Gene editing therapy 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-10938513 · Data updated 2026-11-12
This study is testing a new gene editing treatment for Duchenne muscular dystrophy that uses CRISPR technology to fix the genetic problems causing the disease, and it's being done in mice to see how well it works over a longer time, which could lead to better options for patients in the future.
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 | 2024 |
| Award (this year) | $110,000 |
| NIH project number | 1R21TR005162-01 |
| Project ID | NIH-10938513 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 mutations that can be targeted by the gene editing approach.
✕Less likely to help
Patients with forms of muscular dystrophy that are not caused by the specific mutations targeted in this research may not benefit.
What this research studies
This research investigates a gene editing therapy aimed at treating Duchenne muscular dystrophy (DMD) by correcting specific genetic mutations. The approach utilizes CRISPR technology to remove certain exons from the DMD gene, which is expected to restore the production of dystrophin, a crucial protein for muscle function. The therapy is being tested in a humanized mouse model to evaluate its long-term effectiveness and durability over a six-month period compared to shorter two-month assessments. Patients may benefit from a more stable and effective treatment option that addresses the underlying genetic cause of their condition.
Why it matters
Potential benefit: If successful, this research could lead to a more effective and durable treatment for patients with Duchenne muscular dystrophy.
How similar studies have gone: Other research using CRISPR technology for gene editing in muscular dystrophy has shown promising results, indicating potential for success in this approach.
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.