Gene therapy
Developing gene replacement strategies for rare pediatric neurological disorders.
FM Kirby Neurobiology Center
Advancing treatments for epilepsy caused by genetic disease and brain injury at Boston Children’s Hospital.
Our lab brings together preclinical discovery and clinical research to move promising ideas toward meaningful treatments.
In rodent models, our projects include gene replacement strategies for rare pediatric disorders of GABA homeostasis, including succinic semialdehyde dehydrogenase deficiency (SSADHD) and SLC6A1 neurodevelopmental disorder (SLC6A1-NDD). We also study interventions designed to suppress drug-resistant seizures and prevent epilepsy after traumatic brain injury.
In parallel, our clinical research program includes trials of novel anti-seizure therapies and the development of transcranial magnetic stimulation (TMS) protocols for children. We use TMS both to probe brain excitability and network function and to deliver targeted therapeutic stimulation.
Developing gene replacement strategies for rare pediatric neurological disorders.
Testing approaches to suppress seizures and prevent epilepsy after brain injury.
Using TMS and tDCS to measure and modify brain network function.