Extracellular vesicles to monitor and treat Duchenne muscular dystrophy
(Project 3) Extracellular Vesicles in Monitoring and Treatment of Dystrophinopathy
This project uses tiny particles called extracellular vesicles to track disease activity and help deliver treatments for boys and young men with Duchenne muscular dystrophy.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Research Inst Nationwide Children's Hosp NIH-funded |
| Lab location | 1 site (Columbus, United States) |
| Project ID | NIH-11169994 on NIH RePORTER |
What this research studies
This project will look at extracellular vesicles—small particles released into the blood—as a non‑invasive way to follow how Duchenne muscular dystrophy (DMD) changes over time and how well gene therapies are working. Researchers plan to collect blood samples repeatedly to measure vesicle markers that might reflect muscle health and compare those markers to standard clinical outcome measures. They will also explore whether vesicles can carry therapeutic molecules to muscle cells in lab models to improve or prolong the effects of gene therapies. The work aims to make monitoring easier for patients and to inform safer, more effective gene treatment strategies.
Who could benefit from this research
Good fit: Ideal candidates are boys and young men with genetically confirmed Duchenne muscular dystrophy, including those who have received or may receive gene therapy.
Not a fit: People without DMD, or patients unwilling to provide blood samples or attend follow-up visits, are unlikely to benefit from this project.
Why it matters
Potential benefit: If successful, this could make DMD monitoring easier and less invasive and help improve the safety and durability of gene therapies.
How similar studies have performed: Using blood biomarkers and extracellular vesicles in DMD is an emerging approach with some promising early results but not yet proven in large clinical studies.
Where this research is happening
Columbus, United States
- Research Inst Nationwide Children's Hosp — Columbus, United States (Active)
Researchers
- Principal investigator: Saad, Nizar — Research Inst Nationwide Children's Hosp
- Study coordinator: Saad, Nizar
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.