Using CRISPR technology to develop a gene therapy for ALS caused by SOD1 mutations
Development of a CRISPR-Cas13 Gene Therapy for SOD1-Linked ALS
This study is testing a new gene therapy that uses a special technology to help reduce harmful proteins in people with ALS caused by a specific gene mutation, aiming to provide a safer and more effective treatment option in the future.
Quick facts
| Grant type | R01 grant |
|---|---|
| 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-11005731 on NIH RePORTER |
What this research studies
This research focuses on developing a novel gene therapy using CRISPR-Cas13 technology to target and reduce the harmful effects of mutant SOD1 proteins in patients with amyotrophic lateral sclerosis (ALS). By utilizing RNA-targeting CRISPR systems, the approach aims to safely lower the levels of the mutant protein without causing DNA damage, which is a risk associated with traditional gene-editing methods. The study will explore the effectiveness and safety of this therapy in preclinical models before considering human trials. Patients may benefit from a more effective and less invasive treatment option for SOD1-linked ALS.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with familial ALS caused by SOD1 mutations.
Not a fit: Patients with sporadic forms of ALS or those without SOD1 mutations may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a safer and more effective treatment option for patients with SOD1-linked ALS.
How similar studies have performed: While gene therapy approaches for ALS are being explored, the use of CRISPR-Cas13 for this specific application is relatively novel and has not been widely tested in clinical settings.
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.