Gene therapy for Limb Girdle Muscular Dystrophy Type R9
(Project 1) Bicistronic Gene Therapies for the Muscular Dystrophies
This study is testing a new gene therapy for people with Limb Girdle Muscular Dystrophy Type R9 that aims to replace the faulty gene and help rebuild muscle strength, with hopes of improving muscle function and slowing down the disease.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Research Inst Nationwide Children's Hosp NIH-funded |
| Lab location | 1 site (Columbus, United States) |
| Project ID | NIH-10992526 on NIH RePORTER |
What this research studies
This research aims to develop a novel gene therapy that targets Limb Girdle Muscular Dystrophy Type R9 (LGMDR9) by using a bicistronic approach, which involves delivering two genes instead of one. The therapy is designed to not only replace the mutated gene responsible for the disease but also to promote muscle rebuilding and strength restoration. Pre-clinical studies will be conducted to assess the safety and effectiveness of this therapy before seeking FDA approval. Patients may benefit from a treatment that could potentially halt disease progression and improve muscle function.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Limb Girdle Muscular Dystrophy Type R9.
Not a fit: Patients with other forms of muscular dystrophy or those who do not have LGMDR9 may not benefit from this specific research.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option that prevents further muscle degeneration and promotes muscle recovery for patients with LGMDR9.
How similar studies have performed: Previous research has shown promise in gene therapy for muscular dystrophies, making this approach a potentially viable option for LGMDR9.
Where this research is happening
Columbus, United States
- Research Inst Nationwide Children's Hosp — Columbus, United States (Active)
Researchers
- Principal investigator: Martin, Paul Taylor — Research Inst Nationwide Children's Hosp
- Study coordinator: Martin, Paul Taylor
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.