Improving Gene Editing for Duchenne Muscular Dystrophy
Modulating the adaptive immune response to enhance the efficiency of in vivo gene editing in muscle
This work explores how to make gene editing more effective for Duchenne muscular dystrophy by managing the body's natural defenses.
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-11124889 on NIH RePORTER |
What this research studies
Duchenne muscular dystrophy (DMD) is a serious condition where muscles weaken due to a faulty gene. Our approach uses a special delivery method, called AAV, to bring gene-editing tools directly into muscle cells. These tools aim to fix the faulty gene, allowing the body to produce a vital protein called Dystrophin. We are working to understand and control the body's immune response to these gene-editing tools, which is crucial for making the treatment work better and last longer. The goal is to restore muscle function and protect both skeletal muscles and the heart.
Who could benefit from this research
Good fit: This research is foundational for individuals with Duchenne muscular dystrophy caused by specific mutations in the Dmd gene.
Not a fit: Patients with muscle conditions not related to Duchenne muscular dystrophy or different genetic causes may not directly benefit from this specific approach.
Why it matters
Potential benefit: If successful, this work could lead to more effective and lasting gene-editing treatments for Duchenne muscular dystrophy, potentially improving muscle strength and heart health.
How similar studies have performed: Previous work from this lab and others has shown promising results in animal models, demonstrating the feasibility of this gene-editing strategy.
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.