Understanding how AAV gene therapy is silenced in the human liver
Official title: Dissecting AAV silencing in humanized mice
Funded at Duke University · NIH-11013878 · Data updated 2026-11-12
This study is looking into why some gene therapies using special viral vectors don't work well in the liver, and it's for people with liver diseases who might benefit from better treatments.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Duke University NIH-funded |
| Lab location | 1 site (Durham, United States) |
| Project start | February 1, 2023 |
| Project end | January 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $616,213 |
| NIH project number | 5R01DK134408-03 |
| Project ID | NIH-11013878 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals with liver diseases, particularly those affected by conditions like methylmalonic acidemia.
✕Less likely to help
Patients with conditions unrelated to liver function or those who do not have access to AAV gene therapy may not benefit from this research.
What this research studies
This research investigates the challenges of using Adeno-Associated Viral (AAV) vectors for gene therapy, particularly focusing on why these therapies can be silenced in the human liver. By utilizing a novel humanized liver model, the researchers aim to explore the molecular mechanisms behind AAV transgene silencing in both healthy and diseased liver cells. They will also examine the role of the Human Silencing Hub (HUSH) complex and test various vector designs and pharmacological strategies to overcome this silencing issue. This work could lead to improved AAV gene therapies for liver-related diseases.
Why it matters
Potential benefit: If successful, this research could enhance the effectiveness of AAV gene therapies, potentially leading to better treatment options for patients with liver diseases.
How similar studies have gone: While AAV gene therapy has been explored in various contexts, this specific investigation into AAV silencing mechanisms is novel and aims to address a significant barrier in the field.
Where this research is happening
-
Duke University
Durham, United States Active
Researchers
- Principal investigator: Bissig, Karl-Dimiter — Duke University
- Study coordinator: Bissig, Karl-Dimiter
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.