Understanding genes that change Duchenne Muscular Dystrophy
Genetic modifiers of Duchenne Muscular Dystrophy
This project looks at genetic differences in people with Duchenne muscular dystrophy to understand why the disease affects them differently.
Quick facts
| Grant type | R01 grant |
|---|---|
| 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-11164582 on NIH RePORTER |
What this research studies
Duchenne muscular dystrophy is a serious muscle condition that causes muscle weakness and loss of movement. We know that mutations in a specific gene cause DMD, but how severe the disease becomes can vary greatly among individuals. This project uses a special database of information and DNA samples from over 900 patients with DMD to find other genes that might influence how the disease progresses. By studying these "modifier genes," we hope to learn why some patients experience more severe symptoms than others.
Who could benefit from this research
Good fit: This project uses existing data and samples from individuals previously diagnosed with Duchenne muscular dystrophy.
Not a fit: Patients not diagnosed with Duchenne muscular dystrophy would not directly benefit from this specific genetic analysis.
Why it matters
Potential benefit: If successful, this work could help us understand why Duchenne muscular dystrophy affects people differently and potentially lead to new ways to predict or slow down disease progression.
How similar studies have performed: Previous work has shown that one gene, LTBP4, can influence the age at which patients with DMD lose the ability to walk, suggesting that other genetic modifiers also exist.
Where this research is happening
Columbus, United States
- Research Inst Nationwide Children's Hosp — Columbus, United States (Active)
Researchers
- Principal investigator: Flanigan, Kevin M — Research Inst Nationwide Children's Hosp
- Study coordinator: Flanigan, Kevin M
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.