Improving Gene Therapy for Alpha-1-Antitrypsin Deficiency by Modulating Immune Responses
Modulation of Immune responses to gene therapy by Creation of AAV-specific Chimeric antigen receptor regulatory T cells (CAR-Tregs)
['FUNDING_P01'] · UNIV OF MASSACHUSETTS MED SCH WORCESTER · NIH-11146486
This project is developing special immune cells to help gene therapy work better and last longer for people with alpha-1-antitrypsin (AAT) deficiency.
Quick facts
| Phase | ['FUNDING_P01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIV OF MASSACHUSETTS MED SCH WORCESTER (nih funded) |
| Locations | 1 site (WORCESTER, UNITED STATES) |
| Trial ID | NIH-11146486 on ClinicalTrials.gov |
What this research studies
Gene therapy offers a promising way to treat conditions like AAT deficiency by delivering new genes, but sometimes our immune system fights off the treatment. This project is creating unique immune cells, called CAR-Tregs, that can calm down the body's immune response to gene therapy. We hope these cells will prevent the immune system from attacking the gene therapy, allowing the new genes to work effectively and for a longer time. This could also make it possible to give multiple doses of gene therapy or treat patients who already have some immunity to the therapy.
Who could benefit from this research
Good fit: This research is relevant for patients with alpha-1-antitrypsin deficiency who might one day receive gene therapy.
Not a fit: Patients without alpha-1-antitrypsin deficiency or those not considering gene therapy would not directly benefit from this specific research.
Why it matters
Potential benefit: If successful, this work could make gene therapy a more effective and widely available treatment option for AAT deficiency and potentially other genetic conditions.
How similar studies have performed: Similar approaches to control immune responses have shown promise in early gene therapy trials for other conditions like hemophilia.
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.