AAV-CRISPR therapy for a genetic cause of ALS/FTD
Official title: Research Project II: AAV-CRISPR to Treat Amyotrophic Lateral Sclerosis
Funded at University of California Berkeley · NIH-11369870 · Data updated 2026-11-12
A team is testing a gene-editing approach to potentially treat a genetic form of ALS and a related dementia by targeting the C9orf72 gene with a precise CRISPR method.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California Berkeley NIH-funded |
| Lab location | 1 site (Berkeley, United States) |
| Project start | May 15, 2023 |
| Project end | April 30, 2028 |
| Funding year | 2026 |
| Award (this year) | $427,885 |
| NIH project number | 5U19NS132303-04 |
| Project ID | NIH-11369870 on NIH RePORTER |
Who could benefit from this research
✓Could help
Individuals with the C9orf72-related neurodegenerative disease could in the future be candidates for this therapy once approved.
✕Less likely to help
Patients without the C9orf72 expansion or those not eligible for gene therapy may not benefit.
What this research studies
This project aims to develop a gene-editing therapy that targets a known genetic cause of ALS and FTD, using an AAV delivery system. Researchers will test allele-specific CRISPR/Cas9 edits in neurons and evaluate delivery, editing efficiency, and safety in animal models. The work plans IND-enabling studies and will inform future clinical trial design for people with the C9orf72-related disease. The study may pave the way for a one-time genetic intervention that could alter disease progression in affected individuals.
Why it matters
Potential benefit: If successful, this work could lead to a gene therapy approach that slows or stops disease progression for people with C9orf72-related ALS/FTD.
How similar studies have gone: Prior gene-editing efforts in neurodegenerative disease show promise, but this is a stepped, IND-enabling approach that remains exploratory.
Where this research is happening
-
University of California Berkeley
Berkeley, United States Active
Researchers
- Principal investigator: Clelland, Claire — University of California Berkeley
- Study coordinator: Clelland, Claire
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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.