Developing safe and effective gene therapies using viral vectors
Official title: Viral Vector and Regulatory Core
Funded at Jackson Laboratory · NIH-11089349 · Data updated 2026-11-12
This study is working on making safer and better gene therapies using special tools called AAV vectors, which help deliver treatments to patients, so you can expect improved options for your condition in the future!
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Jackson Laboratory NIH-funded |
| Lab location | 1 site (Bar Harbor, United States) |
| Project start | May 16, 2023 |
| Project end | April 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $716,846 |
| NIH project number | 5U19NS132304-03 |
| Project ID | NIH-11089349 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for participation or benefit from this research are individuals with genetic disorders that could be treated with gene therapy.
✕Less likely to help
Patients with conditions that are not amenable to gene therapy or those who do not have genetic disorders may not receive benefit from this research.
What this research studies
This research focuses on the development and regulatory management of adeno-associated viral (AAV) vectors for gene therapy applications. It aims to ensure the safe and effective production of these vectors, which are crucial for delivering gene-editing treatments. The project includes creating protocols to manage immune responses to these therapies and navigating the regulatory pathways necessary for clinical trials. Patients may benefit from improved gene therapies that are safer and more effective due to rigorous testing and quality control.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective gene therapies for various genetic conditions.
How similar studies have gone: Previous research has shown success in using AAV vectors for gene therapy, indicating a promising approach for future treatments.
Where this research is happening
-
Jackson Laboratory
Bar Harbor, United States Active
Researchers
- Principal investigator: Gray, Steven J — Jackson Laboratory
- Study coordinator: Gray, Steven J
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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.