Gene Editing for Familial High Cholesterol
Nonviral Delivery of CRISPR-Cas9 into Hepatocytes Combined with APAP Selection for Treatment of Familial Hypercholesterolemia
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 ID | NIH-11047691 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: 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.
Not a fit: Patients without familial hypercholesterolemia or those with other forms of high cholesterol would not directly benefit from this specific gene-editing approach.
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 performed: 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, United States
- Clemson University — Clemson, United States (Active)
Researchers
- Principal investigator: Cottle, Renee Nicole — Clemson University
- Study coordinator: Cottle, Renee Nicole
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.