Investigating how natural viruses affect gene therapy vectors in liver cells
Official title: In vitro and in vivo modeling of gene therapy vector stability when challenged by natural virus infection in human hepatocytes
Funded at Univ of Massachusetts Med Sch Worcester · NIH-11054776 · Data updated 2026-11-12
This study is looking at how natural viral infections might affect the stability of gene therapy treatments that use adeno-associated virus (AAV) vectors, especially in liver cells, to help ensure these therapies are safe and effective for people who need them.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of Massachusetts Med Sch Worcester NIH-funded |
| Lab location | 1 site (Worcester, United States) |
| Project start | November 13, 2024 |
| Project end | October 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $284,331 |
| NIH project number | 1R21AI183080-01A1 |
| Project ID | NIH-11054776 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with genetic disorders that are being treated with AAV-based gene therapies.
✕Less likely to help
Patients who do not have genetic diseases or are not receiving AAV-based gene therapies may not benefit from this research.
What this research studies
This research focuses on understanding the stability of adeno-associated virus (AAV) vectors used in gene therapy when faced with natural viral infections. By modeling these interactions in both laboratory settings and living organisms, the study aims to uncover how such infections might destabilize the therapeutic gene vectors in human liver cells, which are a common target for these treatments. The research will explore the potential changes in the vector genomes caused by these infections, providing critical insights into the safety and efficacy of AAV-based gene therapies.
Why it matters
Potential benefit: If successful, this research could enhance the safety and effectiveness of gene therapies for patients with genetic diseases.
How similar studies have gone: While gene therapy using AAV vectors has shown promise, this specific investigation into the effects of natural viral infections on vector stability is a novel approach.
Where this research is happening
-
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.