Gene editing to target a mutation causing ALS and frontotemporal dementia
CRISPR-Cas9 editing in C9orf72 patient derived iPSC
This study is exploring how to use a special gene-editing tool to fix a harmful mutation in a gene linked to ALS and frontotemporal dementia, with the hope of finding new treatments that could help patients feel better.
Quick facts
| Grant type | Career grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project ID | NIH-10883603 on NIH RePORTER |
What this research studies
This research focuses on using CRISPR-Cas9 gene editing technology to address a specific genetic mutation in the C9orf72 gene, which is linked to amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The project aims to develop methods to correct or silence the harmful effects of this mutation in patient-derived cells. By evaluating different editing strategies, the research seeks to understand how effectively these approaches can restore normal cellular function and reduce disease symptoms. Patients may benefit from advancements in gene therapy that could lead to new treatment options for these neurodegenerative diseases.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with ALS or FTD who carry the C9orf72 mutation.
Not a fit: Patients without the C9orf72 mutation or those with other forms of ALS or FTD may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to groundbreaking treatments that directly target the genetic cause of ALS and FTD, potentially halting or reversing disease progression.
How similar studies have performed: While gene editing approaches are still emerging, preliminary studies have shown promise in similar applications, indicating potential for success in this area.
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.