CRISPR-based gene editor to slow Alzheimer's by tweaking APP
Official title: Testing Optimal Gene Editor for an Alzheimer's CRISPR therapeutic.
Funded at University of California, San Diego · NIH-11583686 · Data updated 2026-11-12
A gene-editing approach using CRISPR to modify a specific part of the APP gene in brain cells aims to slow Alzheimer's disease and move toward human trials.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| 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, 2028 |
| Funding year | 2026 |
| Award (this year) | $667,495 |
| NIH project number | 4U01NS134063-02 |
| Project ID | NIH-11583686 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with Alzheimer’s disease or related dementia, including those with early-onset forms or Down syndrome where APP is implicated.
✕Less likely to help
People without Alzheimer’s disease or with non-APP related dementia are unlikely to benefit.
What this research studies
This project focuses on refining a CRISPR-based therapy that targets APP to reduce harmful pathways in Alzheimer’s disease. It plans to identify the best genome-editor and guide RNA combination and ready the approach for pre-IND discussions. The work involves testing in models to ensure safety and efficacy, with an emphasis on making a therapy that could be used in people with Alzheimer’s in the future.
Why it matters
Potential benefit: If successful, this approach could lead to a new, disease-modifying therapy for Alzheimer's that shifts APP processing toward protective pathways.
How similar studies have gone: Previous gene-editing strategies show safety and efficacy in models, but this exact APP-targeting approach is advancing toward clinical feasibility.
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
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.