Gene therapy for patients with a rare form of muscular dystrophy
Official title: IND-enabling studies of non-immunogenic gene therapy for ultra-rare DMD patients excluded from dystrophin trials
Funded at University of Pennsylvania · NIH-10947911 · Data updated 2026-11-12
This study is testing a new gene therapy for people with very rare types of Duchenne muscular dystrophy who can't join other trials, using a special virus to deliver a modified gene that helps protect against side effects, with the hope of improving muscle function and overall quality of life.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pennsylvania NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | August 1, 2024 |
| Project end | July 31, 2027 |
| Funding year | 2024 |
| Award (this year) | $1,541,019 |
| NIH project number | 1U01NS134672-01A1 |
| Project ID | NIH-10947911 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with ultra-rare forms of Duchenne muscular dystrophy who have not responded to or are ineligible for current dystrophin-targeted therapies.
✕Less likely to help
Patients with common forms of Duchenne muscular dystrophy or those who do not have a genetic mutation suitable for this gene therapy may not benefit from this research.
What this research studies
This research focuses on developing a non-immunogenic gene therapy specifically for patients with ultra-rare forms of Duchenne muscular dystrophy (DMD) who have been excluded from existing dystrophin trials. The approach involves using adeno-associated virus (AAV) vectors to deliver a modified version of the dystrophin gene, aiming to minimize immune responses that can lead to serious side effects. By utilizing preclinical models, the research seeks to ensure safety and efficacy before moving to human trials, ultimately aiming to improve muscle function and quality of life for affected individuals.
Why it matters
Potential benefit: If successful, this research could provide a new treatment option for patients with ultra-rare DMD, potentially improving their muscle function and overall quality of life.
How similar studies have gone: While gene therapy approaches for DMD have been explored, this specific non-immunogenic strategy for ultra-rare forms is novel and has not been widely tested in clinical settings.
Where this research is happening
-
University of Pennsylvania
Philadelphia, United States Active
Researchers
- Principal investigator: Stedman, Hansell H — University of Pennsylvania
- Study coordinator: Stedman, Hansell H
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.