Using nonviral methods to deliver CRISPR technology for treating high cholesterol
Official title: Nonviral Delivery of CRISPR-Cas9 into Hepatocytes Combined with APAP Selection for Treatment of Familial Hypercholesterolemia
Funded at Clemson University · NIH-10882890 · Data updated 2026-11-12
This study is testing a new way to help people with familial hypercholesterolemia, a genetic condition that causes high cholesterol, by using a safe method to change liver cells and lower cholesterol levels, which could lead to better heart health.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Clemson University NIH-funded |
| Lab location | 1 site (Clemson, United States) |
| Project start | September 1, 2024 |
| Project end | June 30, 2028 |
| Funding year | 2024 |
| Award (this year) | $591,640 |
| NIH project number | 1R01HL168093-01A1 |
| Project ID | NIH-10882890 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with familial hypercholesterolemia who have high cholesterol levels and are at risk for cardiovascular issues.
✕Less likely to help
Patients without familial hypercholesterolemia or those who do not have elevated cholesterol levels may not benefit from this research.
What this research studies
This research focuses on developing a new method to treat familial hypercholesterolemia, a genetic condition that leads to high cholesterol and increased risk of heart disease. The approach involves using CRISPR-Cas9 technology to edit genes in liver cells, specifically targeting the ANGPTL3 gene to lower cholesterol levels. Instead of using viral delivery methods, which can have safety concerns, this study aims to use nonviral techniques to introduce the CRISPR components into liver cells outside the body, followed by transplanting these modified cells back into the patient. This method could potentially provide a safer and more effective treatment option for individuals with this condition.
Why it matters
Potential benefit: If successful, this research could lead to a permanent reduction in cholesterol levels for patients with familial hypercholesterolemia, significantly lowering their risk of cardiovascular disease.
How similar studies have gone: While gene editing approaches have shown promise in other contexts, this specific nonviral delivery method for treating familial hypercholesterolemia is novel and has not been extensively tested.
Where this research is happening
-
Clemson University
Clemson, United States Active
Researchers
- Principal investigator: Cottle, Renee Nicole — Clemson University
- Study coordinator: Cottle, Renee Nicole
Related research
Other NIH-funded projects on the same topics.
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Using behavioral nudges to improve family screening for inherited high cholesterol
This project uses two patient-facing approaches based on behavioral economics to help people with familial hypercholesterolemia get their relatives screened for the condition.
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Improving Risk Stratification for Familial Hypercholesterolemia (FH)
A patient-facing look at a study that aims to better predict who with familial hypercholesterolemia is at highest risk for early heart disease.
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Nonviral CRISPR-Cas9 delivery to liver cells to treat familial hypercholesterolemia
A new approach uses nonviral methods to deliver CRISPR-Cas9 to liver cells and select edited cells with acetaminophen, aiming to permanently lower bad cholesterol in people with familial hypercholesterolemia.
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Implementing multi-component approaches to help blood donors with familial hypercholesterolemia(
A program to help people who donate blood and have familial hypercholesterolemia get the cholesterol-lowering treatment they need through a blood-donation center.
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Helping families find and treat inherited high cholesterol
This project tests a clinic-based program to help primary care doctors find and treat children and adults with inherited high cholesterol (familial hypercholesterolemia).
-
Familial hypercholesterolemia: closing gaps in prevention of heart disease
A new NIH grant is exploring why people with familial hypercholesterolemia often go undiagnosed or receive uneven care, with the goal of improving early detection and prevention of heart disease.
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.