Using stem cells to treat muscle disorders caused by dystroglycanopathies
Targeting Dystroglycanopathies using Pluripotent-derived Myogenic Progenitors
This study is exploring a new treatment using stem cells to help people with muscle disorders caused by changes in the FKRP gene, aiming to repair damaged muscles and improve their function.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Minnesota NIH-funded |
| Lab location | 1 site (Minneapolis, United States) |
| Project ID | NIH-10995350 on NIH RePORTER |
What this research studies
This research focuses on developing a stem cell-based therapy to address dystroglycanopathies, a group of muscle disorders caused by mutations in the FKRP gene. The approach involves creating myogenic progenitors from induced pluripotent stem cells (iPSCs) that can regenerate damaged muscle tissue. By transplanting these cells into affected muscles, the goal is to restore normal muscle function and potentially provide long-term benefits. This innovative method aims to tackle the underlying cause of muscle wasting associated with these conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with FKRP-associated dystroglycanopathies, including various forms of muscular dystrophy.
Not a fit: Patients with muscle disorders not related to dystroglycanopathies or those with advanced disease stages may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment option for patients suffering from dystroglycanopathies, improving muscle function and quality of life.
How similar studies have performed: While the use of stem cell therapies for muscle disorders is a promising area of research, this specific approach targeting dystroglycanopathies is relatively novel and has not been extensively tested in clinical settings.
Where this research is happening
Minneapolis, United States
- University of Minnesota — Minneapolis, United States (Active)
Researchers
- Principal investigator: Perlingeiro, Rita C. R. — University of Minnesota
- Study coordinator: Perlingeiro, Rita C. R.
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.