Improving CRISPR technology to treat a genetic cause of frontotemporal dementia
Official title: Optimization of CRISPR genome editor and its delivery strategy for C9orf72 frontotemporal dementia
Funded at University of California, San Francisco · NIH-10746565 · Data updated 2026-11-12
This study is exploring a new way to use CRISPR technology to fix a gene linked to frontotemporal dementia and ALS, aiming to help patients by delivering treatment directly to the brain.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project start | August 18, 2023 |
| Project end | July 31, 2025 |
| Funding year | 2023 |
| Award (this year) | $869,959 |
| NIH project number | 1U01NS134062-01 |
| Project ID | NIH-10746565 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adults diagnosed with frontotemporal dementia or those who carry the genetic mutation in the C9orf72 gene.
✕Less likely to help
Patients with frontotemporal dementia not linked to the C9orf72 gene mutation may not benefit from this research.
What this research studies
This research focuses on developing a CRISPR genome editing approach to target the C9orf72 gene, which is linked to frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The team has created a dual sgRNA strategy that can effectively remove the harmful repeat expansion in the gene while preserving its essential functions. Additionally, they have engineered a silica nanocapsule platform that can deliver these genome editors across the blood-brain barrier, potentially allowing for treatment directly in the brain. This innovative method aims to provide a new therapeutic option for patients suffering from these neurodegenerative diseases.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment for frontotemporal dementia, offering hope to patients with this currently untreatable condition.
How similar studies have gone: This approach is novel, but similar gene-editing strategies have shown promise in other genetic disorders, indicating potential for success.
Where this research is happening
-
University of California, San Francisco
San Francisco, United States Active
Researchers
- Principal investigator: Clelland, Claire — University of California, San Francisco
- 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.