Using CRISPR technology to reduce harmful gene expression in FSHD
CRISPR-inhibition for FSHD
This study is testing a new treatment using a special CRISPR technology to help people with facioscapulohumeral muscular dystrophy (FSHD) by reducing the harmful gene that causes the condition, with the hope of improving muscle health for those affected.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Nevada Reno NIH-funded |
| Lab location | 1 site (Reno, United States) |
| Project ID | NIH-10869942 on NIH RePORTER |
What this research studies
This research focuses on facioscapulohumeral muscular dystrophy (FSHD), a genetic condition caused by abnormal gene expression in muscle cells. The team aims to use a modified CRISPR technology to inhibit the expression of the DUX4 gene, which is responsible for the disease. By delivering this CRISPR-based therapy directly to skeletal muscles, the researchers hope to achieve long-term reduction of the harmful gene activity. The approach has shown promise in laboratory settings, indicating potential therapeutic benefits for patients.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with facioscapulohumeral muscular dystrophy (FSHD).
Not a fit: Patients with other forms of muscular dystrophy or those without a diagnosis of FSHD may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a new treatment that significantly improves muscle function and quality of life for patients with FSHD.
How similar studies have performed: Previous research using CRISPR technology for gene inhibition has shown promising results, suggesting that this approach could be effective for FSHD as well.
Where this research is happening
Reno, United States
- University of Nevada Reno — Reno, United States (Active)
Researchers
- Principal investigator: Jones, Peter L — University of Nevada Reno
- Study coordinator: Jones, Peter L
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.