Targeted brain-delivery AAV toolkit for cell-type specific gene expression
Official title: Scaled identification of receptor-targeted AAVs for potent and cell type-specific transgene delivery
Funded at California Institute of Technology · NIH-11401064 · Data updated 2026-11-12
This NIH grant aims to create a toolkit of engineered AAVs to deliver genes to specific brain cell types in rodents and non-human primates, improving delivery across the blood-brain barrier.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | California Institute of Technology NIH-funded |
| Lab location | 1 site (Pasadena, United States) |
| Project start | September 1, 2025 |
| Project end | May 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $2,578,585 |
| NIH project number | 5U01MH139785-02 |
| Project ID | NIH-11401064 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates include individuals with brain diseases where gene therapy or neuroscience research could eventually inform treatment strategies, or people who may participate in future related trials or samples related to brain gene delivery research.
✕Less likely to help
Participants in early tool development would not receive a therapeutic benefit themselves and may not be directly impacted unless later translated.
What this research studies
Researchers will develop and scale AAV capsids designed to cross the blood-brain barrier and target particular brain cell types in mice and primates. The project combines capsid engineering, receptor-mediated transcytosis insights, and in vitro screening to identify promising BBB receptors. They will produce a library of hundreds of capsids and dozens of cargo designs to enable broad cell-type specific expression in the brain. The work is aimed at enabling primate-wide delivery and efficient screening of neuronal control elements for future translational studies.
Why it matters
Potential benefit: If successful, patients could benefit from more precise brain-targeted gene therapies and research tools that enable safer, wider delivery of therapeutic payloads.
How similar studies have gone: Previous work shows promise in AAV capsid engineering and BBB crossing, with ongoing advances in rodent and primate models, though this project aims to reach parity in potency between species.
Where this research is happening
-
California Institute of Technology
Pasadena, United States Active
Researchers
- Principal investigator: Gradinaru, Viviana — California Institute of Technology
- Study coordinator: Gradinaru, Viviana
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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.