Understanding immune responses to gene therapy using AAV vectors
Vector Immunology Core
['FUNDING_P01'] · UNIV OF MASSACHUSETTS MED SCH WORCESTER · NIH-10914217
This study is looking at how the immune system reacts to a type of virus used in gene therapy, which could help make these treatments work better for patients.
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-10914217 on ClinicalTrials.gov |
What this research studies
This research focuses on developing and standardizing immunological assays to study how the immune system responds to adeno-associated virus (AAV) vectors, which are used in gene therapy. By examining B-cell and T-cell responses in animal models, including mice and ferrets, the research aims to characterize immune reactions to both the viral capsid and the therapeutic gene product. The findings will provide essential data to improve the effectiveness of gene and cell therapies, ultimately enhancing patient outcomes.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with conditions that could benefit from AAV-based gene therapies, such as AAT deficiency or cystic fibrosis.
Not a fit: Patients who do not have conditions treatable by AAV-based therapies or those who are not eligible for gene therapy may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective gene therapies by improving our understanding of immune responses and how to manage them.
How similar studies have performed: Previous research has shown promise in understanding immune responses to AAV vectors, indicating that this approach has potential for success.
Where this research is happening
WORCESTER, UNITED STATES
- UNIV OF MASSACHUSETTS MED SCH WORCESTER — WORCESTER, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: GAO, GUANGPING — UNIV OF MASSACHUSETTS MED SCH WORCESTER
- Study coordinator: GAO, GUANGPING
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.
Conditions: Airway infections