Award
National Institute of Neurological Disorders and Stroke 5R01NS136487-02
Microglia replacement using engineered HSCs for treatment of leukodystrophies
Recipient
University of Pennsylvania, Philadelphia, PA, United States
Award Amount
$665,504.00
Ceiling
$665,504.00
Awarded
March 24, 2026
Identifier
5R01NS136487-02
The award supports research on genetically engineered HSCs to improve microglia replacement as a treatment for Krabbe disease, focusing on safety and efficacy in preclinical models.
Description
This project aims to develop a clinically feasible immune cell therapy for Krabbe disease by genetically modifying hematopoietic stem cells (HSCs) to enhance microglia replacement in the central nervous system. The approach is validated in mouse and nonhuman primate models, targeting treatment of central nervous system pathology in globoid cell leukodystrophy (GLD).