Improving gene therapy for a specific type of ALS
Optimization of an in vivo base editing strategy to treat SOD1-linked ALS
This study is testing a new gene therapy for people with ALS caused by a specific gene mutation, using a safe method to change the DNA and stop the harmful protein from being made, with the hope of making the treatment more effective and safer for patients.
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-10991834 on NIH RePORTER |
What this research studies
This research focuses on enhancing a gene therapy designed to treat amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene. The approach utilizes CRISPR base editing to precisely modify DNA without causing harmful breaks, aiming to permanently stop the production of the mutant SOD1 protein. By delivering this therapy via adeno-associated virus directly to the spinal cord, the researchers hope to improve safety and effectiveness compared to existing treatments. The study builds on promising results from animal models that showed extended survival and slowed disease progression.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with familial ALS linked to SOD1 mutations.
Not a fit: Patients with sporadic ALS or those with mutations not related to SOD1 may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a long-lasting treatment option for patients with SOD1-linked ALS, potentially improving their quality of life and extending survival.
How similar studies have performed: Previous research using CRISPR-based gene editing has shown promise in animal models, indicating potential for success in this novel approach.
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.