Using extracellular vesicles to monitor and treat Duchenne Muscular Dystrophy
(Project 3) Extracellular Vesicles in Monitoring and Treatment of Dystrophinopathy
This study is looking at ways to track how Duchenne Muscular Dystrophy affects boys over time and to see how well new gene therapies are working, using tiny particles from cells to make the process easier and more helpful for patients.
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-10992528 on NIH RePORTER |
What this research studies
This research focuses on Duchenne Muscular Dystrophy (DMD), a genetic condition affecting muscle function in boys. It aims to develop non-invasive methods to monitor disease progression and the effectiveness of new gene therapies. By analyzing extracellular vesicles, which are tiny particles released by cells, the researchers hope to gain insights into how well treatments are working over time. This approach could simplify the monitoring process for patients and provide valuable information for future therapies.
Who could benefit from this research
Good fit: Ideal candidates for this research are boys diagnosed with Duchenne Muscular Dystrophy.
Not a fit: Patients with other forms of muscular dystrophy or those without a diagnosis of Duchenne Muscular Dystrophy may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to improved monitoring of treatment effects in DMD, potentially accelerating the development of effective therapies.
How similar studies have performed: Previous research has shown promise in using similar non-invasive monitoring techniques in other conditions, suggesting potential success in this area as well.
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.