Blood-delivered gene editing to convert APOE4 to APOE3 for Alzheimer's
Optimization and preclinical evaluation of brain permeant SE-CRISPR to edit ApoE4 after IV infusion in AD models
This project is testing an IV-delivered gene-editing approach aimed at people with Alzheimer's or at high risk who carry the APOE-ε4 gene variant.
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 ID | NIH-11245742 on NIH RePORTER |
What this research studies
Researchers are using tiny synthetic exosome particles to carry CRISPR base-editing tools across the blood-brain barrier after a single IV infusion. In mouse models of Alzheimer’s that carry the human APOE4 gene, they aim to change the harmful APOE4 sequence into the more benign APOE3 form inside brain cells. The team has early data showing this approach can increase APOE3 mRNA in mouse brains and plans further optimization and safety testing. This is preclinical work done at UCLA and is meant to lay the groundwork for future human trials.
Who could benefit from this research
Good fit: The approach is targeted to adults who have or are at high risk for Alzheimer's and who carry the APOE-ε4 genetic variant.
Not a fit: People who do not carry the APOE-ε4 variant or whose dementia has non-APOE-related causes are unlikely to benefit from this specific approach.
Why it matters
Potential benefit: If successful, the approach could reduce the genetic risk from APOE4 and slow or prevent Alzheimer’s-related brain damage when given systemically.
How similar studies have performed: Related gene-editing and APOE-targeting approaches have shown promise in animal studies, but human safety and effectiveness remain untested.
Where this research is happening
Los Angeles, United States
- University of California Los Angeles — Los Angeles, United States (Active)
Researchers
- Principal investigator: John, Varghese — University of California Los Angeles
- Study coordinator: John, Varghese
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 below.