Understanding the genetic mechanisms of Duchenne muscular dystrophy and developing new therapies
Three-model platform for understanding DMD epigenetic mechanisms and advancing small molecule therapies
This study is looking at Duchenne muscular dystrophy (DMD) to understand how problems in muscle development start the disease and to find new medicines that could fix these early issues, which might lead to better treatments for those living with DMD.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Seattle Children's Hospital NIH-funded |
| Lab location | 1 site (Seattle, United States) |
| Project ID | NIH-10994141 on NIH RePORTER |
What this research studies
This research investigates Duchenne muscular dystrophy (DMD), a severe muscle disease caused by mutations in the dystrophin gene. It aims to explore how early defects in muscle development contribute to the disease and to identify new pharmacological therapies that can target these early changes. By using human induced pluripotent stem cells, the research seeks to understand the epigenetic factors involved in DMD and how small molecules can potentially correct these issues. This approach may lead to more effective treatments that address the underlying causes of DMD rather than just managing symptoms.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Duchenne muscular dystrophy, particularly those in the early stages of the disease.
Not a fit: Patients with other forms of muscular dystrophy or those who are not diagnosed with DMD may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to innovative therapies that significantly improve muscle function and quality of life for patients with DMD.
How similar studies have performed: Previous research has shown promise in using epigenetic approaches for other genetic disorders, suggesting potential success for this novel approach in DMD.
Where this research is happening
Seattle, United States
- Seattle Children's Hospital — Seattle, United States (Active)
Researchers
- Principal investigator: Maves, Lisa — Seattle Children's Hospital
- Study coordinator: Maves, Lisa
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.