Improving Gene Therapy for Liver Conditions
Next-generation human liver gene therapy
This project is finding better ways to deliver gene therapy to human liver cells, aiming to make treatments safer and more effective for various liver diseases.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project ID | NIH-11117194 on NIH RePORTER |
What this research studies
Current gene therapies for liver conditions, like hemophilia, have shown promise but also have limitations in how well they work and potential side effects. This project aims to overcome these challenges by discovering new and improved ways to deliver therapeutic genes directly to liver cells. Researchers will test different gene delivery methods using donated human livers kept alive outside the body, which helps them understand how these methods will work in people. The goal is to find the most efficient and safest delivery systems to help more patients with liver diseases.
Who could benefit from this research
Good fit: Patients with various liver diseases who might benefit from gene therapy could be ideal candidates for future clinical applications stemming from this research.
Not a fit: Patients whose liver conditions are not treatable by gene therapy or who have contraindications for such treatments may not directly benefit from this specific research.
Why it matters
Potential benefit: If successful, this work could lead to more effective and safer gene therapies for a wider range of liver diseases, improving patient outcomes.
How similar studies have performed: Pioneering clinical trials have established the basic effectiveness of AAV gene therapy for liver conditions, but this project seeks to significantly improve upon existing methods.
Where this research is happening
San Francisco, United States
- University of California, San Francisco — San Francisco, United States (Active)
Researchers
- Principal investigator: Willenbring, Holger — University of California, San Francisco
- Study coordinator: Willenbring, Holger
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 below.