CRISPR-based gene editing therapy for Duchenne muscular dystrophy using AAV delivery
Official title: CRISPR editing therapy for Duchenne muscular dystrophy
Funded at University of Missouri-Columbia · NIH-11351023 · Data updated 2026-11-12
A scientist is testing a gene-editing approach to treat Duchenne muscular dystrophy by delivering CRISPR components with an AAV vector to muscle tissue.
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 | 2026 |
| Award (this year) | $543,960 |
| NIH project number | 5R01NS131416-04 |
| Project ID | NIH-11351023 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with Duchenne muscular dystrophy who have genetic mutations amenable to CRISPR-based correction and are able to participate in preclinical to translational studies.
✕Less likely to help
Patients with non-DMD conditions or without the specific genetic target would not directly benefit from this project.
What this research studies
Duchenne muscular dystrophy is caused by mutations in the dystrophin gene, and this project uses CRISPR/Cas9 to repair the gene. Because DMD affects the entire body, the researchers aim to deliver the therapy systemically through AAV vectors that can reach many muscles. They are studying how to minimize immune responses and off-target edits while preserving long-term expression of the editing machinery. The work includes preclinical studies in canine models to better predict outcomes in humans before any trials. This grant supports a translational path from concept toward potential human use, focusing on safety, fidelity, and meaningful muscle restoration.
Why it matters
Potential benefit: If successful, this research could lead to a one-time gene-editing therapy that improves muscle function for people with DMD.
How similar studies have gone: Previous studies in mouse models show potential, but canine studies are used here to bridge to human applicability, making outcomes promising but still early.
Where this research is happening
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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.