Acute and chronic limits of RNA interference therapies for liver diseases
Official title: Acute/chronic limitations to transcriptional RNAi therapies for infectious and other liver diseases
Funded at Stanford University · NIH-11326864 · Data updated 2026-11-12
This grant explores why certain RNA-based therapies delivered by a virus can cause liver toxicity, with the goal of making safer, one-time liver treatments possible.
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 | June 30, 2031 |
| Funding year | 2026 |
| Award (this year) | $770,320 |
| NIH project number | 4R37AI071068-21 |
| Project ID | NIH-11326864 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with liver diseases caused by genetic mutations and who might benefit from future RNAi therapies could be eligible for related future trials.
✕Less likely to help
The current work is preclinical and does not enroll patients.
What this research studies
If this work advances, it could help people with liver-related genetic disorders by improving RNAi therapies delivered with AAV vectors. Researchers study how high levels of therapeutic RNAi might briefly disrupt liver cells and how to separate the harmful effects from beneficial ones in cells and mouse models. The team will map how these RNAs interact with DNA and chromatin to understand exact mechanisms. The findings could guide development of safer liver-targeted gene-silencing treatments in the future.
Why it matters
Potential benefit: Safer, potentially one-time RNA-based therapies for liver diseases could emerge.
How similar studies have gone: Other studies have shown that RNAi therapies can be effective but may cause liver toxicity at high expression levels; this project aims to reduce such risks.
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.