Developing tools for targeted gene delivery in brain cells across different species
Engineered AAV Identification, Validation, and Dissemination Pipeline for Brain Cell Type-Specific Manipulation Across Species
This study is working on special viruses that can safely deliver genes to specific brain cells, helping scientists better understand how the brain works and how to treat brain disorders, with the goal of making these tools available for research in both animals and humans.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | California Institute of Technology NIH-funded |
| Lab location | 1 site (Pasadena, United States) |
| Project ID | NIH-10350260 on NIH RePORTER |
What this research studies
This research focuses on creating specialized adeno-associated viruses (AAVs) that can deliver genes to specific types of brain cells in a non-invasive manner. By characterizing these engineered AAVs, the team aims to enhance our understanding of brain function and disorders through precise anatomical and functional analysis. The project will involve collaboration with the neuroscience community to ensure these tools are widely accessible and useful for various research applications. Ultimately, this initiative seeks to improve the ability to monitor and manipulate brain activity across species, including humans.
Who could benefit from this research
Good fit: Ideal candidates for participation or benefit from this research would include individuals with neurological conditions that could be addressed through gene therapy.
Not a fit: Patients with conditions unrelated to brain function or those who do not meet specific eligibility criteria for gene therapy may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to advanced methods for studying and treating brain disorders by enabling targeted gene therapies.
How similar studies have performed: Previous research has shown promise in using engineered AAVs for targeted gene delivery, indicating that this approach has potential for success.
Where this research is happening
Pasadena, United States
- California Institute of Technology — Pasadena, United States (Active)
Researchers
- Principal investigator: Gradinaru, Viviana — California Institute of Technology
- Study coordinator: Gradinaru, Viviana
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.