Improving gene editing techniques for treating muscle diseases
Official title: Technologies to enable safe and effective gene editing in skeletal muscle
Funded at University of Florida · NIH-10999595 · Data updated 2026-11-12
This study is working on improving gene editing techniques to help treat muscle diseases by finding better ways to deliver the treatment directly to muscle cells, making it safer and more effective for patients.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Florida NIH-funded |
| Lab location | 1 site (Gainesville, United States) |
| Project start | September 10, 2024 |
| Project end | July 31, 2029 |
| Funding year | 2024 |
| Award (this year) | $531,109 |
| NIH project number | 1R01AR084841-01 |
| Project ID | NIH-10999595 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with genetic muscle disorders who may benefit from advanced gene editing therapies.
✕Less likely to help
Patients with non-genetic muscle conditions or those who do not have skeletal muscle diseases may not benefit from this research.
What this research studies
This research focuses on enhancing gene editing methods specifically for skeletal muscle diseases using adeno-associated viruses (AAV). The team aims to overcome significant challenges in delivering gene editing tools effectively to muscle tissues, ensuring that these tools can reach and modify the necessary cells without causing prolonged effects. By developing innovative strategies to maximize the delivery and control of gene editing enzymes, the research seeks to create safer and more efficient treatments for patients with muscle disorders.
Why it matters
Potential benefit: If successful, this research could lead to more effective and safer gene therapies for patients suffering from skeletal muscle diseases.
How similar studies have gone: Previous research has shown promise in using AAV for gene delivery, indicating that this approach has potential for success in treating muscle diseases.
Where this research is happening
-
University of Florida
Gainesville, United States Active
Researchers
- Principal investigator: Wang, Eric T — University of Florida
- Study coordinator: Wang, Eric T
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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.