Investigating new treatments for frontotemporal dementia and ALS using gene editing
Official title: C9orf72 frontotemporal dementia (FTD) and amyotrophic lateral sclerosis(ALS): using patient cells and CRISPR to reveal therapeutic approaches
Funded at J. David Gladstone Institutes · NIH-11089394 · Data updated 2026-11-12
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
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | J. David Gladstone Institutes NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project start | May 1, 2021 |
| Project end | April 30, 2026 |
| Funding year | 2025 |
| Award (this year) | $694,575 |
| NIH project number | 5R01AG072052-03 |
| Project ID | NIH-11089394 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with frontotemporal dementia or amyotrophic lateral sclerosis, particularly those with the C9orf72 genetic mutation.
✕Less likely to help
Patients without the C9orf72 mutation or those with other forms of dementia or motor neuron diseases may not benefit from this research.
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.
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 gone: 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
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J. David Gladstone Institutes
San Francisco, United States Active
Researchers
- Principal investigator: Conklin, Bruce R — J. David Gladstone Institutes
- Study coordinator: Conklin, Bruce R
Related research
Other NIH-funded projects on the same topics.
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Investigating how DNA damage and repair mechanisms are altered in ALS and frontotemporal dementia.
This study is looking at how problems with DNA repair might contribute to conditions like ALS and frontotemporal dementia, and it’s testing ways to fix these issues in cells from patients to help develop new treatments.
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C9orf72 ALS/FTD Prevent: prepping for future treatment
This grant aims to get ready for a preventive treatment for people who carry the C9orf72 genetic change that can lead to ALS or frontotemporal dementia.
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Understanding how a genetic repeat causes harmful protein production in ALS/FTD
Aimed at uncovering how a genetic repeat in the C9orf72 gene triggers abnormal protein production that damages nerve cells in ALS and FTD.
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How C9orf72 RNA repeats make toxic proteins in ALS and FTD
This research looks for how a repeated RNA sequence in the C9orf72 gene creates toxic proteins that can harm people with ALS or FTD.
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Cell and tissue changes that lead to sporadic ALS and frontotemporal dementia
This project looks at how physical changes inside cells and in their tissue can make TDP‑43 protein clump in people with sporadic ALS and frontotemporal dementia.
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Understanding how certain genetic repeats cause neurodegenerative diseases
This study is looking into how a specific genetic change in the C9orf72 gene causes harmful proteins to form, which are linked to ALS and Frontotemporal Dementia, and it aims to understand how this happens in nerve cells to help find better ways to tackle these conditions.
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.