Gene editing therapy for Duchenne muscular dystrophy
Official title: CRISPR editing therapy for Duchenne muscular dystrophy
Funded at University of Missouri-Columbia · NIH-10881958 · Data updated 2026-11-12
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 start | July 15, 2023 |
| Project end | June 30, 2028 |
| Funding year | 2024 |
| Award (this year) | $552,403 |
| NIH project number | 5R01NS131416-02 |
| Project ID | NIH-10881958 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 genetic mutations in the dystrophin 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 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.
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 gone: 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
-
University of Missouri-Columbia
Columbia, United States Active
Researchers
- Principal investigator: Duan, Dongsheng — University of Missouri-Columbia
- Study coordinator: Duan, Dongsheng
Related research
Other NIH-funded projects on the same topics.
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Advancing manufacturing and release testing for a Duchenne gene editing therapy
A project to optimize how a dual-vector AAV gene editing treatment for Duchenne muscular dystrophy is made and tested, so it can move toward clinical use.
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Understanding why Duchenne Muscular Dystrophy worsens and how to stop it
A research team at USC is studying Duchenne muscular dystrophy to find out why the disease continues to progress and how to interrupt harmful changes in muscle tissue.
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Stopping steroid-related muscle and bone wasting in Duchenne muscular dystrophy
Researchers are testing ways to protect muscles and bones in people with Duchenne muscular dystrophy who take long-term steroid medicines.
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MR clues to how nNOS affects energy use and blood flow in Becker and Duchenne muscular dystrophy
This project looks at how advanced MRI tests can track muscle energy and blood flow changes in people with Becker and Duchenne muscular dystrophy as new gene therapies are explored.
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Digital motor-skill tracking for infants with Duchenne Muscular Dystrophy using wearables
A wearable-sensor project aims to quietly track and quantify how motor skills develop in babies and young boys with Duchenne Muscular Dystrophy.
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Genetic factors that influence Duchenne Muscular Dystrophy progression
A study to find genes that change how severe Duchenne muscular dystrophy is in boys and young men, using patient data and DNA samples.
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.