Understanding how FXR1 affects muscle health and disease
Deciphering the roles of FXR1 in health and myopathy
This study is looking at a protein called FXR1 to see how it affects muscle health and function, especially in people with Duchenne Muscular Dystrophy (DMD), and it hopes to find ways to improve muscle strength by restoring FXR1 levels.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Icahn School of Medicine at Mount Sinai NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-10900652 on NIH RePORTER |
What this research studies
This research investigates the role of FXR1, an RNA-binding protein, in muscle function and disease progression, particularly in conditions like Duchenne Muscular Dystrophy (DMD). By studying animal models, the research aims to uncover how FXR1 regulates important mRNAs that are crucial for muscle contraction and overall muscle health. The team will explore the effects of restoring FXR1 levels in various models of DMD to assess potential improvements in muscle structure and function. This could lead to new insights into treatments for muscle-related diseases.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Duchenne Muscular Dystrophy or related muscle disorders.
Not a fit: Patients with muscle disorders not related to FXR1 or those without a muscular dystrophy diagnosis may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapeutic strategies for improving muscle function in patients with myopathies, including Duchenne Muscular Dystrophy.
How similar studies have performed: Previous research has shown promising results in restoring FXR1 levels in animal models, indicating potential for success in similar therapeutic approaches.
Where this research is happening
New York, United States
- Icahn School of Medicine at Mount Sinai — New York, United States (Active)
Researchers
- Principal investigator: Gregorio, Carol C — Icahn School of Medicine at Mount Sinai
- Study coordinator: Gregorio, Carol C
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.