Evaluating a gene editing therapy for Duchenne muscular dystrophy
Official title: Further assessment of durability of a gene editing therapy for Duchenne muscular dystrophy
Funded at Myogene Bio, LLC · NIH-11066316 · Data updated 2026-11-12
This study is testing a new gene editing treatment for Duchenne muscular dystrophy that aims to fix the gene responsible for the condition, and it's designed to help people with this muscle-wasting disease by improving their muscle function over time.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Myogene Bio, LLC NIH-funded |
| Lab location | 1 site (San Diego, UNITED STATES) |
| Project start | August 1, 2019 |
| Project end | April 30, 2027 |
| Funding year | 2025 |
| Award (this year) | $1,321,604 |
| NIH project number | 9R44NS141438-04 |
| Project ID | NIH-11066316 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 deletions in exons 45-55 of the dystrophin gene.
✕Less likely to help
Patients with forms of muscular dystrophy other than Duchenne or those without the specific genetic deletions targeted by this therapy may not benefit from this research.
What this research studies
This research investigates a gene editing therapy designed to treat Duchenne muscular dystrophy by permanently editing the patient's DNA to restore the reading frame of the dystrophin gene. The therapy utilizes a method called CRISPR/Cas9 delivered through adeno-associated viruses to target specific exons in the gene. The effectiveness and safety of this approach will be assessed in humanized mouse models, focusing on muscle function and molecular markers over different time periods and dosing regimens. The findings aim to provide insights into the long-term benefits of gene editing for patients with this condition.
Why it matters
Potential benefit: If successful, this research could lead to a durable treatment option that significantly improves muscle function and quality of life for patients with Duchenne muscular dystrophy.
How similar studies have gone: Previous research has shown promising results with gene editing approaches in animal models, indicating potential for success in human applications.
Where this research is happening
-
Myogene Bio, LLC
San Diego, United States Active
Researchers
- Principal investigator: Young, Courtney S — Myogene Bio, LLC
- Study coordinator: Young, Courtney S
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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.