Safer, more effective gene therapy for inherited muscle disorders
Technologies to enable safe and effective gene editing in skeletal muscle
This project aims to improve virus-delivered gene editing tools so people with inherited muscle diseases can get safer, more precise treatments.
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 ID | NIH-11190853 on NIH RePORTER |
What this research studies
From a patient point of view, this work is trying to make gene fixes in muscle safer and more reliable. The team is improving AAV virus carriers so editing enzymes reach more of the many nuclei inside muscle fibers, using smaller gene-editing enzymes that fit into a single virus, and building chemical on/off switches so the editors can be turned off after they work. They will test these strategies in labs and animal models and then integrate the best components into single, lower-dose viral packages. The goal is to create approaches that could be translated into human therapies with fewer side effects.
Who could benefit from this research
Good fit: People with inherited skeletal muscle conditions caused by faulty genes (for example certain forms of muscular dystrophy) who might be candidates for future gene-editing therapies are the most relevant group.
Not a fit: Patients with non-genetic muscle problems, those too far progressed for gene correction, or those ineligible for viral delivery methods would be unlikely to benefit directly.
Why it matters
Potential benefit: If successful, this could enable lower-dose, one-time gene corrections for muscle disorders with broader muscle coverage and fewer long-term risks.
How similar studies have performed: Preclinical studies have shown promise for myotropic AAV capsids and miniaturized Cas enzymes, but achieving safe, widespread editing across whole human muscle remains largely unproven and this work combines several novel strategies.
Where this research is happening
Gainesville, United States
- University of Florida — Gainesville, United States (Active)
Researchers
- Principal investigator: Wang, Eric T — University of Florida
- Study coordinator: Wang, Eric T
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.