Investigating how chronic signaling affects immune cell function in a rare childhood disease
Official title: Chronic STAT3 signaling in STAT3 GOF causes CD8 T cell dysfunction via CD39 upregulation
Funded at Children's Hosp of Philadelphia · NIH-11024835 · Data updated 2026-11-12
This study is looking at a rare genetic condition that affects children and makes it harder for their immune system to fight infections and manage inflammation, and it aims to understand how a specific protein might be slowing down their immune response so that new treatments can be developed to help them feel better.
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 | December 20, 2024 |
| Project end | November 30, 2029 |
| Funding year | 2025 |
| Award (this year) | $658,426 |
| NIH project number | 1R01AI187202-01 |
| Project ID | NIH-11024835 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children diagnosed with STAT3 gain-of-function syndrome who experience immune dysfunction.
✕Less likely to help
Patients without STAT3 gain-of-function syndrome or those with other unrelated immune disorders may not benefit from this research.
What this research studies
This research focuses on a rare genetic condition known as STAT3 gain-of-function (GOF), which leads to increased inflammatory signaling and results in both viral infections and autoimmune issues in affected children. The study aims to understand how this condition impairs the function of CD8 T cells, which are crucial for fighting infections. Researchers will explore the role of a protein called CD39, which may inhibit immune responses by increasing levels of adenosine, a molecule that dampens T cell activity. By examining these mechanisms, the research seeks to uncover potential therapeutic targets to improve immune function in patients.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that enhance immune responses in children with STAT3 GOF, potentially reducing infections and autoimmune complications.
How similar studies have gone: While the specific approach of targeting CD39 in STAT3 GOF is novel, similar strategies targeting immune regulation have shown promise in other autoimmune and inflammatory conditions.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Henrickson, Sarah E — Children's Hosp of Philadelphia
- Study coordinator: Henrickson, Sarah E
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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.