Nonviral CRISPR-Cas9 delivery to liver cells to treat familial hypercholesterolemia
Official title: Nonviral Delivery of CRISPR-Cas9 into Hepatocytes Combined with APAP Selection for Treatment of Familial Hypercholesterolemia
Funded at Clemson University · NIH-11370567 · Data updated 2026-11-12
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.
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 | 2026 |
| Award (this year) | $624,177 |
| NIH project number | 5R01HL168093-03 |
| Project ID | NIH-11370567 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with familial hypercholesterolemia who are eligible for hepatocyte-based therapies and willing to pursue a cell-based treatment approach.
✕Less likely to help
Patients not eligible for hepatocyte transplantation or those with contraindications to cell-based therapies may not benefit.
What this research studies
This project focuses on treating familial hypercholesterolemia by editing the ANGPTL3 gene in liver cells using nonviral delivery, then transplanting the edited cells back into patients. Researchers plan to grow and edit hepatocytes outside the body to reduce immune-related issues and improve safety. A novel selection method using acetaminophen will enrich for successfully edited cells before transplantation. The work combines gene editing, cell therapy, and a liver-directed delivery strategy to lower LDL cholesterol and triglycerides.
Why it matters
Potential benefit: If successful, this could provide a durable reduction of LDL cholesterol and triglycerides, lowering cardiovascular risk for people with FH.
How similar studies have gone: Previous approaches have shown promise in gene editing and cell-based therapies, but this specific nonviral ex vivo hepatocyte strategy with acetaminophen selection is novel.
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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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).
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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.
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Better heart-risk prediction for inherited high cholesterol
This project aims to give people with familial high cholesterol clearer, more personalized estimates of their future heart disease risk using genetic information, cholesterol measures, and other health factors.
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.