How different brain cells cause damage in Aicardi‑Goutières
Interactions Between Diverse Brain Cell Types Drive Aicardi-Goutieres Neuropathology
['FUNDING_R01'] · CHILDREN'S HOSP OF PHILADELPHIA · NIH-11166453
This project tests whether specific brain or blood‑vessel immune cells cause the brain injury seen in people with Aicardi‑Goutières to help guide better treatments for patients.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | CHILDREN'S HOSP OF PHILADELPHIA (nih funded) |
| Locations | 1 site (PHILADELPHIA, UNITED STATES) |
| Trial ID | NIH-11166453 on ClinicalTrials.gov |
What this research studies
Researchers will use new rodent models that carry human AGS mutations to see which cell types lead to type I interferon‑driven brain damage. They will use viral tools to target and manipulate particular brain and vascular cells and create immune chimeras that mix peripheral and brain immune cells to see where the damage starts. The team will track effects on neurons and oligodendrocytes (the cells that make myelin) to determine which compartments need rescue. The work is led by investigators at Children's Hospital of Philadelphia and the University of Pittsburgh.
Who could benefit from this research
Good fit: This is preclinical laboratory research and does not enroll patients, but it specifically focuses on people with genetically confirmed Aicardi‑Goutières syndrome.
Not a fit: People without AGS or whose symptoms arise from unrelated causes are unlikely to benefit directly from these experiments.
Why it matters
Potential benefit: If successful, this work could point to specific cell types to target with therapies that prevent or reduce brain injury in AGS.
How similar studies have performed: Related animal‑model and cell‑targeting approaches have helped clarify mechanisms in other genetic neuroinflammatory diseases, but applying viral targeting and immune chimeras to AGS is a new and novel approach.
Where this research is happening
PHILADELPHIA, UNITED STATES
- 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 below.
Conditions: Aicardi Goutieres syndrome