Improving gene therapy for alpha-1-antitrypsin disease using modified immune cells
Modulation of Immune responses to gene therapy by Creation of AAV-specific Chimeric antigen receptor regulatory T cells (CAR-Tregs)
This study is looking at a new way to make gene therapy for alpha-1-antitrypsin disease work better by using special immune cells that help control the body's response, so patients can get more effective treatment and possibly receive it multiple times if needed.
Quick facts
| Grant type | P01 program project |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of Massachusetts Med Sch Worcester NIH-funded |
| Lab location | 1 site (Worcester, United States) |
| Project ID | NIH-10914227 on NIH RePORTER |
What this research studies
This research focuses on enhancing the effectiveness of gene therapy for alpha-1-antitrypsin (AAT) disease by using specially designed immune cells called chimeric antigen receptor regulatory T cells (CAR-Tregs). These CAR-Tregs are engineered to modulate the immune response to adeno-associated virus (AAV) gene therapy, which can be hindered by pre-existing immunity in patients. The researchers will optimize these CAR-Tregs to ensure they can effectively suppress unwanted immune reactions, allowing for better and longer-lasting therapeutic effects. The study will also explore the potential for re-dosing AAV vectors, which could improve treatment outcomes for patients needing multiple therapies.
Who could benefit from this research
Good fit: Ideal candidates for this research are patients diagnosed with alpha-1-antitrypsin deficiency who may benefit from gene therapy.
Not a fit: Patients with conditions unrelated to AAT deficiency or those who do not respond to AAV-based therapies may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective and durable gene therapies for patients with AAT deficiency.
How similar studies have performed: Previous research has shown promise in using modified immune cells to enhance gene therapy, indicating a potential for success in this novel approach.
Where this research is happening
Worcester, United States
- Univ of Massachusetts Med Sch Worcester — Worcester, United States (Active)
Researchers
- Principal investigator: Keeler-Klunk, Allison May — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Keeler-Klunk, Allison May
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.