Targeted treatment for fatty liver disease using genetic medicine
Official title: A Precision Targeted Therapeutics for Nonalcoholic Fatty Liver Disease
Funded at Wayne State University · NIH-10832053 · Data updated 2026-11-12
This study is testing a new genetic treatment for people with nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH) who have a specific gene mutation, to see if it can help reduce fat in the liver and improve their health.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Wayne State University NIH-funded |
| Lab location | 1 site (Detroit, United States) |
| Project start | July 1, 2021 |
| Project end | April 30, 2026 |
| Funding year | 2024 |
| Award (this year) | $403,158 |
| NIH project number | 5R01DK124612-04 |
| Project ID | NIH-10832053 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with nonalcoholic fatty liver disease or nonalcoholic steatohepatitis who carry the PNPLA3 148M allele.
✕Less likely to help
Patients without the PNPLA3 148M allele or those with other forms of liver disease may not benefit from this research.
What this research studies
This research investigates a new approach to treat nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH) by using a specialized genetic medicine that targets a specific genetic mutation. The study focuses on developing an allele-specific small interfering RNA (siRNA) that can reduce the harmful effects of a protein associated with the progression of these liver diseases. By targeting the PNPLA3 gene mutation, the research aims to prevent the accumulation of fat in the liver and improve patient outcomes. Patients with this genetic mutation may have a unique response to this treatment, which could lead to significant advancements in managing these conditions.
Why it matters
Potential benefit: If successful, this research could provide a novel treatment option for patients suffering from fatty liver disease, potentially reversing liver damage and improving overall health.
How similar studies have gone: This approach is novel and has not been extensively tested in previous research, making it a potentially groundbreaking strategy for treating NAFLD/NASH.
Where this research is happening
-
Wayne State University
Detroit, United States Active
Researchers
- Principal investigator: Liu, Wanqing — Wayne State University
- Study coordinator: Liu, Wanqing
Related research
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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.