Improving a gene-editing method to treat ALS caused by SOD1 mutations
Official title: Optimization of an in vivo base editing strategy to treat SOD1-linked ALS
Funded at University of Illinois at Urbana-Champaign · NIH-11034633 · Data updated 2026-11-12
This study is exploring a new way to safely change genes to turn off a harmful protein linked to ALS, using a technique that could help improve treatment options for people living with this condition.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Illinois at Urbana-Champaign NIH-funded |
| Lab location | 1 site (Champaign, United States) |
| Project start | February 16, 2024 |
| Project end | November 30, 2026 |
| Funding year | 2024 |
| Award (this year) | $151,047 |
| NIH project number | 3U01NS122102-03S1 |
| Project ID | NIH-11034633 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with ALS linked to SOD1 mutations, particularly those who are 21 years or older.
✕Less likely to help
Patients with ALS not caused by SOD1 mutations or those with advanced stages of the disease may not benefit from this research.
What this research studies
This research focuses on developing a new gene-editing technique to target and inactivate the harmful SOD1 protein associated with amyotrophic lateral sclerosis (ALS). Using a method called CRISPR base editing, the team aims to make precise changes to the DNA without causing significant damage, which is a common issue with traditional gene-editing methods. The research will involve testing this approach in a non-human primate model to better understand its effectiveness and safety before considering human trials.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment for ALS, potentially halting or reversing the progression of the disease.
How similar studies have gone: While gene-editing approaches for ALS are still emerging, preliminary studies have shown promise in targeting genetic mutations, indicating potential for success in this novel approach.
Where this research is happening
-
University of Illinois at Urbana-Champaign
Champaign, United States Active
Researchers
- Principal investigator: Gaj, Thomas — University of Illinois at Urbana-Champaign
- Study coordinator: Gaj, Thomas
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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.