How genes and immune cells drive bile duct scarring in children with PSC
Genetic and microenvironmental regulation of macrophage functions driving biliary fibrosis in pediatric PSC
This project looks at how inherited genes and the liver environment change immune cells that cause scarring of the bile ducts in children with primary sclerosing cholangitis.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cincinnati Childrens Hosp Med Ctr NIH-funded |
| Lab location | 1 site (Cincinnati, United States) |
| Project ID | NIH-11302683 on NIH RePORTER |
What this research studies
From a patient's point of view, researchers will study liver tissue from children with early-onset primary sclerosing cholangitis (often with autoimmune hepatitis) to understand why bile ducts become damaged and scarred. They will use cutting-edge single‑cell and single‑nucleus methods that read both gene activity and regulatory DNA in individual cells, focusing on macrophages and bile‑acid sensing pathways. The team will link those cell‑level findings to known genetic risk sites from genome studies to see which risk variants alter cell behavior. The goal is to pinpoint the cell types and molecular switches that drive fibrosis so future treatments can target them.
Who could benefit from this research
Good fit: Ideal candidates are children with pediatric‑onset primary sclerosing cholangitis (including those with overlapping autoimmune hepatitis) whose clinical samples or tissue can be shared with the research team.
Not a fit: People without PSC or those seeking immediate treatment benefits are unlikely to gain direct clinical benefit from this basic and translational research.
Why it matters
Potential benefit: If successful, this work could identify specific immune cells and molecular targets that lead to new therapies to slow or prevent bile duct scarring in children with PSC.
How similar studies have performed: Previous single‑cell and genetic studies have implicated macrophages and bile‑acid signaling in PSC, but applying multiome (paired chromatin and RNA) analyses specifically to pediatric PSC is a newer and less tested approach.
Where this research is happening
Cincinnati, United States
- Cincinnati Childrens Hosp Med Ctr — Cincinnati, United States (Active)
Researchers
- Principal investigator: Miethke, Alexander — Cincinnati Childrens Hosp Med Ctr
- Study coordinator: Miethke, Alexander
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.