Seattle Wellstone Muscular Dystrophy Center
Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center - Seattle
Working on better gene-delivery treatments and trial tools for people with muscular dystrophies, especially Duchenne and FSHD.
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 ID | NIH-11190903 on NIH RePORTER |
What this research studies
From a patient perspective, this center develops and tests improved AAV-based gene-delivery approaches meant to reach skeletal and heart muscle. The team works on safer, more effective micro-dystrophin designs and novel vector strategies to deliver larger or more powerful genes. They also use AAV methods to silence the harmful DUX4 gene in models of facioscapulohumeral muscular dystrophy (FSHD) and build long-term clinical and MRI datasets to guide future trials. Together these lab, animal, and human-cohort efforts aim to speed the path from lab findings to real clinical trials.
Who could benefit from this research
Good fit: Ideal candidates include people with muscular dystrophies—particularly those with Duchenne muscular dystrophy or FSHD—who may join clinical cohorts, imaging studies, or future trials.
Not a fit: People without muscular dystrophy or with types not targeted by AAV gene approaches are unlikely to benefit directly from this center's work.
Why it matters
Potential benefit: If successful, this work could lead to safer, more effective gene therapies and better-designed clinical trials for multiple forms of muscular dystrophy.
How similar studies have performed: AAV-based gene therapies have shown promise in animal studies and some early human work, but delivery limits and immune responses remain challenges this center aims to overcome.
Where this research is happening
Seattle, United States
- University of Washington — Seattle, United States (Active)
Researchers
- Principal investigator: Chamberlain, Jeffrey S — University of Washington
- Study coordinator: Chamberlain, Jeffrey S
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.