C2 domain therapies for muscular dystrophy
Official title: C2 domain therapeutics for muscular dystrophy
Funded at University of Maryland Baltimore · NIH-11345565 · Data updated 2026-11-12
A patient-friendly look at a lab-driven approach to help people with dysferlin-related muscular dystrophy through targeted C2 domain proteins.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Maryland Baltimore NIH-funded |
| Lab location | 1 site (Baltimore, United States) |
| Project start | July 30, 2024 |
| Project end | June 30, 2029 |
| Funding year | 2026 |
| Award (this year) | $507,581 |
| NIH project number | 5R01AR082469-03 |
| Project ID | NIH-11345565 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with LGMD 2B or Miyoshi Myopathy caused by DYSF mutations, who may benefit from future translation of this research.
✕Less likely to help
Patients without DYSF-related muscular dystrophy are unlikely to benefit.
What this research studies
This project focuses on how a protein domain called C2A within dysferlin helps repair muscle membranes and support calcium signaling, which are disrupted in certain muscular dystrophies. Researchers are exploring whether a chimeric protein that combines C2A with another C2 domain can localize correctly in muscle cells and restore function. The work uses cell and animal models to study membrane repair and calcium handling, aiming to translate these findings into potential therapies for LGMD 2B and Miyoshi Myopathy. If successful, this could lead to new strategies to protect muscle fibers and improve strength and endurance for patients.
Why it matters
Potential benefit: If the approach succeeds, it could pave the way for therapies that improve muscle repair and calcium signaling in people with dysferlin-related muscular dystrophies.
How similar studies have gone: Previous work shows C2A has unique activities and that TJ-targeting strategies can influence localization and function, suggesting potential for this approach.
Where this research is happening
-
University of Maryland Baltimore
Baltimore, United States Active
Researchers
- Principal investigator: Bloch, Robert J — University of Maryland Baltimore
- Study coordinator: Bloch, Robert J
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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.