CRISPR gene editing for Duchenne muscular dystrophy
Official title: CRISPR editing therapy for Duchenne muscular dystrophy
Funded at University of Missouri-Columbia · NIH-11175406 · Data updated 2026-11-12
This project explores using CRISPR gene technology to correct the genetic cause of 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 start | July 15, 2023 |
| Project end | June 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $556,979 |
| NIH project number | 5R01NS131416-03 |
| Project ID | NIH-11175406 on NIH RePORTER |
Who could benefit from this research
✓Could help
Future patients with Duchenne muscular dystrophy who could benefit from gene-editing treatments might be ideal candidates for this type of therapy.
✕Less likely to help
Patients without Duchenne muscular dystrophy or those with other forms of muscular dystrophy would not directly benefit from this specific gene-editing approach.
What this research studies
Duchenne muscular dystrophy (DMD) is a genetic condition that causes muscles throughout the body to weaken over time. This research aims to use CRISPR/Cas9 gene editing to fix the faulty gene responsible for DMD. To deliver this gene-editing tool to all muscles, scientists are using a special virus called AAV, which can reach muscles throughout the body. A key challenge is ensuring the CRISPR system works effectively without causing unwanted side effects or being rejected by the body, especially since the Cas9 protein can trigger an immune response. This project uses canine models, which are very similar to humans, to understand and overcome these challenges, paving the way for future human treatments.
Why it matters
Potential benefit: If successful, this approach could offer a lasting treatment for Duchenne muscular dystrophy by fixing the underlying genetic problem.
How similar studies have gone: Early studies in mouse models have shown promising results with similar CRISPR approaches, leading to improved muscle function and long-term Cas9 expression.
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.