New methods to correct genetic mutations causing ALS
Official title: Advanced Base and Prime Editing Strategies to Correct Common ALS-causing SOD1 Mutations
Funded at Univ of Massachusetts Med Sch Worcester · NIH-11055160 · Data updated 2026-11-12
This study is exploring new ways to fix a specific gene mutation that causes familial ALS, using advanced gene editing techniques, with the hope that it could lead to better treatments for people living with this condition.
Quick facts
| Grant type | Career grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of Massachusetts Med Sch Worcester NIH-funded |
| Lab location | 1 site (Worcester, United States) |
| Project start | December 1, 2024 |
| Project end | November 30, 2026 |
| Funding year | 2025 |
| Award (this year) | $124,246 |
| NIH project number | 1K99NS136849-01A1 |
| Project ID | NIH-11055160 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adults diagnosed with familial ALS linked to SOD1 mutations.
✕Less likely to help
Patients with sporadic ALS or those without SOD1 mutations may not benefit from this research.
What this research studies
This research focuses on developing advanced gene editing techniques to correct specific mutations in the SOD1 gene that cause familial amyotrophic lateral sclerosis (ALS). Using innovative base and prime editing strategies delivered by adeno-associated viruses (AAV), the project aims to achieve precise gene correction in laboratory models. The researchers will test these techniques in vitro and in vivo to ensure effectiveness and minimize unintended effects. If successful, this approach could lead to new treatments for patients with genetic forms of ALS.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for patients with familial ALS caused by SOD1 mutations.
How similar studies have gone: Other research has shown promise with similar gene editing approaches, indicating potential for success in this area.
Where this research is happening
-
Univ of Massachusetts Med Sch Worcester
Worcester, United States Active
Researchers
- Principal investigator: Meijboom, Katharina E — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Meijboom, Katharina E
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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.