Negative Regulation of SOX4 in liver bile duct development (Alagille syndrome)
Official title: NEGATIVE REGULATION OF INTRAHEPATIC BILIARY DEVELOPMENT BY SOX4
Funded at Baylor College of Medicine · NIH-11465056 · Data updated 2026-11-12
A patient-focused study aims to understand how a gene called SOX4 affects bile duct growth in a genetic liver condition called Alagille syndrome, with the goal of finding new ways to help affected children.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Baylor College of Medicine NIH-funded |
| Lab location | 1 site (Houston, United States) |
| Project start | August 10, 2026 |
| Project end | April 30, 2030 |
| Funding year | 2026 |
| Award (this year) | $821,504 |
| NIH project number | 1R01DK145618-01A1 |
| Project ID | NIH-11465056 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are children with Alagille syndrome or Alagille-Watson syndrome who have or are at risk for bile duct paucity.
✕Less likely to help
Patients without ALGS or without bile duct development issues may not directly benefit from this study.
What this research studies
This research uses a mouse model that mimics Alagille syndrome to explore how reduced SOX4 activity impacts bile duct formation. The team will examine liver tissue and gene activity, and test the effects of gene-therapy approaches using safe delivery methods to see if bile ducts can develop more normally. The goal is to identify targets that could lead to therapies that reduce liver disease severity and the need for transplant in kids with ALGS. Results from these studies could guide future human trials and personalized treatment ideas for patients with BD paucity.
Why it matters
Potential benefit: If successful, this work could point toward new treatments that support bile duct development and improve liver function in ALGS patients.
How similar studies have gone: Previous work in animal models suggests that modulating pathways related to bile duct development can alter disease severity, but human-specific therapies remain experimental.
Where this research is happening
-
Baylor College of Medicine
Houston, United States Active
Researchers
- Principal investigator: Jafar-Nejad, Hamed — Baylor College of Medicine
- Study coordinator: Jafar-Nejad, Hamed
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.