Using nonviral methods to deliver CRISPR technology for treating high cholesterol
Nonviral Delivery of CRISPR-Cas9 into Hepatocytes Combined with APAP Selection for Treatment of Familial Hypercholesterolemia
['FUNDING_R01'] · CLEMSON UNIVERSITY · NIH-10882890
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
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | CLEMSON UNIVERSITY (nih funded) |
| Locations | 1 site (CLEMSON, UNITED STATES) |
| Trial ID | NIH-10882890 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with familial hypercholesterolemia who have high cholesterol levels and are at risk for cardiovascular issues.
Not a fit: Patients without familial hypercholesterolemia or those who do not have elevated cholesterol levels may not benefit from this research.
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 performed: 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, 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.