Targeting FSHD with a new gene technology
Official title: CRISPR-inhibition for FSHD
Funded at University of Nevada Reno · NIH-11125886 · Data updated 2026-11-12
This project explores a new gene technology to turn off the harmful gene causing Facioscapulohumeral muscular dystrophy (FSHD).
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 start | August 4, 2021 |
| Project end | June 30, 2026 |
| Funding year | 2025 |
| Award (this year) | $517,866 |
| NIH project number | 5R01AR079884-05 |
| Project ID | NIH-11125886 on NIH RePORTER |
Who could benefit from this research
✓Could help
This research is focused on understanding and treating Facioscapulohumeral muscular dystrophy (FSHD).
✕Less likely to help
Patients without Facioscapulohumeral muscular dystrophy (FSHD) would not directly benefit from this specific research.
What this research studies
Facioscapulohumeral muscular dystrophy (FSHD) is a genetic muscle disease where a specific gene, DUX4, is turned on incorrectly in muscle cells, leading to muscle weakness. This project is developing a new gene technology, called CRISPR inhibition (CRISPRi), to specifically turn off the DUX4 gene. CRISPRi works by using a modified gene-editing tool to repress, or silence, the DUX4 gene without permanently changing the DNA. The goal is to deliver this technology safely and effectively to muscle cells to reduce the harmful effects of DUX4. Researchers are working to make sure this treatment can be delivered efficiently to muscles and provide long-lasting benefits.
Why it matters
Potential benefit: If successful, this approach could lead to a new treatment that reduces muscle damage and improves quality of life for individuals with FSHD.
How similar studies have gone: Early laboratory work has shown that this gene technology can successfully turn off the harmful gene in FSHD muscle cells.
Where this research is happening
-
University of Nevada Reno
Reno, United States Active
Researchers
- Principal investigator: Jones, Peter L — University of Nevada Reno
- Study coordinator: Jones, Peter L
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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.