DWORF gene therapy for heart failure and muscular dystrophy
Official title: Developing DWORF gene therapy to treat heart failure and muscular dystrophy
Funded at Cincinnati Childrens Hosp Med Ctr · NIH-11241166 · Data updated 2026-11-12
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.
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 | 2026 |
| Award (this year) | $581,971 |
| NIH project number | 5R01HL171221-03 |
| Project ID | NIH-11241166 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with heart failure or genetic forms of muscular dystrophy that affect heart and skeletal muscle would be the likely candidates for this approach.
✕Less likely to help
People whose condition is not driven by calcium handling problems, young children, or those who cannot receive viral gene therapy may not benefit from this approach.
What this research studies
If you have heart failure or muscular dystrophy, researchers aim to develop a one-time gene treatment that delivers the DWORF gene to heart and skeletal muscle using a harmless viral carrier. They will optimize the viral delivery, dosing, and safety in laboratory studies and animal models before any human use. The team will measure whether DWORF improves calcium handling inside muscle cells and protects against muscle damage and weak heart contractions. Work is focused on steps needed to move this approach toward future clinical testing in patients.
Why it matters
Potential benefit: If successful, the therapy could improve heart pumping and reduce muscle degeneration by restoring normal calcium control in muscle cells.
How similar studies have gone: AAV gene delivery has helped other genetic diseases and boosting SERCA activity showed benefit in animal studies, but delivering DWORF is a newer, mainly preclinical approach.
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
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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.