Award
National Institute of Neurological Disorders and Stroke 5R01NS136487-02
Microglia replacement using engineered HSCs for treatment of leukodystrophies
Recipient
University of Pennsylvania
Award Amount
$665,504.00
Ceiling
$665,504.00
Awarded
March 24, 2026
Identifier
5R01NS136487-02
The award funds research to develop a gene therapy using engineered hematopoietic stem cells to treat Krabbe disease by replacing microglia in the brain, with validation in animal models and plans for clinical translation.
Description
This project aims to develop a gene therapy approach to replace microglia in the brain using engineered hematopoietic stem cells (HSCs). The project involves validating gene editing techniques in mouse models and establishing safety and efficacy in nonhuman primates, with the goal of translating this therapy into clinical trials.