Safer viral gene delivery for brain and nervous system disorders
Developing a Synthetic Adeno-Associated Virus (AAV) for Engineering Safer Gene Therapies
Researchers are designing a safer viral carrier to deliver gene therapies for people with brain and central nervous system disorders.
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 ID | NIH-11266161 on NIH RePORTER |
What this research studies
This project is redesigning the AAV virus that carries therapeutic genes to make it less likely to cause harmful immune or organ reactions. The team will modify known toxic DNA elements in the AAV genome and test the new synthetic AAV in laboratory experiments and preclinical models to check where it goes in the body and whether it causes harm. The goal is to preserve the long-lasting gene expression and brain-targeting benefits of AAV while reducing risks such as inflammation, kidney injury, or other serious adverse effects reported in recent trials. If successful, the work could enable safer gene therapy options for a range of CNS conditions.
Who could benefit from this research
Good fit: People with genetic or other disorders of the brain or central nervous system who are interested in gene therapy or in contributing samples for research would be the most relevant candidates.
Not a fit: Patients without brain or CNS conditions, or those not eligible for gene therapy, are unlikely to see direct benefit from this project.
Why it matters
Potential benefit: Could make gene therapies for brain diseases safer and reduce serious side effects, enabling more people to receive these treatments.
How similar studies have performed: AAV-based gene therapies have produced FDA-approved treatments and many clinical trials, but modifying AAV genomes for improved human safety is an active area with limited clinical precedent after recent serious adverse events.
Where this research is happening
La Jolla, United States
- University of California, San Diego — La Jolla, United States (Active)
Researchers
- Principal investigator: Shtrahman, Matthew — University of California, San Diego
- Study coordinator: Shtrahman, Matthew
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.