Developing stem cell therapy for muscular dystrophy
Preclinical studies of pluripotent stem cell-derived myogenic progenitors in non-human primates
This research is creating new stem cell treatments to help muscles regenerate in people with Duchenne Muscular Dystrophy.
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-11140548 on NIH RePORTER |
What this research studies
Muscular dystrophies, like Duchenne Muscular Dystrophy (DMD), cause muscles to weaken and break down over time. Currently, there isn't an effective treatment, but cell-based therapies offer a promising way to help muscles heal and grow stronger. Our team has developed a unique method to create special muscle-building cells from stem cells. These cells have shown great success in repairing muscle tissue in mice with muscular dystrophy. Now, we are testing these stem cell treatments in non-human primates to ensure they are safe and effective before potentially moving to human studies.
Who could benefit from this research
Good fit: This preclinical work is foundational for future therapies that could benefit patients with Duchenne Muscular Dystrophy and similar muscle-wasting conditions.
Not a fit: Patients seeking immediate treatment or direct participation in a human clinical trial would not benefit from this early-stage, non-human primate research.
Why it matters
Potential benefit: If successful, this work could lead to a new cell therapy that helps regenerate damaged muscle tissue and improve muscle function for individuals with muscular dystrophy.
How similar studies have performed: While this specific method of generating therapeutic muscle progenitors is novel, cell-based therapies for muscle regeneration have shown promise in earlier animal models.
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.