How common viral infections might change AAV gene therapy in liver cells
In vitro and in vivo modeling of gene therapy vector stability when challenged by natural virus infection in human hepatocytes
['FUNDING_R21'] · UNIV OF MASSACHUSETTS MED SCH WORCESTER · NIH-11231265
This work looks at whether routine viral infections can change how AAV-based gene therapies behave in human liver cells used to treat genetic liver conditions.
Quick facts
| Phase | ['FUNDING_R21'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIV OF MASSACHUSETTS MED SCH WORCESTER (nih funded) |
| Locations | 1 site (WORCESTER, UNITED STATES) |
| Trial ID | NIH-11231265 on ClinicalTrials.gov |
What this research studies
Researchers will expose human liver cells and living models to common helper viruses (such as adenoviruses, herpesviruses, and papillomaviruses) to see how those infections affect therapeutic AAV vector genomes. They will use lab-grown human hepatocytes and in vivo models to mimic what happens after a person receives an AAV-based liver gene therapy. The team will measure changes in the amount and structure of the therapeutic vector DNA and look for signs of amplification or rearrangement (called "mobilization"). Results will be used to better understand risks to long-term stability of AAV therapies in treated tissues.
Who could benefit from this research
Good fit: Ideal participants would include people with inherited liver conditions who have received or may receive AAV-based gene therapy, and individuals willing to donate liver tissue or cells for research.
Not a fit: People whose conditions are unrelated to AAV-based liver gene therapies or who cannot provide tissue or cell samples are unlikely to directly benefit from participation.
Why it matters
Potential benefit: If successful, this work could help make AAV-based liver gene therapies safer and more reliable by identifying infection-related risks to the therapy's stability.
How similar studies have performed: AAV gene therapies have shown clinical success, but few prior studies have directly modeled how natural co-infections might destabilize therapeutic AAV genomes, so this approach is relatively novel.
Where this research is happening
WORCESTER, UNITED STATES
- UNIV OF MASSACHUSETTS MED SCH WORCESTER — WORCESTER, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: TAI, PHILLIP — UNIV OF MASSACHUSETTS MED SCH WORCESTER
- Study coordinator: TAI, PHILLIP
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.