Enhancing gene therapy effectiveness using natural cell transport mechanisms
Improving the reach and efficacy of gene therapy by in vivo EV targeting
['FUNDING_R21'] · EMORY UNIVERSITY · NIH-11035914
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
| Phase | ['FUNDING_R21'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | EMORY UNIVERSITY (nih funded) |
| Locations | 1 site (ATLANTA, UNITED STATES) |
| Trial ID | NIH-11035914 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: 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.
Not a fit: Patients with acquired conditions or those over the age of 11 may not benefit from this research.
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 performed: 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
ATLANTA, UNITED STATES
- EMORY UNIVERSITY — ATLANTA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: FRIDOVICH-KEIL, JUDITH L. — EMORY UNIVERSITY
- Study coordinator: FRIDOVICH-KEIL, JUDITH L.
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.