Using a new humanized mouse to test gene-editing treatments for hepatitis B
A new humanized mouse model to study HBV gene editing
Researchers are testing gene-editing tools delivered by viral vectors or lipid nanoparticles to try to remove the viral DNA that keeps chronic hepatitis B alive.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Fred Hutchinson Cancer Center NIH-funded |
| Lab location | 1 site (Seattle, United States) |
| Project ID | NIH-11159791 on NIH RePORTER |
What this research studies
This project uses a low-cost 'humanized' mouse whose liver contains human hepatocytes so treatments interact with human liver cells. Investigators will deliver different gene-editing nucleases using AAV vectors or lipid nanoparticles to cut HBV covalently closed circular DNA (cccDNA) and compare which approaches best reduce the virus. They will also test chromatin-modifying agents to improve editing and measure how well each delivery method reaches human liver cells and lowers viral markers. Results will help pick the most promising strategies to move toward future human trials.
Who could benefit from this research
Good fit: People with chronic hepatitis B who remain on antiviral therapy or cannot clear the virus and who would consider future gene-editing clinical trials would be the likely candidates.
Not a fit: People who are vaccinated, already cured of hepatitis B, or who have very advanced liver failure or contraindications to gene therapy would likely not benefit from these approaches.
Why it matters
Potential benefit: If successful, this work could point to gene-editing treatments that permanently disable or remove HBV DNA in the liver, offering a path toward a cure for chronic hepatitis B.
How similar studies have performed: Gene-editing against HBV cccDNA has shown promise in cell and animal models but has not yet led to proven cures in people.
Where this research is happening
Seattle, United States
- Fred Hutchinson Cancer Center — Seattle, United States (Active)
Researchers
- Principal investigator: Jerome, Keith R — Fred Hutchinson Cancer Center
- Study coordinator: Jerome, Keith R
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.