Investigating new treatments for frontotemporal dementia and ALS using gene editing
C9orf72 frontotemporal dementia (FTD) and amyotrophic lateral sclerosis(ALS): using patient cells and CRISPR to reveal therapeutic approaches
['FUNDING_R01'] · J. DAVID GLADSTONE INSTITUTES · NIH-11089394
This study is looking at ways to help people with frontotemporal dementia and ALS by using a special gene-editing tool to fix a harmful gene that causes these diseases.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | J. DAVID GLADSTONE INSTITUTES (nih funded) |
| Locations | 1 site (SAN FRANCISCO, UNITED STATES) |
| Trial ID | NIH-11089394 on ClinicalTrials.gov |
What this research studies
This research focuses on understanding and potentially treating frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS), which are severe neurodegenerative diseases linked to a genetic mutation in the C9orf72 gene. By utilizing CRISPR gene editing technology, the researchers aim to modify or silence the harmful genetic changes in patient-derived cells. The study will explore three innovative methods to target the disease-causing gene, with the goal of correcting the underlying pathology in neurons relevant to these conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals diagnosed with frontotemporal dementia or amyotrophic lateral sclerosis, particularly those with the C9orf72 genetic mutation.
Not a fit: Patients without the C9orf72 mutation or those with other forms of dementia or motor neuron diseases may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to groundbreaking therapies that effectively treat or even reverse the effects of FTD and ALS.
How similar studies have performed: While gene editing approaches are being explored in various contexts, this specific application targeting the C9orf72 gene is novel and has not been extensively tested in clinical settings.
Where this research is happening
SAN FRANCISCO, UNITED STATES
- J. DAVID GLADSTONE INSTITUTES — SAN FRANCISCO, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: CONKLIN, BRUCE R — J. DAVID GLADSTONE INSTITUTES
- Study coordinator: CONKLIN, BRUCE R
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