CRISPR-Cas13 gene therapy for SOD1-linked ALS
Development of a CRISPR-Cas13 Gene Therapy for SOD1-Linked ALS
['FUNDING_R01'] · UNIVERSITY OF ILLINOIS AT URBANA-CHAMPAIGN · NIH-11258045
A one-time CRISPR-Cas13 treatment delivered by a harmless virus aims to lower the harmful SOD1 protein in people with SOD1-linked ALS.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF ILLINOIS AT URBANA-CHAMPAIGN (nih funded) |
| Locations | 1 site (CHAMPAIGN, UNITED STATES) |
| Trial ID | NIH-11258045 on ClinicalTrials.gov |
What this research studies
Researchers are developing a gene therapy that uses an adeno-associated virus (AAV) to deliver CRISPR-Cas13, an RNA-targeting tool, into nerve cells to reduce production of the toxic SOD1 protein that causes inherited ALS. Because Cas13 targets RNA rather than DNA, the approach is designed to avoid permanent changes to a patient’s genes and to reduce risks of DNA damage. The team will optimize delivery, specificity, and safety in lab and preclinical models before moving toward human safety testing. If those steps succeed, the goal is a longer-lasting treatment that could require fewer repeat administrations than current options.
Who could benefit from this research
Good fit: People with a confirmed SOD1 gene mutation causing familial ALS, typically early in their disease and meeting safety criteria, would be the ideal candidates.
Not a fit: People with ALS not caused by SOD1 mutations or those with very advanced disease are unlikely to benefit from this approach.
Why it matters
Potential benefit: If successful, this could provide a long-lasting way to lower toxic SOD1 and slow disease progression in people with SOD1 mutations.
How similar studies have performed: Antisense oligonucleotide therapies have successfully lowered SOD1 in patients but require repeated dosing; RNA-targeting CRISPR-Cas13 is a newer, largely untested approach 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.
Conditions: Amyotrophic Lateral Sclerosis Motor Neuron Disease