Investigating new ways to treat cholestasis caused by bile salt export pump deficiency
Molecular targets in cholestasis caused by bile salt export pump deficiency
This study is looking at how changes in a specific gene can cause a liver disease called PFIC2, and it's trying to find new ways to help people with this condition by using zebrafish to discover other ways the liver can get rid of bile acids.
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-10456780 on NIH RePORTER |
What this research studies
This research focuses on understanding how mutations in the ABCB11 gene lead to a serious liver disease known as progressive familial intrahepatic cholestasis type II (PFIC2). The researchers aim to identify alternative mechanisms that can restore bile excretion in patients who lack the bile salt export pump (BSEP). By using zebrafish models, they will explore potential alternative transporters that could be activated to help excrete bile acids. The ultimate goal is to develop targeted therapies that could improve outcomes for patients suffering from cholestatic liver diseases.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals diagnosed with progressive familial intrahepatic cholestasis type II or other cholestatic liver diseases.
Not a fit: Patients with cholestatic liver diseases not related to BSEP deficiency may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new treatments for cholestasis, potentially reducing the need for liver transplants in affected patients.
How similar studies have performed: While the approach of identifying alternative transporters is innovative, similar research has shown promise in understanding cholestatic conditions, indicating potential for success.
Where this research is happening
Cincinnati, United States
- Cincinnati Childrens Hosp Med Ctr — Cincinnati, United States (Active)
Researchers
- Principal investigator: Yin, Chunyue — Cincinnati Childrens Hosp Med Ctr
- Study coordinator: Yin, Chunyue
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.