Muscular dystrophy gene therapy center in Seattle
Official title: Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center - Seattle
Funded at University of Washington · NIH-11395621 · Data updated 2026-11-12
A Seattle-based program studies gene therapy approaches to muscular dystrophy to help patients in the Northwest and beyond.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Washington NIH-funded |
| Lab location | 1 site (Seattle, United States) |
| Project start | May 7, 2014 |
| Project end | August 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $1,647,619 |
| NIH project number | 5P50AR065139-13 |
| Project ID | NIH-11395621 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with muscular dystrophy, including FSHD, or those at risk who might be eligible for future trials and gene therapy approaches.
✕Less likely to help
Some patients with non-eligible muscular dystrophy subtypes or those not pursuing gene therapy may not directly benefit.
What this research studies
This program focuses on advancing muscular dystrophy therapies, particularly through AAV-based gene delivery. Researchers will optimize AAV variants for muscles, reduce immune responses to dystrophin constructs, and explore delivery strategies that reach skeletal and heart muscle. They will also push forward foundations for FSHD clinical trials, including long-term clinical and MRI data collection to map disease progression. The work includes translational and pre-clinical studies in mouse and large animal models to prepare for future human trials.
Why it matters
Potential benefit: If successful, patients with muscular dystrophy could benefit from safer and more effective gene therapies and clearer pathways to participating in future trials.
How similar studies have gone: Prior work in gene therapy for muscular dystrophy has shown promise in animals and early human studies, but translational progress remains ongoing.
Where this research is happening
-
University of Washington
Seattle, United States Active
Researchers
- Principal investigator: Chamberlain, Jeffrey S — University of Washington
- Study coordinator: Chamberlain, Jeffrey S
Related research
Other NIH-funded projects on the same topics.
-
Advancing manufacturing and release testing for a Duchenne gene editing therapy
A project to optimize how a dual-vector AAV gene editing treatment for Duchenne muscular dystrophy is made and tested, so it can move toward clinical use.
-
Understanding why Duchenne Muscular Dystrophy worsens and how to stop it
A research team at USC is studying Duchenne muscular dystrophy to find out why the disease continues to progress and how to interrupt harmful changes in muscle tissue.
-
Endocrine role of intramuscular fat in muscular dystrophy
This grant studies how fat that builds up inside muscles in Duchenne and Becker muscular dystrophy may release hormones that affect overall health, especially metabolism.
-
Stopping steroid-related muscle and bone wasting in Duchenne muscular dystrophy
Researchers are testing ways to protect muscles and bones in people with Duchenne muscular dystrophy who take long-term steroid medicines.
-
Curation of muscular dystrophy genes to improve diagnosis and care
A grant to help doctors better interpret genetic tests for muscular dystrophy and myopathy so patients get clearer diagnoses and faster access to therapies.
-
MR clues to how nNOS affects energy use and blood flow in Becker and Duchenne muscular dystrophy
This project looks at how advanced MRI tests can track muscle energy and blood flow changes in people with Becker and Duchenne muscular dystrophy as new gene therapies are explored.
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.