Investigating the role of brain macrophages in a rare pediatric neurodegenerative disease.
Role of Brain Macrophages in the Pathogenesis and Treatment of Globoid Cell Leukodystrophy
This study is looking at how certain brain cells might help or hurt babies with globoid cell leukodystrophy (GLD), and it’s testing if giving them healthy cells can make things better.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pennsylvania NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project ID | NIH-11059843 on NIH RePORTER |
What this research studies
This research focuses on globoid cell leukodystrophy (GLD), a severe neurodegenerative condition affecting infants. The study aims to understand the role of specific brain cells, known as macrophages, in the disease's progression and treatment. Researchers will explore how these cells contribute to the disease and whether replacing them with healthy cells can improve outcomes. The approach involves advanced techniques in animal models to assess the effectiveness of potential therapies.
Who could benefit from this research
Good fit: Ideal candidates for this research are infants diagnosed with globoid cell leukodystrophy or those at risk due to genetic factors.
Not a fit: Patients with other neurodegenerative diseases or those who are not diagnosed with globoid cell leukodystrophy may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for children suffering from globoid cell leukodystrophy.
How similar studies have performed: While the approach of studying brain macrophages in this context is relatively novel, similar research has shown promise in understanding other neurodegenerative diseases.
Where this research is happening
Philadelphia, United States
- University of Pennsylvania — Philadelphia, United States (Active)
Researchers
- Principal investigator: Bennett, Frederick — University of Pennsylvania
- Study coordinator: Bennett, Frederick
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.