Gene Therapy for ALS
Official title: Spinal Subpial Gene Delivery for Treatment of Amyotrophic Lateral Sclerosis
Funded at University of California, San Diego · NIH-11128828 · Data updated 2026-11-12
This project is exploring a new gene therapy delivered to the spine to help stop or slow down Amyotrophic Lateral Sclerosis (ALS) in its early stages.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Diego NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project start | September 28, 2022 |
| Project end | July 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $636,840 |
| NIH project number | 5R01NS130104-04 |
| Project ID | NIH-11128828 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for future human studies would likely be adults with early-stage or pre-symptomatic ALS caused by the SOD1 gene mutation.
✕Less likely to help
Patients with advanced ALS or forms of ALS not linked to the SOD1 gene mutation may not directly benefit from this specific gene therapy approach.
What this research studies
For people living with ALS, finding effective treatments is a critical need. This project is exploring a new way to deliver gene therapy directly to the spinal cord, which is where the nerve cells affected by ALS are located. Researchers are using a special virus, AAV9, to carry a gene-silencing tool that targets the mutated SOD1 gene, a known cause of some forms of ALS. The goal is to protect nerve cells and prevent the disease from getting worse, building on promising results seen in animal models. This work aims to define how long the treatment effect lasts and to study potential toxicity, generating crucial data for future human applications.
Why it matters
Potential benefit: If successful, this approach could offer a novel way to slow or halt the progression of ALS, especially when started early in the disease course.
How similar studies have gone: Previous work and preliminary data in animal models have shown this gene delivery method to be highly effective in blocking disease development or progression.
Where this research is happening
-
University of California, San Diego
La Jolla, United States Active
Researchers
- Principal investigator: Marsala, Martin — University of California, San Diego
- Study coordinator: Marsala, Martin
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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.