Award

National Institute of Neurological Disorders and Stroke 5R01NS120960-05

Role of Brain Macrophages in the Pathogenesis and Treatment of Globoid Cell Leukodystrophy

Recipient

University of Pennsylvania, Philadelphia, PA, United States

Award Amount

$468,456.00

Ceiling

$468,456.00

Awarded

April 17, 2025

Identifier

5R01NS120960-05

The NIH-funded project investigates the role of brain macrophages in the fatal pediatric neurodegenerative disease Globoid Cell Leukodystrophy (GLD) and explores microglia replacement therapies to treat the disease.

Description

This project aims to understand the role of brain macrophages, including globoid cells, in GLD pathogenesis and to develop microglia replacement therapies. It involves defining the origin of reactive macrophages, testing microglia replacement, and evaluating HSC-derived cells as microglial surrogates.

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