Improving gene editing in muscle to treat Duchenne muscular dystrophy
Modulating the adaptive immune response to enhance the efficiency of in vivo gene editing in muscle
This study is exploring a new way to use gene editing to help people with Duchenne muscular dystrophy (DMD) by fixing the faulty gene that causes the condition, which could lead to better muscle function and less damage during activities.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Joslin Diabetes Center NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-10981883 on NIH RePORTER |
What this research studies
This research focuses on enhancing the effectiveness of gene editing techniques to treat Duchenne muscular dystrophy (DMD), a severe muscle-wasting condition. By using adeno-associated virus (AAV) to deliver CRISPR-Cas9 technology, the project aims to repair the defective Dmd gene responsible for DMD. The approach seeks to not only restore the production of the essential Dystrophin protein in muscle tissues but also to ensure long-lasting effects by targeting muscle stem cells. Patients may benefit from improved muscle function and reduced damage during physical activity.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Duchenne muscular dystrophy who may benefit from gene editing therapies.
Not a fit: Patients with other forms of muscular dystrophy or those without a diagnosis of Duchenne muscular dystrophy may not receive any benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment that restores muscle function in patients with Duchenne muscular dystrophy.
How similar studies have performed: Previous research has shown promise in using AAV-mediated gene editing approaches, indicating potential for success in this novel application.
Where this research is happening
Boston, United States
- Joslin Diabetes Center — Boston, United States (Active)
Researchers
- Principal investigator: Serwold, Thomas F — Joslin Diabetes Center
- Study coordinator: Serwold, Thomas F
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.