Modulating immune response to improve in-body gene editing for muscular dystrophy
Official title: Modulating the adaptive immune response to enhance the efficiency of in vivo gene editing in muscle
Funded at Joslin Diabetes Center · NIH-11337447 · Data updated 2026-11-12
A study tests a gene-editing approach delivered by a virus to repair the dystrophin gene in muscle, aiming to help people with Duchenne muscular dystrophy.
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 start | July 1, 2024 |
| Project end | June 30, 2029 |
| Funding year | 2026 |
| Award (this year) | $545,534 |
| NIH project number | 5R01NS135175-03 |
| Project ID | NIH-11337447 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are patients with DMD who could benefit from therapies aiming to restore Dystrophin through muscle-targeted gene editing.
✕Less likely to help
Patients without access to viral gene-delivery options or with conditions that preclude gene editing may not benefit.
What this research studies
This project looks at using AAV-delivered CRISPR-Cas9 to fix the Dmd gene in muscle fibers, heart muscle, and muscle stem cells in living animals. It also explores how the immune system might respond to the bacterial Cas9 protein and strategies to reduce any harmful reactions. By improving delivery and persistence of the gene-editing effect, the research seeks to restore Dystrophin production and strengthen muscle function. Findings could inform future therapies that directly address the genetic basis of Duchenne muscular dystrophy in patients.
Why it matters
Potential benefit: If successful, this approach could lead to durable restoration of Dystrophin and improved muscle strength for people with DMD.
How similar studies have gone: Prior in vivo gene-editing work has shown promise in restoring Dystrophin in animal models, with ongoing work to address immune and delivery challenges.
Where this research is happening
-
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.