Enhancing gene therapy effectiveness using natural cell transport mechanisms
Official title: Improving the reach and efficacy of gene therapy by in vivo EV targeting
Funded at Emory University · NIH-11035914 · Data updated 2026-11-12
This study is exploring a new way to help kids with inherited disorders by using tiny particles from the body to deliver helpful genes or proteins more effectively, aiming to make treatments work better than current methods.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Emory University NIH-funded |
| Lab location | 1 site (Atlanta, United States) |
| Project start | June 1, 2025 |
| Project end | May 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $430,375 |
| NIH project number | 1R21HD117191-01 |
| Project ID | NIH-11035914 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children under 11 years old with genetic disorders that are currently difficult to treat with existing AAV-based therapies.
✕Less likely to help
Patients with acquired conditions or those over the age of 11 may not benefit from this research.
What this research studies
This research aims to improve gene therapy for inherited disorders in children by utilizing naturally occurring extracellular vesicles (EVs) to deliver therapeutic mRNA or proteins more effectively. The approach focuses on overcoming limitations of current adeno-associated virus (AAV) therapies, which often fail to adequately transduce enough cells or maintain gene expression over time. By harnessing EVs, the research seeks to enable better distribution of therapeutic agents among cells and across tissues, potentially improving treatment outcomes for patients with genetic conditions.
Why it matters
Potential benefit: If successful, this research could lead to more effective gene therapies that provide lasting benefits for children with inherited disorders.
How similar studies have gone: While the use of EVs in gene therapy is a novel approach, preliminary studies have shown promise in enhancing the delivery of therapeutic agents.
Where this research is happening
-
Emory University
Atlanta, United States Active
Researchers
- Principal investigator: Fridovich-Keil, Judith L. — Emory University
- Study coordinator: Fridovich-Keil, Judith L.
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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.