Designing new AAV vectors to target liver cells and avoid immune response
Official title: Rational design of AAV vectors with human hepatocyte tropism and neutralizing antibody evasion
Funded at Bedrock Therapeutics, INC. · NIH-10546241 · Data updated 2026-11-12
This study is working on a new way to deliver gene therapy directly to liver cells while avoiding the body's immune response, which could lead to better treatment options for people with liver diseases.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Bedrock Therapeutics, INC. NIH-funded |
| Lab location | 1 site (Chapel Hill, United States) |
| Project start | September 1, 2022 |
| Project end | August 31, 2025 |
| Funding year | 2022 |
| Award (this year) | $261,080 |
| NIH project number | 1R41DK133021-01A1 |
| Project ID | NIH-10546241 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research would be patients with liver diseases who may benefit from gene therapy.
✕Less likely to help
Patients with conditions unrelated to liver function or those who do not require gene therapy may not benefit from this research.
What this research studies
This research focuses on developing adeno-associated virus (AAV) vectors that can effectively target human liver cells while avoiding the immune system's neutralizing antibodies. The approach involves engineering the AAV capsid to enhance its ability to deliver gene therapy specifically to the liver, which is crucial for treating various liver-related disorders. By utilizing a mouse model with human hepatocytes, the researchers aim to create AAV vectors that are more effective in humans than those tested in traditional animal models. This innovative methodology could lead to improved gene therapy options for patients with liver diseases.
Why it matters
Potential benefit: If successful, this research could provide more effective gene therapy treatments for patients with liver disorders.
How similar studies have gone: While AAV vectors have shown success in clinical applications, this specific approach to enhance liver targeting and evade immune responses is relatively novel.
Where this research is happening
-
Bedrock Therapeutics, INC.
Chapel Hill, United States Active
Researchers
- Principal investigator: Li, Chengwen — Bedrock Therapeutics, INC.
- Study coordinator: Li, Chengwen
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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.