Improving gene delivery for therapy by overcoming cellular barriers
Official title: Intracellular Trafficking of DNA for Gene Therapy
Funded at University of Rochester · NIH-10932914 · Data updated 2026-11-12
This study is looking at ways to make it easier for gene therapy to work by figuring out how to help DNA get into cells and reach the part of the cell where it needs to go, which could lead to better treatments for people who need gene therapy.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Rochester NIH-funded |
| Lab location | 1 site (Rochester, United States) |
| Project start | September 22, 2023 |
| Project end | August 31, 2027 |
| Funding year | 2024 |
| Award (this year) | $398,090 |
| NIH project number | 5R01GM151450-02 |
| Project ID | NIH-10932914 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with genetic conditions that could benefit from gene therapy.
✕Less likely to help
Patients with conditions that do not involve genetic components or those who are not candidates for gene therapy may not benefit from this research.
What this research studies
This research focuses on enhancing the delivery of genes into cells, which is crucial for effective gene therapy. It investigates the various barriers that prevent successful gene transfer, including the need for DNA to cross the cell membrane, navigate through the cytoplasm, and enter the nucleus. The study aims to identify mechanisms that can facilitate this process, particularly through the use of specific DNA sequences that help target the nucleus. By understanding how certain proteins interact with DNA during this journey, the research seeks to improve the efficiency of gene therapy treatments.
Why it matters
Potential benefit: If successful, this research could lead to more effective gene therapies for a variety of genetic disorders.
How similar studies have gone: Previous research has shown promise in enhancing gene delivery methods, but this approach is exploring novel mechanisms that have not been extensively tested.
Where this research is happening
-
University of Rochester
Rochester, United States Active
Researchers
- Principal investigator: Dean, David a — University of Rochester
- Study coordinator: Dean, David a
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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.