Editing a gene linked to Alzheimer's disease using a new delivery method
Official title: Optimization and preclinical evaluation of brain permeant SE-CRISPR to edit ApoE4 after IV infusion in AD models
Funded at University of California Los Angeles · NIH-11030024 · Data updated 2026-11-12
This study is testing a new way to change a gene linked to Alzheimer's disease in the brain, using a special delivery system to help make it safer for people at risk, and it's being done in mice that have been designed to show similar symptoms to humans.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California Los Angeles NIH-funded |
| Lab location | 1 site (Los Angeles, United States) |
| Project start | December 15, 2024 |
| Project end | November 30, 2029 |
| Funding year | 2025 |
| Award (this year) | $751,442 |
| NIH project number | 1R01AG090773-01 |
| Project ID | NIH-11030024 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals over 21 years old who carry the ApoE4 gene and are at risk for Alzheimer's disease.
✕Less likely to help
Patients who do not carry the ApoE4 gene or who are not at risk for Alzheimer's disease may not benefit from this research.
What this research studies
This research focuses on developing a novel therapy to edit the ApoE4 gene, which is a significant genetic risk factor for Alzheimer's disease. The approach utilizes a new delivery system that allows for the infusion of CRISPR technology directly into the brain, aiming to convert the harmful ApoE4 variant into a benign form, ApoE3. By using synthetic exosomes to carry the CRISPR components, the therapy seeks to cross the blood-brain barrier effectively, potentially reducing the risk of Alzheimer's in patients. The research is conducted in preclinical models, specifically engineered mice that mimic human Alzheimer's disease.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment that significantly lowers the risk of developing Alzheimer's disease for individuals with the ApoE4 gene.
How similar studies have gone: While gene editing approaches are being explored in various contexts, this specific application of SE-CRISPR for Alzheimer's disease is novel and has not been extensively tested in humans.
Where this research is happening
-
University of California Los Angeles
Los Angeles, United States Active
Researchers
- Principal investigator: John, Varghese — University of California Los Angeles
- Study coordinator: John, Varghese
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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.