A new therapy to correct genetic mutations in Duchenne muscular dystrophy
Official title: Controllable base editing therapy for DMD
Funded at Indiana University Indianapolis · NIH-10909367 · Data updated 2026-11-12
This study is working on a new treatment for Duchenne muscular dystrophy (DMD) that uses a special virus to fix the genetic mistakes causing muscle problems, with the hope of helping patients regain muscle strength and avoid heart issues.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Indiana University Indianapolis NIH-funded |
| Lab location | 1 site (Indianapolis, United States) |
| Project start | September 1, 2023 |
| Project end | August 31, 2027 |
| Funding year | 2024 |
| Award (this year) | $587,533 |
| NIH project number | 5R01HL170260-02 |
| Project ID | NIH-10909367 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 specific genetic mutations that can be targeted by the base editing therapy.
✕Less likely to help
Patients with forms of muscular dystrophy other than Duchenne muscular dystrophy may not benefit from this research.
What this research studies
This research focuses on developing a controllable base editing therapy for Duchenne muscular dystrophy (DMD), a severe muscle degenerative disease. The approach utilizes adeno-associated virus (AAV) vectors to deliver precise genetic corrections without causing harmful DNA breaks. By targeting the specific mutations that lead to the loss of dystrophin protein, the therapy aims to restore muscle function and prevent heart failure in affected patients. The research is conducted in animal models, with the goal of translating these findings into clinical applications for human patients.
Why it matters
Potential benefit: If successful, this research could provide a safe and effective treatment option for patients with Duchenne muscular dystrophy, potentially improving their quality of life and longevity.
How similar studies have gone: Other research has shown promise in using gene editing techniques for similar genetic disorders, indicating a potential for success with this novel approach.
Where this research is happening
-
Indiana University Indianapolis
Indianapolis, United States Active
Researchers
- Principal investigator: Han, Renzhi — Indiana University Indianapolis
- Study coordinator: Han, Renzhi
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.