Improving Gene Delivery for Muscle Conditions
Development and optimization of skeletal muscle delivery vehicles
This project aims to create better ways to deliver gene therapies to muscles, which could help people with genetic muscle diseases like muscular dystrophy.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cincinnati Childrens Hosp Med Ctr NIH-funded |
| Lab location | 1 site (Cincinnati, United States) |
| Project ID | NIH-11134685 on NIH RePORTER |
What this research studies
Genetic muscle diseases, such as muscular dystrophies, are challenging to treat because muscles are spread throughout the body and hard to reach effectively. While current gene therapies for Duchenne muscular dystrophy use AAV viruses to deliver corrective genes, this project explores a new approach. Researchers are working to adapt lentiviruses, which have different properties, to deliver genes specifically to muscle cells. They are testing if natural muscle-specific proteins can help these viruses fuse with muscle cells, potentially making gene delivery more efficient and targeted.
Who could benefit from this research
Good fit: This foundational research is not directly recruiting patients, but future clinical trials based on this work would likely target individuals with genetic muscle diseases like Duchenne muscular dystrophy.
Not a fit: Patients without genetic muscle diseases or those whose conditions are not treatable by gene therapy would not directly benefit from this specific research.
Why it matters
Potential benefit: If successful, this work could lead to more effective and safer gene therapies for patients with Duchenne muscular dystrophy and other genetic muscle diseases.
How similar studies have performed: While AAV-based gene therapies are currently in clinical trials, this project explores a novel approach using lentiviruses and muscle-specific proteins for gene delivery.
Where this research is happening
Cincinnati, United States
- Cincinnati Childrens Hosp Med Ctr — Cincinnati, United States (Active)
Researchers
- Principal investigator: Millay, Douglas Paul — Cincinnati Childrens Hosp Med Ctr
- Study coordinator: Millay, Douglas Paul
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.