CRISPR gene editing for Duchenne muscular dystrophy
CRISPR editing therapy for Duchenne muscular dystrophy
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 ID | NIH-11175406 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Future patients with Duchenne muscular dystrophy who could benefit from gene-editing treatments might be ideal candidates for this type of therapy.
Not a fit: Patients without Duchenne muscular dystrophy or those with other forms of muscular dystrophy would not directly benefit from this specific gene-editing approach.
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 performed: 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
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.