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Finding new therapies for epileptic encephalopathies by employing knowledge from rare genetic diseases
Grants AwardedFor this project, his team will use human cell models and zebrafish to develop novel therapies for epileptic encephalopathy by targeting metabolic processes implicated in these pathways.
Dietary And Activity Treatments For Modulating Post-Traumatic Brain Hyperexcitability
Grants AwardedIn this two-year project, Dr. Schwartzkroin will study potential protective therapies in a rat model of traumatic brain injury.
Developing the Inhibitory Opsin eOPN3 as a Treatment for Drug Resistant Epilepsy
Grants AwardedIn this project, the team will build the devices and test the treatment in a large‑animal model of focal, drug‑resistant epilepsy.
UBA5-Related Epilepsy: From Cellular Models to Novel Therapies
Grants AwardedTo better understand how these changes affect brain development, Dr. Mefford’s team will study 3D disease-in-a-dish models (called brain organoids) made from patient cells.
Minimizing Seizure-Associated Adverse Effects Upon Gene Replacement Therapy in slc6a1 Epileptic Encephalopathy
Grants AwardedIn this study, Dr. Lee will test whether gene replacement therapy, a treatment aimed at delivering normal copies of the SLC6A1 gene to the brain, can improve symptoms in mice lacking the SLC6A1 gene.
Safety and Efficacy of a Novel MicroRNA-Based Therapy for Epilepsy in a Translational Animal Model
Grants AwardedDr. Gutierrez-Quintana’s team will now test the effects of Ant-134 in a population of dogs with naturally occurring drug-resistant epilepsy, an important step towards translating their findings into treatments for humans.
Restoration of Circadian Function as a Novel Therapy for Epilepsy
Grants AwardedDr. Reschke will study how disruption of circadian rhythms could affect gene expression during epileptogenesis.
Adenosine Kinase Inhibitors for Antiepileptogenic Therapy
Grants AwardedOur goal in this project is to optimize and test a new epilepsy preventing drug, which meets criteria for future clinical development.
Functional Outcomes and Advancing Therapy for FAM177A1-related Neurodevelopmental Disorder Using Patient-derived Neurons and Brain Organoids
Grants AwardedDr. Berrak’s team will use patient derived neuronal cellular and 3D brain-in-a-dish (organoids) models to examine where the FAM177A1 protein is located in neurons, protein it interacts with, and how its loss may affect neuronal growth and function.