Improving CRISPR technology to treat a genetic cause of frontotemporal dementia
Optimization of CRISPR genome editor and its delivery strategy for C9orf72 frontotemporal dementia
['FUNDING_U01'] · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · NIH-10746565
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
| Phase | ['FUNDING_U01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF CALIFORNIA, SAN FRANCISCO (nih funded) |
| Locations | 1 site (SAN FRANCISCO, UNITED STATES) |
| Trial ID | NIH-10746565 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are adults diagnosed with frontotemporal dementia or those who carry the genetic mutation in the C9orf72 gene.
Not a fit: Patients with frontotemporal dementia not linked to the C9orf72 gene mutation may not benefit from this research.
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 performed: 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
SAN FRANCISCO, UNITED STATES
- UNIVERSITY OF CALIFORNIA, SAN FRANCISCO — SAN FRANCISCO, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: CLELLAND, CLAIRE — UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
- Study coordinator: CLELLAND, CLAIRE
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.
Conditions: Alzheimer disease dementia, Alzheimer syndrome