Understanding how liver-specific sugars affect a protein linked to Niemann-Pick disease
Official title: Investigating the importance of liver-specific glycans on NPC1 proteostasis
Funded at University of Iowa · NIH-11055968 · Data updated 2026-11-12
This study is looking at how certain sugars in the liver affect a protein called NPC1, which is important for moving cholesterol and is linked to Niemann-Pick disease type C, to find ways to help improve liver health for people with this condition.
Quick facts
| Grant type | R03 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Iowa NIH-funded |
| Lab location | 1 site (Iowa City, United States) |
| Project start | March 15, 2024 |
| Project end | February 28, 2026 |
| Funding year | 2025 |
| Award (this year) | $104,963 |
| NIH project number | 5R03DK138132-02 |
| Project ID | NIH-11055968 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Niemann-Pick disease type C, particularly those experiencing liver-related symptoms.
✕Less likely to help
Patients without Niemann-Pick disease type C or those whose condition does not involve liver complications may not benefit from this research.
What this research studies
This research investigates the role of liver-specific glycans in the stability and function of the NPC1 protein, which is crucial for cholesterol transport and is implicated in Niemann-Pick disease type C. The study aims to explore how these glycans influence the degradation and trafficking of the NPC1 protein, particularly in the liver, where defects can lead to severe health issues. By examining the differences in glycosylation between liver and brain NPC1, the research seeks to identify potential therapeutic targets for improving liver function in affected patients. The approach includes biochemical assays and cellular models to assess NPC1 proteostasis.
Why it matters
Potential benefit: If successful, this research could lead to new therapeutic strategies for managing liver complications in Niemann-Pick disease type C patients.
How similar studies have gone: While the specific focus on liver NPC1 proteostasis is novel, similar approaches targeting protein stability in other contexts have shown promise in previous research.
Where this research is happening
-
University of Iowa
Iowa City, United States Active
Researchers
- Principal investigator: Schultz, Mark Louis — University of Iowa
- Study coordinator: Schultz, Mark Louis
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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.