Creating a safer viral vector for gene therapies
Official title: Developing a Synthetic Adeno-Associated Virus (AAV) for Engineering Safer Gene Therapies
Funded at University of California, San Diego · NIH-11010758 · Data updated 2026-11-12
This study is working on creating a safer version of a virus used in gene therapy, so that people with genetic disorders, especially those affecting the brain and nervous system, can have better and less risky treatment options.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Diego NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project start | February 1, 2023 |
| Project end | January 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $386,587 |
| NIH project number | 5R01NS131151-03 |
| Project ID | NIH-11010758 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children and adults with genetic disorders that could be treated with gene therapy, particularly those under 11 years old.
✕Less likely to help
Patients with conditions that do not involve genetic components or those who are not candidates for gene therapy may not benefit from this research.
What this research studies
This research focuses on developing a synthetic version of the adeno-associated virus (AAV) to improve the safety and effectiveness of gene therapies. By engineering a safer viral vector, the project aims to reduce the risk of adverse effects associated with current AAV-based therapies, which have been linked to serious complications such as acute kidney failure and other toxicities. The approach involves modifying the viral genome to minimize toxicity while maintaining its ability to deliver therapeutic genes effectively. Patients may benefit from safer gene therapy options for various genetic disorders, particularly those affecting the central nervous system.
Why it matters
Potential benefit: If successful, this research could lead to safer gene therapies with fewer side effects for patients with genetic disorders.
How similar studies have gone: While there have been successful gene therapies using AAV, the approach of creating a synthetic version to enhance safety is relatively novel and untested.
Where this research is happening
-
University of California, San Diego
La Jolla, United States Active
Researchers
- Principal investigator: Shtrahman, Matthew — University of California, San Diego
- Study coordinator: Shtrahman, Matthew
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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.