Advancing precision medicine: genome engineering for on-demand gene therapy in Dravet Syndrome

Lead researcher: Dr Jenna Carpenter

Partners: Epilepsy Research Institute

Start date: 2024  

As part of the Epilepsy Research Institute’s 2024 Research Awards, Dravet Syndrome UK co-funded two Fellowship projects with the Epilepsy Research Institute to develop future leaders investigating Dravet Syndrome and epilepsy. One of these Fellowships has been awarded to Dr Jenna Carpenter - with the aim of advancing precision medicine: genome engineering for on-demand gene therapy in Dravet Syndrome.

Dr Jenna Carpenter’s project aims to develop a ground-breaking gene therapy for Dravet Syndrome. This innovative therapy would offer a permanent treatment, uniquely designed to adapt dynamically to the patient's developing brain over their lifetime. Beyond stopping seizures, this on-demand therapy could significantly alleviate other debilitating aspects of the condition. By integrating cutting-edge technologies into their research, the team are setting a new standard in treatments for Dravet Syndrome and other rare genetic epilepsies. 

This research will enhance our understanding of how the SCN1A gene is read and give us more information about the changes in this gene that cause Dravet Syndrome and other related epilepsies. If successful, this project will establish a ground-breaking treatment strategy for Dravet Syndrome and other genetic epilepsies.

You can read more about this research here.