Understanding Gene Therapy for Muscular Dystrophy
Project 3 - Walter_Vandenborne
This project uses advanced imaging to better understand how gene therapy helps patients with Duchenne muscular dystrophy and a rare form of limb girdle muscular dystrophy.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Florida NIH-funded |
| Lab location | 1 site (Gainesville, United States) |
| Project ID | NIH-11130234 on NIH RePORTER |
What this research studies
We are looking closely at how gene therapy affects patients with Duchenne muscular dystrophy (DMD) who have received a recently approved treatment. Our team uses special MRI scans to see changes in muscles and the heart, which helps us track how the disease progresses and how well the therapy is working. We are also studying a less common condition called limb girdle muscular dystrophy (LGMDR3/R5) to learn more about its progression and find new ways to help these patients. By collecting information from these patients, we hope to find better ways to monitor treatments and identify new targets for future gene therapies.
Who could benefit from this research
Good fit: This project focuses on individuals with Duchenne muscular dystrophy who have received micro-dystrophin gene therapy and patients with sarcoglycanopathy (LGMDR3/R5).
Not a fit: Patients without Duchenne muscular dystrophy or sarcoglycanopathy (LGMDR3/R5) would not directly benefit from this specific research.
Why it matters
Potential benefit: If successful, this work could lead to improved ways to monitor gene therapy effectiveness and identify new treatment targets for muscular dystrophies.
How similar studies have performed: Micro-dystrophin gene therapy has shown promise and recently received FDA approval, while this project aims to further understand its impact and characterize a rarer condition.
Where this research is happening
Gainesville, United States
- University of Florida — Gainesville, United States (Active)
Researchers
- Principal investigator: Walter, Glenn — University of Florida
- Study coordinator: Walter, Glenn
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.