Gene therapy for Limb Girdle Muscular Dystrophy Type R9
Official title: (Project 1) Bicistronic Gene Therapies for the Muscular Dystrophies
Funded at Research Inst Nationwide Children's Hosp · NIH-10992526 · Data updated 2026-11-12
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 start | August 15, 2024 |
| Project end | July 31, 2028 |
| Funding year | 2024 |
| Award (this year) | $300,029 |
| NIH project number | 1P50HD117373-01A1 |
| Project ID | NIH-10992526 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Limb Girdle Muscular Dystrophy Type R9.
✕Less likely to help
Patients with other forms of muscular dystrophy or those who do not have LGMDR9 may not benefit from this specific research.
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.
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 gone: 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
-
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
Related research
Other NIH-funded projects on the same topics.
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Noninvasive breathing imaging to guide care for people with muscular dystrophy
A noninvasive imaging tool called electrical impedance tomography (EIT) will be studied to help decide when to start noninvasive ventilation for people with muscular dystrophy.
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Bicistronic gene therapy for limb girdle muscular dystrophy type R9
A patient-facing overview of a first-in-class gene therapy approach using two genes delivered by AAV to help people with LGMDR9.
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Understanding why scarring happens in muscular dystrophy muscles
Researchers are studying why scar tissue forms in muscles affected by muscular dystrophy and how to slow or reverse it in patients.
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Addressing Shortcomings of AAV Gene Therapies for Muscular Dystrophy
A national research center is working to improve AAV-based gene therapies for Duchenne and related muscular dystrophies so that patients may get safer, more effective treatments.
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Two-gene AAV therapy for Limb-Girdle Muscular Dystrophy R9
Developing a two-gene AAV gene therapy to replace the faulty gene and rebuild muscle for people with LGMDR9 (Limb Girdle Muscular Dystrophy Type R9).
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Improving Gene Therapies for Muscular Dystrophies
This center is working to improve gene therapies for Duchenne and Limb Girdle Muscular Dystrophy, aiming to make them safer and more effective for patients.
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.