How the liver protein RXRα controls PCSK9 and cholesterol levels
Official title: Role of Retinoid X Receptor Alpha in regulating PCSK9 transcription in the liver
Funded at New York University D/b/a NYU Long Island School of Medicine · NIH-11290359 · Data updated 2026-11-12
This research is testing whether boosting a liver protein called RXRα can lower PCSK9 and reduce LDL (bad) cholesterol for people with fatty liver disease or atherosclerotic heart disease.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | New York University D/b/a NYU Long Island School of Medicine NIH-funded |
| Lab location | 1 site (Mineola, United States) |
| Project start | April 1, 2023 |
| Project end | January 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $624,547 |
| NIH project number | 5R01DK135630-04 |
| Project ID | NIH-11290359 on NIH RePORTER |
Who could benefit from this research
✓Could help
The eventual target population would be people with nonalcoholic fatty liver disease (NAFLD) or atherosclerotic cardiovascular disease who have elevated LDL cholesterol.
✕Less likely to help
Because this is preclinical work in cells and animals, people without fatty liver or high LDL or those needing immediate treatment are unlikely to benefit directly from this project now.
What this research studies
You should know that the team uses liver-focused genetic approaches in cells and animals to turn RXRα off and on and measure resulting changes in PCSK9 and blood cholesterol. They use hepatocyte-specific knockout models and AAV8 gene delivery to restore RXRα in the liver, and they track cholesterol and LDL-C in the blood. The researchers will also study how RXRα interacts with PPARα signaling and other molecular steps that control PCSK9 production. Results are intended to point toward molecular targets that could be tested in people in future studies.
Why it matters
Potential benefit: If successful, this work could identify new ways to lower PCSK9 and LDL cholesterol that lead to better treatments for people with fatty liver disease and high cardiovascular risk.
How similar studies have gone: Existing PCSK9-targeting drugs (antibodies and RNAi) have lowered LDL but have limitations, and directly targeting RXRα regulation of PCSK9 is a relatively novel, mainly preclinical approach.
Where this research is happening
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New York University D/b/a NYU Long Island School of Medicine
Mineola, United States Active
Researchers
- Principal investigator: Miao, Qing — New York University D/b/a NYU Long Island School of Medicine
- Study coordinator: Miao, Qing
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.