Bicistronic 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-11397452 · Data updated 2026-11-12
A patient-facing overview of a first-in-class gene therapy approach using two genes delivered by AAV to help people with LGMDR9.
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 | 2026 |
| Award (this year) | $279,921 |
| NIH project number | 5P50HD117373-03 |
| Project ID | NIH-11397452 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are patients with LGMDR9 who could participate in future clinical testing or contribute samples for related studies.
✕Less likely to help
People without LGMDR9 or those not eligible for future trials may not benefit from this specific research.
What this research studies
This project aims to finish pre-clinical safety and proof-of-concept work to file a pre-IND with the FDA for a bicistronic AAV gene therapy targeting LGMDR9. It compares two-gene therapy to single-gene therapies to see which better reverses muscle weakness and loss in a disease model. The team will optimize the formulation and assess safety to prepare for human testing, with the goal of one day restoring muscle size and function. If successful, this work could pave the way for a new treatment option for patients living with LGMDR9.
Why it matters
Potential benefit: If successful, the therapy could slow or reverse muscle loss and improve strength for people with LGMDR9.
How similar studies have gone: Previous gene therapy approaches for muscular dystrophy have shown promise, but bicistronic strategies are still being explored for superior outcomes.
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
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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.
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Stopping Muscle Scarring in Muscular Dystrophies
This project explores how muscle scarring, called fibrosis, develops in muscular dystrophies like Duchenne MD, hoping to find new ways to help muscles heal better.
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.