Gene Editing for Familial 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-11047691 · Data updated 2026-11-12
This project explores a new way to use gene editing to help people with familial hypercholesterolemia, a condition causing very high cholesterol.
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 | 2025 |
| Award (this year) | $652,388 |
| NIH project number | 5R01HL168093-02 |
| Project ID | NIH-11047691 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this type of therapy would be individuals diagnosed with familial hypercholesterolemia who are seeking long-term solutions for managing their high cholesterol.
✕Less likely to help
Patients without familial hypercholesterolemia or those with other forms of high cholesterol would not directly benefit from this specific gene-editing approach.
What this research studies
Familial hypercholesterolemia (FH) is a common condition that leads to dangerously high cholesterol and early heart disease. This work aims to permanently lower cholesterol and triglyceride levels by using CRISPR-Cas9 gene editing to modify a specific gene called ANGPTL3. Instead of using traditional viral methods, which have limitations, this approach involves editing liver cells outside the body and then transplanting them back. A unique method using acetaminophen (fever medicine) will help ensure that only the successfully edited cells thrive in the liver.
Why it matters
Potential benefit: If successful, this approach could offer a permanent way to reduce high cholesterol and triglyceride levels, significantly lowering the risk of heart disease for individuals with familial hypercholesterolemia.
How similar studies have gone: While gene editing for other conditions has shown promise, this specific nonviral ex vivo approach combined with acetaminophen selection for familial hypercholesterolemia is a novel strategy being explored.
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.