Controlled base-editing therapy for Duchenne muscular dystrophy
Controllable base editing therapy for DMD
Researchers are developing a safer gene base-editing approach delivered by AAV9 to correct the mutation that causes Duchenne muscular dystrophy while reducing DNA damage and immune risks.
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 ID | NIH-11145130 on NIH RePORTER |
What this research studies
From a patient's view, the team is working on a precision 'base editor' that can change single DNA letters in the dystrophin gene to restore function in heart and skeletal muscle. They plan to deliver the editor using AAV9 vehicles and add control systems to limit how long the editor is active, lowering the chance of unwanted DNA changes. The work focuses on solving safety problems seen with older CRISPR methods, such as cutting both DNA strands, high viral doses, and immune reactions. Successful steps in animal models will guide future testing toward human clinical trials.
Who could benefit from this research
Good fit: Ideal candidates would be people with Duchenne muscular dystrophy caused by specific single-letter (point) mutations in the dystrophin gene that can be fixed by adenine base editors.
Not a fit: Patients whose DMD is caused by large deletions, duplications, or other mutation types not addressable by base editing, or those with advanced irreversible muscle damage, may not benefit from this approach.
Why it matters
Potential benefit: If successful, this could permanently correct certain DMD mutations in muscle and heart cells, potentially preserving strength and reducing life-threatening heart failure.
How similar studies have performed: Early animal studies have shown efficient correction of DMD point mutations with base editors, but human use remains unproven and safety challenges still need to be solved.
Where this research is happening
Indianapolis, United States
- 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 below.