Creating new vectors for gene therapy to treat Duchenne muscular dystrophy
Official title: Development of myotropic human bocavirus vectors for gene therapy
Funded at University of Kansas Medical Center · NIH-11113826 · Data updated 2026-11-12
This study is exploring a new way to use a special virus to deliver helpful genes to people with Duchenne muscular dystrophy (DMD), with the hope of improving treatments and making life better for those living with this condition.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Kansas Medical Center NIH-funded |
| Lab location | 1 site (Kansas City, United States) |
| Project start | June 12, 2024 |
| Project end | April 30, 2026 |
| Funding year | 2025 |
| Award (this year) | $194,075 |
| NIH project number | 5R21AI182645-02 |
| Project ID | NIH-11113826 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Duchenne muscular dystrophy, particularly those with specific mutations in the DMD gene.
✕Less likely to help
Patients with forms of muscular dystrophy other than Duchenne muscular dystrophy may not benefit from this research.
What this research studies
This research focuses on developing innovative gene therapy vectors to treat Duchenne muscular dystrophy (DMD), a genetic disorder caused by mutations in the DMD gene. The approach involves using a modified adeno-associated virus (AAV) vector that can carry larger genetic material, allowing for more effective delivery of therapeutic genes to both skeletal and cardiac muscles. By utilizing a specific type of virus known as human bocavirus, the researchers aim to enhance the efficiency of gene editing techniques that could potentially correct the underlying genetic mutations in DMD patients. This could lead to improved treatment outcomes for individuals affected by this condition.
Why it matters
Potential benefit: If successful, this research could provide a more effective gene therapy option for patients with Duchenne muscular dystrophy, potentially improving their muscle function and quality of life.
How similar studies have gone: Previous research has shown promise in using modified AAV vectors for gene therapy, indicating a potential for success with this novel approach.
Where this research is happening
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University of Kansas Medical Center
Kansas City, United States Active
Researchers
- Principal investigator: Qiu, Jianming — University of Kansas Medical Center
- Study coordinator: Qiu, Jianming
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.