Gene delivery to treat amyotrophic lateral sclerosis (ALS)
Spinal Subpial Gene Delivery for Treatment of Amyotrophic Lateral Sclerosis
['FUNDING_R01'] · UNIVERSITY OF CALIFORNIA, SAN DIEGO · NIH-10880697
This study is exploring a new gene therapy that aims to help people with ALS by targeting a specific gene that causes the disease, and it's being tested in animals to see how well it works and if it's safe before it can be used in humans.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF CALIFORNIA, SAN DIEGO (nih funded) |
| Locations | 1 site (LA JOLLA, UNITED STATES) |
| Trial ID | NIH-10880697 on ClinicalTrials.gov |
What this research studies
This research investigates a novel approach to treating amyotrophic lateral sclerosis (ALS) by delivering a specific gene therapy using AAV9 vectors. The therapy aims to silence the mutated SOD1 gene, which is known to contribute to ALS, through a targeted delivery method in the spinal cord. The study will assess the effectiveness and safety of this treatment in animal models, focusing on both pre-symptomatic and early-symptomatic stages of the disease. By understanding the duration of treatment effects and potential toxicity, the research aims to pave the way for future clinical applications in humans.
Who could benefit from this research
Good fit: Ideal candidates for this research are adults diagnosed with sporadic or hereditary forms of ALS, particularly those in the early stages of the disease.
Not a fit: Patients with advanced ALS or those who do not have the SOD1 mutation may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment option for ALS patients, potentially slowing or halting disease progression.
How similar studies have performed: While gene therapy approaches for ALS are still emerging, preliminary studies have shown promise in similar methodologies, indicating potential for success.
Where this research is happening
LA JOLLA, UNITED STATES
- UNIVERSITY OF CALIFORNIA, SAN DIEGO — LA JOLLA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: MARSALA, MARTIN — UNIVERSITY OF CALIFORNIA, SAN DIEGO
- Study coordinator: MARSALA, MARTIN
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.