Gene editing to turn off faulty SOD1 in ALS
Optimization of an in vivo base editing strategy to treat SOD1-linked ALS
A one-time gene-editing approach aims to permanently turn off faulty SOD1 genes in people with SOD1-linked ALS.
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 ID | NIH-11235824 on NIH RePORTER |
What this research studies
This project is developing a gene-editing treatment that uses precise 'base editors' to change DNA and stop the production of the toxic SOD1 protein that causes some inherited ALS. The treatment is delivered to the spinal cord using an adeno-associated virus (AAV) so it reaches the motor neurons that degenerate in ALS. In aggressive SOD1 mouse models the approach slowed disease and extended survival, and researchers are now refining the method to improve safety and effectiveness before moving toward human testing. The team is focused on reducing unintended edits and minimizing risks from the viral delivery to make the therapy safer for people.
Who could benefit from this research
Good fit: People with ALS who have a confirmed disease-causing mutation in the SOD1 gene would be the ideal candidates.
Not a fit: People with ALS not caused by SOD1 mutations, or those with very advanced nerve loss, are unlikely to benefit from this specific approach.
Why it matters
Potential benefit: If successful, this could offer a one-time, durable therapy that slows or stops disease progression for people with SOD1-linked ALS.
How similar studies have performed: Related RNA-silencing therapies have shown clinical effects but require repeated dosing, while AAV-delivered base editing produced strong benefits in SOD1 mouse models but is largely untested in humans.
Where this research is happening
Champaign, United States
- 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
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.