Getting therapeutic DNA to the right place inside cells
Intracellular Trafficking of DNA for Gene Therapy
This research looks at how delivered DNA travels inside cells so gene treatments can work better for people with genetic or chronic conditions.
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 ID | NIH-11176981 on NIH RePORTER |
What this research studies
As a patient, this work is trying to understand why lab-made or viral DNA often fails to reach a cell's nucleus and how to overcome those roadblocks. The team studies how DNA crosses the cell membrane, moves through the cytoplasm on cytoskeletal tracks, and enters the nucleus, focusing on molecules like transcription factors and innate DNA sensors. They use cell and animal models and engineered DNA sequences to see what helps DNA bind the right proteins and ride cellular transport systems. The goal is to design better DNA delivery methods that increase the chances gene therapies will turn on the correct genes in target tissues.
Who could benefit from this research
Good fit: People with genetic disorders or chronic conditions that might be treated by gene therapy—especially those requiring delivery into non-dividing cells like muscle or nerve cells—would be the eventual candidates for therapies enabled by this work.
Not a fit: Patients with conditions unrelated to gene-based treatments or those needing immediate clinical care are unlikely to benefit directly from this basic laboratory research in the near term.
Why it matters
Potential benefit: If successful, this could make gene therapies more reliable and effective for more people by improving how therapeutic DNA reaches and works inside patient cells.
How similar studies have performed: Some gene therapies have succeeded clinically for specific diseases, but the detailed intracellular trafficking strategies this project targets remain mostly at the laboratory and early preclinical stage with limited clinical translation so far.
Where this research is happening
Rochester, United States
- University of Rochester — Rochester, United States (Active)
Researchers
- Principal investigator: Dean, David a — University of Rochester
- Study coordinator: Dean, David a
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.