Base Editing APOE4 for Alzheimer's Disease
Official title: Base Editing of APOE4 for Alzheimer's Disease
Funded at University of Kentucky · NIH-11427348 · Data updated 2026-11-12
AIM: test a gene-editing approach that could reduce Alzheimer's risk by correcting APOE4 to a more protective form in a way that might be used in people at risk.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Kentucky NIH-funded |
| Lab location | 1 site (Lexington, United States) |
| Project start | September 1, 2026 |
| Project end | May 31, 2031 |
| Funding year | 2026 |
| Award (this year) | $712,881 |
| NIH project number | 1R01AG103924-01 |
| Project ID | NIH-11427348 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults who carry the APOE4 allele and are at risk for Alzheimer's disease could be candidates for future translational trials.
✕Less likely to help
Individuals without APOE4 or without disease risk may not benefit from this gene-editing approach.
What this research studies
This project focuses on delivering precise DNA base edits to the APOE gene in animal models and possibly future human applications, aiming to shift from the high-risk APOE4 form toward APOE2/3. Researchers will optimize base editors and delivery methods to work in the brain and reduce Alzheimer's pathology and cognitive deficits observed in mice. The work builds on prior in vitro success and uses in vivo models to test safety, specificity, and therapeutic potential before any clinical steps. If successful, the study could pave the way for gene-editing therapies that lower Alzheimer's risk for people carrying APOE4.
Why it matters
Potential benefit: If successful, this research could lead to a novel gene-editing therapy to reduce Alzheimer's disease risk in APOE4 carriers.
How similar studies have gone: Previous in vitro edits of APOE have shown promise, with in vivo demonstrations being an important next step.
Where this research is happening
-
University of Kentucky
Lexington, United States Active
Researchers
- Principal investigator: Johnson, Lance Allen — University of Kentucky
- Study coordinator: Johnson, Lance Allen
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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.