Award
National Institute of Neurological Disorders and Stroke 5R01NS134923-03
Uncovering mechanisms and developing novel therapeutic strategies for TBCD-related developmental and epileptic encephalopathy
Recipient
RESEARCH INST NATIONWIDE CHILDREN'S HOSP
Award Amount
$625,164.00
Ceiling
$625,164.00
Awarded
March 19, 2026
Identifier
5R01NS134923-03
Research project focused on understanding TBCD-related encephalopathy mechanisms and developing gene therapy approaches.
Description
This project aims to determine the neuropathological mechanisms of TBCD variants using patient-derived cerebral organoids and investigate the molecular and clinical phenotypes in a series of TBCD knock-in mouse models. The study will evaluate the efficacy of targeted AAV-mediated gene replacement therapy.