Award
National Institute of Neurological Disorders and Stroke 1R61NS140718-01A1
Evaluation of Gene Replacement Therapy in In Vivo and Patient-Derived In Vitro Models of Vanishing White Matter Disease
Recipient
Research Institutes, Columbus, OH, United States
Award Amount
$381,127.00
Ceiling
$381,127.00
Awarded
March 09, 2026
Identifier
1R61NS140718-01A1
The award supports research to develop and validate an AAV-mediated gene replacement therapy for Vanishing White Matter Disease using mouse and patient-derived models, aiming to prevent or mitigate this severe neurodegenerative condition.
Description
This project aims to develop a targeted adeno-associated virus (AAV) mediated gene replacement therapy for Vanishing White Matter Disease (VWM), a progressive neurodegenerative disease affecting children. The therapy will be optimized and validated in mouse models and patient-derived cerebral organoid models. The goal is to prevent or mitigate VWM disease by developing and testing AAV-mediated EIF2B5 gene replacement therapy.