Whole-body AAV microdystrophin gene therapy for Duchenne muscular dystrophy
Whole body single AAV microgene in canine DMD
A single viral gene treatment aims to deliver a small working version of the dystrophin gene throughout the body to help people with Duchenne muscular dystrophy.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Missouri-Columbia NIH-funded |
| Lab location | 1 site (Columbia, United States) |
| Project ID | NIH-11362624 on NIH RePORTER |
What this research studies
Duchenne muscular dystrophy (DMD) is caused by loss of dystrophin and this project uses an engineered adeno-associated virus (AAV) to carry a shortened but functional microdystrophin gene. Researchers test systemic, one-time delivery in a large dystrophic dog model to measure bodywide dystrophin expression, muscle and heart benefits, and durability. The team is working to reduce risks seen in human trials such as high-dose AAV toxicity and immune reactions and to improve the microdystrophin design for better function. Findings from the canine work are intended to guide safer, more effective human gene therapy approaches.
Who could benefit from this research
Good fit: People with genetically confirmed Duchenne muscular dystrophy who might be candidates for systemic microdystrophin gene therapy in future clinical trials.
Not a fit: Patients with pre-existing strong antibodies to the AAV vector, very advanced disease with irreversible muscle loss, or those not eligible for systemic gene therapy may not benefit from this approach.
Why it matters
Potential benefit: If successful, this work could lead to safer, longer-lasting whole-body gene therapy that preserves muscle and heart function in people with DMD.
How similar studies have performed: Related AAV microdystrophin approaches have shown benefit in animal models and early human trials (and one product received FDA approval), but high-dose toxicity and immune responses remain unresolved challenges.
Where this research is happening
Columbia, United States
- University of Missouri-Columbia — Columbia, United States (Active)
Researchers
- Principal investigator: Duan, Dongsheng — University of Missouri-Columbia
- Study coordinator: Duan, Dongsheng
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.