Creating a human muscle tissue model for LGMD2B
Engineering a Human Skeletal Muscle Tissue Model of LGMD2B
This study is creating a special 3D model of human muscle to help us learn more about limb girdle muscular dystrophy 2B (LGMD2B) and test new treatments, using muscle cells from both healthy people and those with the condition.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Duke University NIH-funded |
| Lab location | 1 site (Durham, United States) |
| Project ID | NIH-10903900 on NIH RePORTER |
What this research studies
This research aims to develop a 3D human skeletal muscle tissue model to better understand and investigate limb girdle muscular dystrophy 2B (LGMD2B), a condition caused by mutations in the dysferlin gene. By using human induced pluripotent stem cells (iPSCs) from both healthy individuals and LGMD2B patients, the researchers will create muscle tissues that mimic the disease's characteristics. This model will allow for patient-specific studies and drug testing, potentially leading to new therapeutic approaches. The project focuses on replicating the muscle's structural and functional features, including how it handles calcium and lipids, which are crucial for muscle health.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with limb girdle muscular dystrophy 2B (LGMD2B) or those who are carriers of dysferlin mutations.
Not a fit: Patients with other forms of muscular dystrophy or unrelated muscle disorders may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that slow the progression of LGMD2B or improve patient outcomes.
How similar studies have performed: While there have been studies on muscular dystrophies, this specific approach of creating a 3D human muscle model for LGMD2B is relatively novel and untested.
Where this research is happening
Durham, United States
- Duke University — Durham, United States (Active)
Researchers
- Principal investigator: Bursac, Nenad — Duke University
- Study coordinator: Bursac, Nenad
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.