Investigating new treatments for Aicardi-Goutières syndrome
Official title: Efficacy and mechanisms of anti-IFN drugs in AGS treatment
Funded at University of Pittsburgh at Pittsburgh · NIH-11015885 · Data updated 2026-11-12
This study is looking at new medications that might help babies with Aicardi-Goutières syndrome by reducing brain inflammation and improving their brain function, using special mouse models that mimic the condition.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pittsburgh at Pittsburgh NIH-funded |
| Lab location | 1 site (Pittsburgh, United States) |
| Project start | February 1, 2024 |
| Project end | January 31, 2029 |
| Funding year | 2025 |
| Award (this year) | $508,545 |
| NIH project number | 5R01NS134651-02 |
| Project ID | NIH-11015885 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are infants and children diagnosed with Aicardi-Goutières syndrome.
✕Less likely to help
Patients with other forms of brain injury or those not diagnosed with Aicardi-Goutières syndrome may not benefit from this research.
What this research studies
This research focuses on Aicardi-Goutières syndrome (AGS), a severe genetic condition that leads to significant cognitive and physical disabilities in infants. The study aims to evaluate the effectiveness of new drugs that inhibit the interferon pathway, specifically targeting brain inflammation associated with AGS. Using newly developed mouse models that mimic the disease's characteristics, researchers will assess how these drugs can improve brain function and reduce damage. The goal is to find a viable therapeutic option for patients suffering from this rare condition.
Why it matters
Potential benefit: If successful, this research could lead to effective treatments that significantly improve the quality of life for patients with Aicardi-Goutières syndrome.
How similar studies have gone: While there have been trials with JAK inhibitors in AGS, this specific approach using newly developed mouse models and targeting brain inflammation is novel.
Where this research is happening
-
University of Pittsburgh at Pittsburgh
Pittsburgh, United States Active
Researchers
- Principal investigator: Wang, Qingde — University of Pittsburgh at Pittsburgh
- Study coordinator: Wang, Qingde
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.