New methods to make repeat gene therapy safer and effective
Complementary Biotherapeutic Delivery Platforms for Enabling Gene Re-delivery
This project is developing delivery tools to help people get AAV-based gene therapy more than once by reducing immune reactions that block repeat dosing.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Utah State Higher Education System--University of Utah NIH-funded |
| Lab location | 1 site (Salt Lake City, United States) |
| Project ID | NIH-11406456 on NIH RePORTER |
What this research studies
Researchers are building three complementary delivery approaches to help gene therapies work again in patients whose immune systems stop repeat doses. One approach engineers regulatory T cells (CAR-Tregs) to travel to sites of immune reaction and calm responses that would otherwise clear the new gene. A second approach uses targeted enzymes to remove or disable the specific antibodies that neutralize viral gene carriers. A third approach focuses on alternative delivery tactics to protect or re-deliver the corrected gene while minimizing harmful immune or cell responses.
Who could benefit from this research
Good fit: People who have previously received AAV-based gene therapy but lost benefit, or who have neutralizing antibodies that prevent a second dose, would be most relevant.
Not a fit: Patients whose conditions are not treated with viral gene therapy or who cannot undergo immune-modulating or cell-based treatments may not benefit from these approaches.
Why it matters
Potential benefit: If successful, these tools could allow patients to receive additional rounds of AAV gene therapy safely and maintain or restore treatment effects.
How similar studies have performed: Some related strategies—like antibody-clearing enzymes and immune-modulating cell therapies—have shown promise in early work, but combining them specifically to enable AAV re-dosing is still relatively new.
Where this research is happening
Salt Lake City, United States
- Utah State Higher Education System--University of Utah — Salt Lake City, United States (Active)
Researchers
- Principal investigator: Mei, Kuo-Ching — Utah State Higher Education System--University of Utah
- Study coordinator: Mei, Kuo-Ching
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.