Using DWORF gene therapy to improve heart function and treat muscular dystrophy
Official title: Developing DWORF gene therapy to treat heart failure and muscular dystrophy
Funded at Cincinnati Childrens Hosp Med Ctr · NIH-10999404 · Data updated 2026-11-12
This study is looking at how a special protein called DWORF can help improve muscle function by balancing calcium levels, and it may offer a new treatment option for people with heart failure or muscular dystrophy.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cincinnati Childrens Hosp Med Ctr NIH-funded |
| Lab location | 1 site (Cincinnati, United States) |
| Project start | December 11, 2023 |
| Project end | November 30, 2027 |
| Funding year | 2025 |
| Award (this year) | $534,463 |
| NIH project number | 5R01HL171221-02 |
| Project ID | NIH-10999404 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals aged 21 and older who are diagnosed with heart failure or muscular dystrophy.
✕Less likely to help
Patients with conditions unrelated to calcium dysregulation in muscle function may not benefit from this research.
What this research studies
This research focuses on the role of calcium in muscle function and how its dysregulation contributes to heart failure and muscular dystrophy. The study investigates a novel microprotein called DWORF, which enhances the activity of a calcium pump in muscle cells, potentially restoring normal calcium levels and improving muscle function. By using gene therapy to increase DWORF levels, the research aims to protect against the damaging effects of these diseases. Patients may be involved in trials that assess the safety and effectiveness of this innovative treatment approach.
Why it matters
Potential benefit: If successful, this research could lead to new gene therapies that significantly improve heart function and muscle strength in patients with heart failure and muscular dystrophy.
How similar studies have gone: Previous research has shown promising results in enhancing calcium transport in muscle cells, indicating that this approach could be effective.
Where this research is happening
-
Cincinnati Childrens Hosp Med Ctr
Cincinnati, United States Active
Researchers
- Principal investigator: Makarewich, Catherine a — Cincinnati Childrens Hosp Med Ctr
- Study coordinator: Makarewich, Catherine a
Related research
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DWORF gene therapy for heart failure and muscular dystrophy
This project is testing a gene therapy that raises levels of a small protein called DWORF to help adults with heart failure or muscular dystrophy improve heart and muscle function.
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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.