNT1-Related Epilepsy
The discovery of epilepsy-causing gene variants has facilitated the development of precision medicines that target the molecular causes of disease. But to realize the full, side effect-free, therapeutic potential of these tools, they need to be targeted to the right cells and brain regions. Dr. Tobin will test strategies to optimize cutting-edge gene and drug therapies by selectively targeting the most severely affected cells and brain networks in a mouse model of KCNT1-related epilepsy. Each treatment will be evaluated not only in terms of seizure control but also in its ability to normalize brain activity – recognizing that our true goal is to holistically restore normal function in the epileptic brain.
This CURE Epilepsy Taking Flight Award is co-funded by the KCNT1 Epilepsy Foundation.