Developing precise methods to target specific brain cells using advanced genetic techniques
Official title: Enhanced Genetic Targeting of Specific Neuronal Populations Using a Minipromoter and Splicing Hybrid Approach
Funded at Tufts University Boston · NIH-10987600 · Data updated 2026-11-12
This study is working on a new way to deliver genetic material directly to specific brain cells, which could help create more precise treatments for neurological conditions, making it easier for patients to get the right therapy for their needs.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Tufts University Boston NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | July 1, 2024 |
| Project end | June 30, 2026 |
| Funding year | 2024 |
| Award (this year) | $468,126 |
| NIH project number | 1R21NS135454-01A1 |
| Project ID | NIH-10987600 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research would include individuals with neurological conditions that could benefit from targeted gene therapy.
✕Less likely to help
Patients with conditions unrelated to neuronal targeting or those who do not require genetic interventions may not benefit from this research.
What this research studies
This research focuses on improving the delivery of genetic material to specific types of neurons in the brain using a novel approach that combines advanced viral vectors and splicing techniques. By enhancing the specificity of adeno-associated viruses (AAVs), the project aims to minimize unintended targeting of non-target cells, which is crucial for effective gene therapy. Patients may benefit from this research as it could lead to more precise treatments for neurological conditions by enabling targeted gene expression in specific neuronal populations. The study will involve designing and testing new constructs that can deliver genetic tools more accurately, ultimately contributing to advancements in brain research and therapy.
Why it matters
Potential benefit: If successful, this research could lead to more effective and targeted gene therapies for neurological disorders.
How similar studies have gone: Previous research has shown promise in using similar viral vector approaches for targeted gene delivery, indicating potential for success in this novel application.
Where this research is happening
-
Tufts University Boston
Boston, United States Active
Researchers
- Principal investigator: Bygrave, Alexei Mansfield — Tufts University Boston
- Study coordinator: Bygrave, Alexei Mansfield
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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.