Improving Gene Therapy for Liver Conditions
Official title: Acute/chronic limitations to transcriptional RNAi therapies for infectious and other liver diseases
Funded at Stanford University · NIH-11137041 · Data updated 2026-11-12
This research aims to make gene therapies safer and more effective for people with liver conditions by understanding how they sometimes cause liver problems.
Quick facts
| Grant type | R37 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Stanford University NIH-funded |
| Lab location | 1 site (Stanford, United States) |
| Project start | August 1, 2006 |
| Project end | July 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $699,711 |
| NIH project number | 5R37AI071068-20 |
| Project ID | NIH-11137041 on NIH RePORTER |
Who could benefit from this research
✓Could help
This foundational research is for future patients with genetic or infectious liver diseases who might benefit from gene therapy.
✕Less likely to help
Patients seeking immediate treatment or direct participation in a clinical trial would not directly benefit from this basic science research at this stage.
What this research studies
Gene therapies that use RNA to treat liver conditions hold great promise, even offering a potential one-time cure for some genetic disorders. However, sometimes these therapies can cause unexpected liver toxicity, which limits their use. Our team discovered one reason for this toxicity, linked to how certain RNA molecules are expressed in the liver. We are now working to understand the specific roles of different RNA components to prevent these side effects. By studying these molecules in cells and animal models, we hope to make gene therapies safer and more widely available.
Why it matters
Potential benefit: If successful, this work could lead to new, safer gene therapy treatments for various infectious and genetic liver diseases, reducing the risk of serious side effects.
How similar studies have gone: While RNAi products are FDA-approved for some liver conditions, this specific approach to understanding and preventing liver toxicity from gene therapy delivery is a novel and ongoing area of investigation.
Where this research is happening
-
Stanford University
Stanford, United States Active
Researchers
- Principal investigator: Kay, Mark a — Stanford University
- Study coordinator: Kay, Mark a
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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.