Finding the best gene editing method for treating Alzheimer's disease.
Official title: Testing Optimal Gene Editor for an Alzheimer's CRISPR therapeutic.
Funded at University of California, San Diego · NIH-10746716 · Data updated 2026-11-12
This study is working on a new treatment for Alzheimer's disease using a special tool called CRISPR to change a specific gene, with the goal of slowing down the disease and giving hope for better options for patients.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Diego NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project start | September 1, 2023 |
| Project end | August 31, 2026 |
| Funding year | 2023 |
| Award (this year) | $2,112,826 |
| NIH project number | 1UF1NS134063-01 |
| Project ID | NIH-10746716 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with Alzheimer's disease, particularly those with early-onset forms or genetic predispositions such as Down syndrome.
✕Less likely to help
Patients with advanced Alzheimer's disease or those who do not have a genetic component related to APP may not benefit from this research.
What this research studies
This research aims to develop a CRISPR-based therapy for Alzheimer's disease by identifying the most effective gene editor and guide RNA combination. The approach focuses on editing a specific part of the APP gene, which plays a crucial role in the disease's progression. By modifying this gene, the researchers hope to shift the balance from harmful to beneficial protein fragments, potentially slowing down the disease's advancement. The study is designed to advance towards clinical trials, offering hope for new treatment options for patients.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking therapy that slows the progression of Alzheimer's disease.
How similar studies have gone: Previous research using CRISPR technology for genetic modifications has shown promise, indicating potential success for this novel approach in treating Alzheimer's.
Where this research is happening
-
University of California, San Diego
La Jolla, United States Active
Researchers
- Principal investigator: Roy, Subhojit — University of California, San Diego
- Study coordinator: Roy, Subhojit
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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.