Gene editing therapy for Duchenne muscular dystrophy
CRISPR editing therapy for Duchenne muscular dystrophy
This study is exploring a new way to treat Duchenne muscular dystrophy (DMD) using a special gene editing tool called CRISPR, delivered through a virus to help all muscles in dogs, so we can see how well it works and if it's safe before considering it for people.
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-10881958 on NIH RePORTER |
What this research studies
This research investigates the use of CRISPR/Cas9 gene editing to treat Duchenne muscular dystrophy (DMD) by targeting the genetic mutations responsible for the disease. The approach involves using an adeno-associated virus (AAV) vector to deliver the gene editing tools throughout the body, ensuring that all muscles receive the treatment. The study aims to assess the effectiveness and safety of this method in canine models, which are more representative of human DMD than traditional mouse models. By monitoring the immune response to the gene editing components, the researchers hope to improve the therapy's efficacy and reduce potential side effects.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Duchenne muscular dystrophy, particularly those with specific genetic mutations in the dystrophin gene.
Not a fit: Patients with forms of muscular dystrophy other than Duchenne muscular dystrophy may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment that addresses the root cause of Duchenne muscular dystrophy, potentially improving muscle function and quality of life for patients.
How similar studies have performed: Previous research using CRISPR and AAV vectors has shown promise in animal models, indicating potential for success in human applications.
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.