Targeting specific brain cells for gene delivery and reprogramming
Official title: Selective targeting of CNS cells in vivo for multiple transgene delivery and neuronal reprogramming
Funded at Rosalind Franklin Univ of Medicine & Sci · NIH-11039998 · Data updated 2026-11-12
This study is looking at ways to make sure that gene therapies can safely and effectively deliver genetic material to specific brain cells, like astrocytes, without affecting other types of cells, so that treatments for brain conditions can work better and more reliably.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Rosalind Franklin Univ of Medicine & Sci NIH-funded |
| Lab location | 1 site (North Chicago, United States) |
| Project start | February 20, 2024 |
| Project end | March 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $197,375 |
| NIH project number | 5R21NS135301-02 |
| Project ID | NIH-11039998 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals aged 21 and older who have conditions affecting the central nervous system and are interested in novel gene therapy approaches.
✕Less likely to help
Patients with conditions unrelated to the central nervous system or those under 21 years old may not benefit from this research.
What this research studies
This research focuses on improving the delivery of genetic material to specific cell types in the central nervous system (CNS) using viral vectors. The goal is to enhance the selectivity of these vectors to ensure they only infect the intended cells, such as astrocytes, while avoiding unintended infections of other cell types like neurons. By achieving this, the researchers aim to reduce variability in study outcomes and improve the effectiveness of gene therapies. This project will explore innovative methods to enable precise targeting and subsequent gene delivery to previously infected cells.
Why it matters
Potential benefit: If successful, this research could lead to more effective gene therapies for neurological conditions by ensuring that treatments are delivered precisely to the right cells.
How similar studies have gone: While the use of viral vectors for gene delivery is well-established, this specific approach to enhance cell-type selectivity is innovative and has not been extensively tested in prior studies.
Where this research is happening
-
Rosalind Franklin Univ of Medicine & Sci
North Chicago, United States Active
Researchers
- Principal investigator: Peterson, Daniel Alan — Rosalind Franklin Univ of Medicine & Sci
- Study coordinator: Peterson, Daniel Alan
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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.