Improving Gene Editing for Duchenne 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-11124889 · Data updated 2026-11-12
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 start | July 1, 2024 |
| Project end | June 30, 2029 |
| Funding year | 2025 |
| Award (this year) | $537,378 |
| NIH project number | 5R01NS135175-02 |
| Project ID | NIH-11124889 on NIH RePORTER |
Who could benefit from this research
✓Could help
This research is foundational for individuals with Duchenne muscular dystrophy caused by specific mutations in the Dmd gene.
✕Less likely to help
Patients with muscle conditions not related to Duchenne muscular dystrophy or different genetic causes may not directly benefit from this specific approach.
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.
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 gone: 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
-
Joslin Diabetes Center
Boston, United States Active
Researchers
- Principal investigator: Serwold, Thomas F — Joslin Diabetes Center
- Study coordinator: Serwold, Thomas F
Related research
Other NIH-funded projects on the same topics.
-
Brown-fat lipids and their mimics for better metabolism
Researchers are studying small fat-related molecules produced by brown fat to understand how they help with metabolism and may lead to new ways to treat type 2 diabetes.
-
Understanding cognitive decline and Alzheimer’s changes in people with long‑duration type 1 diabetes
A long-term study in adults with type 1 diabetes investigates why some people experience memory and thinking difficulties and brain changes typical of Alzheimer's disease.
-
Epitranscriptomics in obesity and type 2 diabetes
Researchers are studying how gene activity changes in fat tissue and other organs to better understand obesity and type 2 diabetes, with the goal of finding new ideas for treatment.
-
APOL1-Linked Kidney Transplant Outcomes Network (APOLLO) Clinical Center
This grant supports a clinical center that follows kidney transplant patients to understand how APOL1 genetics affect transplant results and living donor health.
-
Proteostasis and lipid balance in aging: SKN-1A/Nrf1 in humans
A study in a worm model explores how a human-linked protein (Nrf1) helps balance fat, protein maintenance, and aging, with potential implications for related human diseases.
-
Glucagon Pump Therapy for PBH in People After Bariatric Surgery
A new approach uses a patch pump and smart control to deliver glucagon during meal-related lows in adults who had bariatric surgery and developed dangerous hypoglycemia.
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.