One‑time gene editing of liver ASGR1 to lower heart disease risk
Targeted editing of ASGR1 for cardiovascular diseases
This project develops a one-time gene-editing treatment that targets a liver protein (ASGR1) to lower blood fats and reduce heart disease risk for people with high triglycerides.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Indiana University Indianapolis NIH-funded |
| Lab location | 1 site (Indianapolis, United States) |
| Project ID | NIH-11301027 on NIH RePORTER |
What this research studies
The team is creating a single-dose "one-shot" therapy that uses base editors packaged in engineered viral-like particles to change the ASGR1 gene in liver cells. They will test this approach in New Zealand White rabbits to measure effects on triglycerides, total cholesterol, and other heart-disease markers. Researchers will monitor safety, liver effects, and any off-target edits as part of the preclinical evaluation. Promising results in animals would support moving toward human testing in the future.
Who could benefit from this research
Good fit: Ultimately, the intended patients would be people with persistent high triglycerides or elevated non‑HDL cholesterol and residual cardiovascular risk despite current therapies.
Not a fit: Patients whose heart disease is driven primarily by non-lipid causes or who already have well-controlled lipid levels are unlikely to benefit from this therapy.
Why it matters
Potential benefit: If successful, this could become a durable, possibly one-time treatment to lower harmful lipids and reduce cardiovascular disease risk.
How similar studies have performed: Human genetics and animal studies link ASGR1 loss to lower lipids and reduced heart risk, and gene-editing plus eVLP delivery are promising approaches but remain largely preclinical.
Where this research is happening
Indianapolis, United States
- Indiana University Indianapolis — Indianapolis, United States (Active)
Researchers
- Principal investigator: Han, Renzhi — Indiana University Indianapolis
- Study coordinator: Han, Renzhi
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.