Delivering a complete dystrophin protein to muscle cells for treating Duchenne muscular dystrophy.
Delivery of Full Length Dystrophin to Muscle Cells for DMD Gene Therapy
This study is exploring a new gene therapy that aims to help people with Duchenne muscular dystrophy by delivering a complete version of a protein called dystrophin to their muscle cells, which could improve muscle function and health.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Gigamune, INC. NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project ID | NIH-10695412 on NIH RePORTER |
What this research studies
This research focuses on developing a gene therapy approach to treat Duchenne muscular dystrophy (DMD) by delivering the full-length dystrophin protein to muscle cells. The researchers are utilizing a novel lentiviral platform called GigaLentiTM, which allows for efficient and targeted gene delivery to specific cell types. By using in vitro models, they aim to create a method that can effectively replace the missing dystrophin protein in patients with DMD, potentially improving muscle function and overall health.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Duchenne muscular dystrophy, particularly those with mutations in the dystrophin gene.
Not a fit: Patients with forms of muscular dystrophy other than Duchenne muscular dystrophy may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a long-term treatment option that restores muscle function in patients with Duchenne muscular dystrophy.
How similar studies have performed: Other research has shown promise in using lentiviral vectors for gene therapy, indicating potential success for this novel approach.
Where this research is happening
San Francisco, United States
- Gigamune, INC. — San Francisco, United States (Active)
Researchers
- Principal investigator: Johnson, David Scott — Gigamune, INC.
- Study coordinator: Johnson, David Scott
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.