Understanding how different brain cells contribute to Aicardi-Goutieres syndrome
Official title: Interactions between diverse brain cell types drive Aicardi-Goutieres neuropathology
Funded at Children's Hosp of Philadelphia · NIH-10943522 · Data updated 2026-11-12
This study is looking at how different types of brain cells are involved in Aicardi-Goutieres syndrome (AGS) to help find new ways to treat this challenging condition.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | August 15, 2024 |
| Project end | July 31, 2029 |
| Funding year | 2024 |
| Award (this year) | $686,276 |
| NIH project number | 1R01NS138537-01 |
| Project ID | NIH-10943522 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Aicardi-Goutieres syndrome, particularly those with specific genetic mutations associated with the condition.
✕Less likely to help
Patients with other neurological disorders or those without Aicardi-Goutieres syndrome may not benefit from this research.
What this research studies
This research investigates the role of various brain cell types in Aicardi-Goutieres syndrome (AGS), a severe condition that leads to significant cognitive and physical disabilities. The study aims to identify which specific cells in the immune system or brain contribute to the disease's progression by using advanced rodent models that mimic patient mutations. Researchers will employ innovative techniques to target and analyze these cells, seeking to understand how genetic mutations lead to brain injury. The ultimate goal is to uncover potential therapeutic targets that could lead to effective treatments for AGS.
Why it matters
Potential benefit: If successful, this research could lead to targeted therapies that significantly improve outcomes for patients with Aicardi-Goutieres syndrome.
How similar studies have gone: Previous research has shown promise in understanding the role of immune cells in other neurological conditions, suggesting potential for success in this novel approach.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Bennett, Mariko L. — Children's Hosp of Philadelphia
- Study coordinator: Bennett, Mariko L.
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.