Durable gene edit to restore dystrophin in Duchenne muscular dystrophy
Official title: Durability of a gene editing therapy that restores dystrophin in a humanized mouse model of Duchenne muscular dystrophy
Funded at Myogene Bio, LLC · NIH-11178699 · Data updated 2026-11-12
A gene-editing approach aims to restore dystrophin for people with Duchenne muscular dystrophy by removing DMD exons 45–55 to produce a long-lasting fix.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Myogene Bio, LLC NIH-funded |
| Lab location | 1 site (San Diego, UNITED STATES) |
| Project start | September 1, 2024 |
| Project end | August 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $201,250 |
| NIH project number | 5R21TR005162-02 |
| Project ID | NIH-11178699 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with Duchenne muscular dystrophy whose mutations can be corrected by removing DMD exons 45–55 (roughly half of patients) would be the ideal eventual candidates for this type of therapy.
✕Less likely to help
Patients whose DMD mutations are outside the 45–55 region, or those with advanced disease or contraindications to gene therapies, may not benefit from this specific approach.
What this research studies
This project uses a gene-editing therapy designed to remove DMD exons 45–55 so the body can make more normal dystrophin protein. Researchers test the approach in a humanized mouse model that carries the human DMD gene to mimic patient biology. They have seen benefit at 2 months and are now comparing short-term (2 month) versus longer-term (6 month) outcomes, measuring gene edits, how many therapy copies get into cells, dystrophin protein levels, and muscle structure and function. The goal is to learn whether the edits and muscle improvements hold up over time before moving toward clinical testing.
Why it matters
Potential benefit: If successful, this approach could provide a more durable restoration of dystrophin than current gene-replacement methods, potentially leading to longer-lasting muscle preservation and slower disease progression.
How similar studies have gone: Related preclinical exon-skipping and gene-editing approaches have shown promise in animal models, but durable effects in human-compatible systems and clinical translation remain largely unproven.
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.