Human lab-grown muscle model for LGMD2B
Engineering a Human Skeletal Muscle Tissue Model of LGMD2B
Creating 3D human muscle tissue from patient cells to mirror LGMD2B and help find treatments for people with dysferlin-related muscle weakness.
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-11158687 on NIH RePORTER |
What this research studies
Researchers will grow 3D 'myobundles' from induced pluripotent stem cells (iPSCs) taken from three people with LGMD2B and three healthy donors. These engineered muscles will be tested for contractile strength, calcium handling, and abnormal fat accumulation to reproduce features seen in patients. The team will study muscle interstitial cells that may turn into fat and compare drug responses with results from dysferlin-deficient mice and patient data. The goal is a reproducible human model that can be used to screen therapies and study why muscles fail in LGMD2B.
Who could benefit from this research
Good fit: Ideal participants would be people diagnosed with LGMD2B who can provide a small tissue or blood sample to create iPSC lines or who have previously donated samples to a biobank.
Not a fit: Patients needing immediate clinical treatment or those with other forms of muscular dystrophy are unlikely to receive direct clinical benefit from this lab-modeling project in the short term.
Why it matters
Potential benefit: If successful, this model could speed discovery of treatments and provide a human platform for testing drugs tailored to LGMD2B.
How similar studies have performed: Lab-grown human muscle models have shown useful disease features and drug responses in other muscular dystrophies, but applying a 3D myobundle specifically to LGMD2B is a newer effort.
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.