Augmenting Notch signaling to treat Alagille syndrome
Official title: Augmented Notch signaling as a therapeutic approach for Alagille Syndrome
Funded at Scintillon Institute for Photobiology · NIH-11390997 · Data updated 2026-11-12
A study tests a small molecule that boosts Notch signaling to help people with Alagille syndrome by correcting liver and other organ defects.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Scintillon Institute for Photobiology NIH-funded |
| Lab location | 1 site (San Diego, United States) |
| Project start | August 1, 2022 |
| Project end | May 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $433,180 |
| NIH project number | 5R01DK134099-05 |
| Project ID | NIH-11390997 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates would have Alagille syndrome or related Jag/Notch pathway abnormalities and be available for future translational studies or trials.
✕Less likely to help
Patients without Jag/Notch pathway involvement or with advanced disease may be less likely to benefit.
What this research studies
This project uses a zebrafish model to mimic Alagille syndrome and tests a Notch-activating drug that could restore bile duct formation and reduce organ problems. Researchers will translate findings to mammalian cells and look for rescue of disease-related features. If successful, the work could point toward a new therapeutic option for patients with this genetic condition.
Why it matters
Potential benefit: If successful, it could lead to a therapy that improves liver function and related symptoms in Alagille syndrome.
How similar studies have gone: Earlier models showed that modulating Notch signaling could influence disease features, but this approach is still being validated.
Where this research is happening
-
Scintillon Institute for Photobiology
San Diego, United States Active
Researchers
- Principal investigator: Dong, P. Duc Si — Scintillon Institute for Photobiology
- Study coordinator: Dong, P. Duc Si
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.