Improving gene therapy for liver diseases using advanced viral vectors
Next-generation human liver gene therapy
This study is working on improving gene therapy for liver conditions like hemophilia by creating better delivery systems that can safely target liver cells, so patients can have more effective treatment options.
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-10879162 on NIH RePORTER |
What this research studies
This research focuses on enhancing liver gene therapy for conditions like hemophilia by developing more effective adeno-associated virus (AAV) vectors. It aims to identify and engineer AAV capsids that can efficiently target human liver cells while minimizing side effects. The study utilizes normothermic machine perfusion to create near-clinical conditions for testing these vectors, comparing their performance in human liver cells to those used in current clinical trials. By improving the delivery of gene therapy, the research seeks to provide better treatment options for patients with liver diseases.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with liver diseases, particularly those with hemophilia or related conditions requiring gene therapy.
Not a fit: Patients with liver diseases not related to hemophilia or those who do not require gene therapy may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective and safer gene therapies for patients with liver diseases.
How similar studies have performed: Previous clinical trials have shown the potential of AAV vectors in liver gene therapy, but this research aims to address their limitations, making it a novel approach.
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.