Using DWORF gene therapy to improve heart function and treat muscular dystrophy
Developing DWORF gene therapy to treat heart failure and muscular dystrophy
['FUNDING_R01'] · CINCINNATI CHILDRENS HOSP MED CTR · NIH-10999404
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
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | CINCINNATI CHILDRENS HOSP MED CTR (nih funded) |
| Locations | 1 site (CINCINNATI, UNITED STATES) |
| Trial ID | NIH-10999404 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals aged 21 and older who are diagnosed with heart failure or muscular dystrophy.
Not a fit: Patients with conditions unrelated to calcium dysregulation in muscle function may not benefit from this research.
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 performed: 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, UNITED STATES
- 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
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.